Wednesday, December 26, 2018

CMS Gene Language for 2019: Existed Nov 21-Dec 12, 2018

6. All genomic sequencing procedures and molecular
multianalyte assays (e.g., CPT codes 81410-81471), many
multianalyte assays with algorithmic analyses (e.g., CPT codes
81493-81599, 0004M-XXXXM), and many Proprietary Laboratory
Analyses (PLA) (e.g., CPT codes 0001U-XXXXU) are DNA or RNA
analytic methods that simultaneously assay multiple genes or
genetic regions.  A physician shall not additionally separately
report testing for the same gene or genetic region by a
different methodology (e.g., CPT codes 81105-81408, 81479,
88364-88377).  CMS payment policy does not allow separate
payment for multiple methods to test for the same analyte.


7. A Tier 1 or Tier 2 molecular pathology procedure CPT 
code shall not be reported with a genomic sequencing procedure, 
molecular multianalyte assay, multianalyte assay with 
algorithmic analysis, or proprietary laboratory analysis CPT 
code where the CPT code descriptor includes testing for the 
analyte described by the Tier 1 or Tier 2 molecular pathology 

8. If one laboratory procedure evaluates multiple genes 
utilizing a next generation sequencing procedure, the laboratory 
shall report only one unit of service of one genomic sequencing 
procedure, molecular multianalyte assay, multianalyte assay with 
algorithmic analysis, or proprietary laboratory analysis CPT 
code.  If no CPT code accurately describes the procedure 
performed, the laboratory shall report CPT code 81479 (unlisted 
molecular pathology procedure) with one unit of service.  The 
laboratory shall not report multiple individual CPT codes 
describing the component test results.  If a single procedure is 
performed, only one HCPCS/CPT code with one unit of service may 
be reported for the procedure. 

9. Procedure-to-procedure edits bundling two Tier 1 
molecular pathology procedure CPT codes describe procedures that 
should not routinely be performed and reported together.  For 
example CPT code 81292 describes full sequence gene analysis of 
MLH1, and CPT code 81294 describes duplication/deletion variant 
gene analysis of MLH1.  In evaluating a patient with colon 
carcinoma (vs. constitutional genetic disorder), it may be 
appropriate to perform duplication/deletion testing if the 
disease variant(s) is (are) not identified by performing full 
gene sequencing.  The same principle applies to other code pair 
combinations of testing for the same gene (e.g., 81295/81297, 

Thursday, December 13, 2018

December 2018 Parrish CGM Case Filed in Court

Medicare Coverage Class Action Filed Against Health and Human Services Secretary on Behalf of Diabetes Patients

Case here (36pp):

Lowenstein Sandler and Parrish Law Offices filed a class action in the U.S. District Court for the District of Columbia today against Alex Azar in his capacity as Secretary of the U.S. Department of Health and Human Services (HHS) on behalf of diabetic Medicare recipients denied reimbursement coverage for use of medically prescribed continuous glucose monitors (CGMs). The complaint seeks a declaration that federal law requires HHS to reimburse for CGMs; compensation for Medicare beneficiaries’ past out-of-pocket costs to purchase these devices, the claims for which HHS improperly denied; and a ruling to establish a precedent for all Medicare beneficiaries filing claims for CGMs in the future.
This action asserts that HHS has routinely ignored past court and administrative law decisions and improperly denied CGM users’ Medicare reimbursement requests on the contention that CGMs do not qualify as durable medical equipment (DME) that is primarily or customarily used to serve a medical purpose. As the plaintiffs detail in their complaint, HHS’s decisions violate several federal statutes and are arbitrary and capricious, disregard Congressional intent, and ignore the fact that these devices meet Medicare coverage requirements. Plaintiffs ask the court to reverse HHS’s erroneous past decisions and issue an order finding that a CGM and its related supplies do, in fact, serve an essential medical purpose.
Jointly filed by the Parrish Law Offices and Lowenstein Sandler, the class action complaint asserts that HHS’s coverage denials have serious consequences for patients’ health outcomes and have an impact on health care costs at both the individual and national level. For many who suffer from diabetes, the only way to adequately and consistently monitor their glucose levels is through a CGM, which continually tests those levels and transmits detailed data to patients and caregivers. Such data, especially in instances of dramatic changes in glucose levels, can mean the difference between just-in-time treatment and serious, even fatal, complications that can result from the delays in care that are all too common with older, more traditional monitoring methods.
“The Department has continued to deny diabetes patients potentially lifesaving and cost-reducing medical devices and care,” said James Pistorino, a lawyer with the Parrish Law Offices and lead representative for the plaintiff class. “Nonsensically, the department has claimed that CGMs do not serve a medical purpose and are not necessary, despite their being an FDA-approved and verified method for preventing those with diabetes from facing dangerous consequences, such as falling into a diabetic coma or, even worse, death.”
More than 98 percent of private insurance companies cover CGMs, which are FDA-approved, lifesaving devices providing a proven, 24/7 way to test glucose levels and
prevent complications and, potentially, death from diabetes. Nevertheless, in too many instances, Medicare will not cover CGM use, which means that patients must pay for this costly equipment on their own. Those who simply cannot afford the cost must forgo the device completely and risk acute health complications.
“Health and Human Services has a duty to live up to its name and provide coverage to the distressed populations it claims to assist,” said Jeffrey Blumenfeld, partner at Lowenstein Sandler. “Providing adequate medical coverage to those in need makes common and ethical sense and, in the case of CGMs, is also exactly what Congress intended. By improving and saving lives and boosting positive health care outcomes, CGMs also save the general public, the government, and the economy in terms of short- and long-term costs.”
Plaintiffs Carol A. Lewis and Douglas B. Sargent are both filing the suit after being denied coverage from the Medicare Appeals Council and are represented by James Pistorino and Debra Parrish of the Parrish Law Offices in conjunction with Jeffrey Blumenfeld and Gavin J. Rooney of Lowenstein Sandler.
To see a copy of the complaint, click here.

Debra M. Parrish
Parrish Law Offices
Phone: 412-561-6250

Quinn local file as: 2018 Court Parrish 1213 CGM Case Filed Against HHS 36pp.pdf

Monday, December 10, 2018

Cloud Posting: Five Amazingly Complex SEP1 Documents

As of 2018.12.10, links to five amazingly complex SEP1 process documents. 

Also in one ZIP file here.

This is a document file only.

I wrote actual essays on CMS SEP1 in August (first data release) and in November (performance of academic hospitals).

For a contrasting 2018 article by Walkey on the value of keeping it simple in Sepsis measures, here.

63 page CMS measure guide

256 questions (73pp)

62 page webinar slides

4 page SEP1 tabular summary

4 page SEP1 flow chart diagram

Sunday, December 9, 2018

1997 Balanced Budget Act, Section 4554: Uniform CMS Lab Claims Policies


    (a) Selection of Regional Carriers.--
            (1) In general.--The Secretary of Health and Human Services 
        (in this section referred to as the ``Secretary'') shall--
                    (A) divide the United States into no more than 5 
                regions, and
                    (B) designate a single carrier for each such region,
        for the purpose of payment of claims under part B of title XVIII 
        of the Social Security Act with respect to clinical diagnostic 
        laboratory tests furnished on or after such date (not later than 
        July 1, 1999) as the Secretary specifies.
            (2) Designation.--In designating such carriers, the 
        Secretary shall consider, among other criteria--
                    (A) a carrier's timeliness, quality, and experience 
                in claims processing, and

[[Page 111 STAT. 461]]

                    (B) a carrier's capacity to conduct electronic data 
                interchange with laboratories and data matches with 
                other carriers.
            (3) Single data resource.--The Secretary shall select one of 
        the designated carriers to serve as a central statistical 
        resource for all claims information relating to such clinical 
        diagnostic laboratory tests handled by all the designated 
        carriers under such part.
            (4) Allocation of claims.--The allocation of claims for 
        clinical diagnostic laboratory tests to particular designated 
        carriers shall be based on whether a carrier serves the 
        geographic area where the laboratory specimen was collected or 
        other method specified by the Secretary.
            (5) Secretarial exclusion.--Paragraph (1) shall not apply 
        with respect to clinical diagnostic laboratory tests furnished 
        by physician office laboratories if the Secretary determines 
        that such offices would be unduly burdened by the application of 
        billing responsibilities with respect to more than one carrier.

    (b) Adoption of National Policies for Clinical Laboratory Tests 
            (1) In general.--Not later than January 1, 1999, the 
        Secretary shall first adopt, consistent with paragraph (2), 
        national coverage and administrative policies for clinical 
        diagnostic laboratory tests under part B of title XVIII of the 
        Social Security Act, using a negotiated rulemaking process under 
        subchapter III of chapter 5 of title 5, United States Code.
            (2) Considerations in design of national policies.--The 
        policies under paragraph (1) shall be designed to promote 
        program integrity and national uniformity and simplify 
        administrative requirements with respect to clinical diagnostic 
        laboratory tests payable under such part in connection with the 
                    (A) Beneficiary information required to be submitted 
                with each claim or order for laboratory tests.
                    (B) The medical conditions for which a laboratory 
                test is reasonable and necessary (within the meaning of 
                section 1862(a)(1)(A) of the Social Security Act).
                    (C) The appropriate use of procedure codes in 
                billing for a laboratory test, including the unbundling 
                of laboratory services.
                    (D) The medical documentation that is required by a 
                medicare contractor at the time a claim is submitted for 
                a laboratory test in accordance with section 1833(e) of 
                the Social Security Act.
                    (E) Recordkeeping requirements in addition to any 
                information required to be submitted with a claim, 
                including physicians' obligations regarding such 
                    (F) Procedures for filing claims and for providing 
                remittances by electronic media.
                    (G) Limitation on frequency of coverage for the same 
                tests performed on the same individual.
            (3) Changes in laboratory policies pending adoption of 
        national policy.--During the period that begins on the date of 
        the enactment of this Act and ends on the date the Secretary 
        first implements national policies pursuant to regulations 
        promulgated under this subsection, a carrier under such

[[Page 111 STAT. 462]]

        part may implement changes relating to requirements for the 
        submission of a claim for clinical diagnostic laboratory tests.
            (4) Use of interim policies.--After the date the Secretary 
        first implements such national policies, the Secretary shall 
        permit any carrier to develop and implement interim policies of 
        the type described in paragraph (1), in accordance with 
        guidelines established by the Secretary, in cases in which a 
        uniform national policy has not been established under this 
        subsection and there is a demonstrated need for a policy to 
        respond to aberrant utilization or provision of unnecessary 
        tests. Except as the Secretary specifically permits, no policy 
        shall be implemented under this paragraph for a period of longer 
        than 2 years.
            (5) Interim national policies.--After the date the Secretary 
        first designates regional carriers under subsection (a), the 
        Secretary shall establish a process under which designated 
        carriers can collectively develop and implement interim national 
        policies of the type described in paragraph (1). No such policy 
        shall be implemented under this paragraph for a period of longer 
        than 2 years.
            (6) Biennial review process.--Not less often than once every 
        2 years, the Secretary shall solicit and review comments 
        regarding changes in the national policies established under 
        this subsection. As part of such biennial review process, the 
        Secretary shall specifically review and consider whether to 
        incorporate or supersede interim policies developed under 
        paragraph (4) or (5). Based upon such review, the Secretary may 
        provide for appropriate changes in the national policies 
        previously adopted under this subsection.
            (7) Requirement and notice.--The Secretary shall ensure that 
        any policies adopted under paragraph (3), (4), or (5) shall 
        apply to all laboratory claims payable under part B of title 
        XVIII of the Social Security Act, and shall provide for advance 
        notice to interested parties and a 45-day period in which such 
        parties may submit comments on the proposed change.

    (c) Inclusion of Laboratory Representative on Carrier Advisory 
Committees.--The Secretary shall direct that any advisory committee 
established by a carrier to advise such carrier with respect to coverage 
and administrative policies under part B of title XVIII of the Social 
Security Act shall include an individual to represent the independent 
clinical laboratories and such other laboratories as the Secretary deems 
appropriate. The Secretary shall consider recommendations from national 
and local organizations that represent independent clinical laboratories 
in such selection.

Wednesday, November 28, 2018

FDA's Own Guidance to Issuing Guidance

For over a decade, quite a few legal scholars have commented on the FDA's propensity to issue policy as "guidance documents" rather than as regulation. 

As noting in a newly published request for information on prescription drug software (83FR58574), FDA has a regulation which lays out its own rules for issuing these guidance documents.  See 21 CFR 10.115.

FDA has two types of Guidance Documents, Level 1 or Level 2.  Level 1 sets out FDA interpretation of statute, regulation, or has complex scientific content.  Any other guidance document is "Level 2."

The regulation was established by 64 FR 56477, 13pp, 9/19/2000, with a trivial amendment regarding a web link in 2018, 83 FR 13416, 3/29/2018.

Code of Federal Regulations]
[Title 21, Volume 1]
[Revised as of April 1, 2018]
[CITE: 21CFR10.115]

Subpart B--General Administrative Procedures
Sec. 10.115 Good guidance practices.
(a) What are good guidance practices? Good guidance practices (GGP's) are FDA's policies and procedures for developing, issuing, and using guidance documents.
(b) What is a guidance document? (1) Guidance documents are documents prepared for FDA staff, applicants/sponsors, and the public that describe the agency's interpretation of or policy on a regulatory issue.
(2) Guidance documents include, but are not limited to, documents that relate to: The design, production, labeling, promotion, manufacturing, and testing of regulated products; the processing, content, and evaluation or approval of submissions; and inspection and enforcement policies.
(3) Guidance documents do not include: Documents relating to internal FDA procedures, agency reports, general information documents provided to consumers or health professionals, speeches, journal articles and editorials, media interviews, press materials, warning letters, memoranda of understanding, or other communications directed to individual persons or firms.
(c) What other terms have a special meaning? (1) "Level 1 guidance documents" include guidance documents that:
(i) Set forth initial interpretations of statutory or regulatory requirements;
(ii) Set forth changes in interpretation or policy that are of more than a minor nature;
(iii) Include complex scientific issues; or
(iv) Cover highly controversial issues.
(2) "Level 2 guidance documents" are guidance documents that set forth existing practices or minor changes in interpretation or policy. Level 2 guidance documents include all guidance documents that are not classified as Level 1.
(3) "You" refers to all affected parties outside of FDA.
(d) Are you or FDA required to follow a guidance document? (1) No. Guidance documents do not establish legally enforceable rights or responsibilities. They do not legally bind the public or FDA.
(2) You may choose to use an approach other than the one set forth in a guidance document. However, your alternative approach must comply with the relevant statutes and regulations. FDA is willing to discuss an alternative approach with you to ensure that it complies with the relevant statutes and regulations.
(3) Although guidance documents do not legally bind FDA, they represent the agency's current thinking. Therefore, FDA employees may depart from guidance documents only with appropriate justification and supervisory concurrence.
(e) Can FDA use means other than a guidance document to communicate new agency policy or a new regulatory approach to a broad public audience? The agency may not use documents or other means of communication that are excluded from the definition of guidance document to informally communicate new or different regulatory expectations to a broad public audience for the first time. These GGP's must be followed whenever regulatory expectations that are not readily apparent from the statute or regulations are first communicated to a broad public audience.
(f) How can you participate in the development and issuance of guidance documents? (1) You can provide input on guidance documents that FDA is developing under the procedures described in paragraph (g) of this section.
(2) You can suggest areas for guidance document development. Your suggestions should address why a guidance document is necessary.
(3) You can submit drafts of proposed guidance documents for FDA to consider. When you do so, you should mark the document "Guidance Document Submission" and submit it to Division of Dockets Management (HFA-305), 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. If you wish to submit the draft of a proposed guidance document electronically, submit it through at Docket No. FDA-2013-S-0610. It is only necessary to submit one copy.
(4) You can, at any time, suggest that FDA revise or withdraw an already existing guidance document. Your suggestion should address why the guidance document should be revised or withdrawn and, if applicable, how it should be revised.
(5) Once a year, FDA will publish, both in the Federal Register and on the Internet, a list of possible topics for future guidance document development or revision during the next year. You can comment on this list (e.g., by suggesting alternatives or making recommendations on the topics that FDA is considering).
(6) To participate in the development and issuance of guidance documents through one of the mechanisms described in paragraphs (f)(1), (f)(2), or (f)(4) of this section, you should contact the center or office that is responsible for the regulatory activity covered by the guidance document.
(7) If FDA agrees to draft or revise a guidance document, under a suggestion made under paragraphs (f)(1), (f)(2), (f)(3) or (f)(4) of this section, you can participate in the development of that guidance document under the procedures described in paragraph (g) of this section.
(g) What are FDA's procedures for developing and issuing guidance documents? (1) FDA's procedures for the development and issuance of Level 1 guidance documents are as follows:
(i) Before FDA prepares a draft of a Level 1 guidance document, FDA can seek or accept early input from individuals or groups outside the agency. For example, FDA can do this by participating in or holding public meetings and workshops.
(ii) After FDA prepares a draft of a Level 1 guidance document, FDA will:
(A) Publish a notice in the Federal Register announcing that the draft guidance document is available;
(B) Post the draft guidance document on the Internet and make it available in hard copy; and
(C) Invite your comment on the draft guidance document. Paragraph (h) of this section tells you how to submit your comments.
(iii) After FDA prepares a draft of a Level 1 guidance document, FDA also can:
(A) Hold public meetings or workshops; or
(B) Present the draft guidance document to an advisory committee for review.
(iv) After providing an opportunity for public comment on a Level 1 guidance document, FDA will:
(A) Review any comments received and prepare the final version of the guidance document that incorporates suggested changes, when appropriate;
(B) Publish a notice in the Federal Register announcing that the guidance document is available;
(C) Post the guidance document on the Internet and make it available in hard copy; and
(D) Implement the guidance document.
(v) After providing an opportunity for comment, FDA may decide that it should issue another draft of the guidance document. In this case, FDA will follow the steps in paragraphs (g)(1)(ii), (g)(1)(iii), and (g)(1)(iv) of this section.
(2) FDA will not seek your comment before it implements a Level 1 guidance document if the agency determines that prior public participation is not feasible or appropriate.
(3) FDA will use the following procedures for developing and issuing Level 1 guidance documents under the circumstances described in paragraph (g)(2) of this section:
(i) After FDA prepares a guidance document, FDA will:
(A) Publish a notice in the Federal Register announcing that the guidance document is available;
(B) Post the guidance document on the Internet and make it available in hard copy;
(C) Immediately implement the guidance document; and
(D) Invite your comment when it issues or publishes the guidance document. Paragraph (h) of this section tells you how to submit your comments.
(ii) If FDA receives comments on the guidance document, FDA will review those comments and revise the guidance document when appropriate.
(4) FDA will use the following procedures for developing and issuing Level 2 guidance documents:
(i) After it prepares a guidance document, FDA will:
(A) Post the guidance document on the Internet and make it available in hard copy;
(B) Immediately implement the guidance document, unless FDA indicates otherwise when the document is made available; and
(C) Invite your comment on the Level 2 guidance document. Paragraph (h) of this section tells you how to submit your comments.
(ii) If FDA receives comments on the guidance document, FDA will review those comments and revise the document when appropriate. If a version is revised, the new version will be placed on the Internet.
(5) You can comment on any guidance document at any time. Paragraph (h) of this section tells you how to submit your comments. FDA will revise guidance documents in response to your comments when appropriate.
(h) How should you submit comments on a guidance document? (1) If you choose to submit comments on any guidance document under paragraph (g) of this section, you must send them to the Division of Dockets Management (HFA-305), 5630 Fishers Lane, rm. 1061, Rockville, MD 20852.
(2) Comments should identify the docket number on the guidance document, if such a docket number exists. For documents without a docket number, the title of the guidance document should be included.
(3) Comments will be available to the public in accordance with FDA's regulations on submission of documents to the Division of Dockets Management specified in 10.20(j).
(i) What standard elements must FDA include in a guidance document? (1) A guidance document must:
(i) Include the term "guidance,"
(ii) Identify the center(s) or office(s) issuing the document,
(iii) Identify the activity to which and the people to whom the document applies,
(iv) Prominently display a statement of the document's nonbinding effect,
(v) Include the date of issuance,
(vi) Note if it is a revision to a previously issued guidance and identify the document that it replaces, and
(vii) Contain the word "draft" if the document is a draft guidance.
(2) Guidance documents must not include mandatory language such as "shall," "must," "required," or "requirement," unless FDA is using these words to describe a statutory or regulatory requirement.
(3) When issuing draft guidance documents that are the product of international negotiations (e.g., guidances resulting from the International Conference on Harmonisation), FDA need not apply paragraphs (i)(1) and (i)(2) of this section. However, any final guidance document issued according to this provision must contain the elements in paragraphs (i)(1) and (i)(2) of this section.
(j) Who, within FDA, can approve issuance of guidance documents? Each center and office must have written procedures for the approval of guidance documents. Those procedures must ensure that issuance of all documents is approved by appropriate senior FDA officials.
(k) How will FDA review and revise existing guidance documents? (1) The agency will periodically review existing guidance documents to determine whether they need to be changed or withdrawn.
(2) When significant changes are made to the statute or regulations, the agency will review and, if appropriate, revise guidance documents relating to that changed statute or regulation.
(3) As discussed in paragraph (f)(3) of this section, you may at any time suggest that FDA revise a guidance document.
(l) How will FDA ensure that FDA staff are following GGP's? (1) All current and new FDA employees involved in the development, issuance, or application of guidance documents will be trained regarding the agency's GGP's.
(2) FDA centers and offices will monitor the development and issuance of guidance documents to ensure that GGP's are being followed.
(m) How can you get copies of FDA's guidance documents? FDA will make copies available in hard copy and, as feasible, through the Internet.
(n) How will FDA keep you informed of the guidance documents that are available? (1) FDA will maintain on the Internet a current list of all guidance documents. New documents will be added to this list within 30 days of issuance.
(2) Once a year, FDA will publish in the Federal Register its comprehensive list of guidance documents. The comprehensive list will identify documents that have been added to the list or withdrawn from the list since the previous comprehensive list.
(3) FDA's guidance document lists will include the name of the guidance document, issuance and revision dates, and information on how to obtain copies of the document.
(o) What can you do if you believe that someone at FDA is not following these GGP's? If you believe that someone at FDA did not follow the procedures in this section or that someone at FDA treated a guidance document as a binding requirement, you should contact that person's supervisor in the center or office that issued the guidance document. If the issue cannot be resolved, you should contact the next highest supervisor. You can also contact the center or office ombudsman for assistance in resolving the issue. If you are unable to resolve the issue at the center or office level or if you feel that you are not making progress by going through the chain of command, you may ask the Office of the Chief Mediator and Ombudsman to become involved.
[65 FR 56477, Sept. 19, 2000, as amended at 83 FR 13416, Mar. 29, 2018]

See FDA's announcement of a new program to have pre pivotal trial meetinss with FDA about innovative (eg Bayesian) designs. August 2018.   

Tuesday, November 27, 2018

CY2016 CMS 81479 Billing by Lab

Publicly available CMS data on 81479 use in CY2016 Medicare Part B by lab.

click to enlarge
Cloud XLS here.

CMS source here.

Wednesday, November 21, 2018

MolDx Gene Panel Alert: Archive Version 11/18/2018

Molecular Test Panel Edit Alert (M00101, V4)

MolDX CPT code range affected: 81161-81408
Test Panel Definition: A predetermined set of medical tests composed of individual laboratory tests, related by medical condition, specimen type, frequency ordered, methodology or types of components to aid in the diagnosis/treatment of disease.
Palmetto considers the performance of multiple molecular biomarkers, regardless of whether the test requisition lists the tests as a panel or individually, and completed on a single sample to be a 'panel' of tests. Therefore, each panel should be registered and billed with a single CPT code and a unique MolDX identifier. Based on data analysis of MolDX claims, labs are submitting multiple biomarker 'panels' as individual tests similar to the submission of the previous stacking codes.
Example: A lab receives a patient specimen and performs the following tests:
  • CPT code 81225-CYP2C19, cv
  • CPT code 81240-F2, 20210G>A
  • CPT code 81241-F5, Leiden
The panel of three tests listed above should be registered and claims submitted with CPT code 81479 and a single MolDX ID. If the lab also performs one of the three panel’s biomarkers on patient specimens, each biomarker must also be registered and receive a unique ID to submit on claims when only that biomarker is performed. Again, a single CPT code should be billed with a unique ID.
To correct this example, the lab must register four tests and receive identifiers for MolDX claim submission:
Test #
Test Name
CPT Code
Clotting Test Panel
CYP2C19, cv
F2, 20210G>A
F5, Leiden
CYP2C19, cv
F2, 20210G>A
F2, 20210G>A
F5, Leiden
F5, Leiden
Labs should register all panels and obtain a unique MolDX identifier for each panel. If a lab does NOT perform single biomarker tests, they must notify the registry of this registration error.

Monday, November 5, 2018

How to Make a Lift/Drop Waterfall Chart with Regular Powerpoint Tables

I've seen elaborate waterfall charts that add and subtract financials in sequence but I've never made one myself.  A master deck from a client showed how.

Basically, it's a stacked bar chart where many of the bottom level bars are "white" or "transparent."   Add or subtract bars "float" above the transparent barstack below them.  Easy.

Below, the first bar is 340 and blue.  The next bar is a stacked bar with "340" in white or transparent, and then, a green bar for 30 floating on top of that.   To show a negative, for example, you'd have used instead a bar of 310 for the second bar and a red bar of 30 on top of it. 

click to enlarge
Note that these bars are slightly spaced (gap toothed) because they have a Format Data Series option of 20% gap width. 

I think the numbers floating on top are related to the "Format Data Labels" option but I'm not sure exactly how; I think by checking "Format Data Labels / Label Options / Label Contains / Value."

Note that the example has a truncated left axis (from 300 to 390).

Wednesday, October 31, 2018

October 2018: Milken Institute Conversation with Scott Gottlieb

Word document in the cloud, here.

Scott Gottlieb, Commissioner, U.S. Food and Drug Administration
Tanisha Carino, Executive Director, FasterCures, Milken Institute

Tanisha Carino:               Hello, everyone. I'm delighted to be here with Commissioner Gottlieb. For those of you that don't know Scott ... Can I call you Scott?
Scott Gottlieb:                 Yes.
Tanisha Carino:               Scott is a physician, a policy wonk, an investor, an industry expert, former investor, a cancer survivor, a husband, and a father. Now, he's the 23rd Commissioner of food and drugs. Over the last 18 months ... This is your third tour of FDA?
Scott Gottlieb:                 That's right. Yeah. Yep.
Tanisha Carino:               In your first year, you approved more new drugs, more generics. You also have had a very aggressive public health agenda that included limiting nicotine levels, regulating e-cigarettes, getting dangerous opioids off of the market. You're responsible for the fact that if I go to a McDonald's, I know how many calories I eat with a Big Mac.
Tanisha Carino:               For those of you that haven't been following the news as closely, this past week Scott was voted Time Magazine's one of 50 most influential leaders. You are accomplished, you're influential, and people also think you're a fashionista.
Tanisha Carino:               For those of you that don't follow our commissioner on Twitter, I highly suggest that you do so. Because I knew I was going to be next to him on stage today, I made sure to absolutely dress up my shoes today. I hope that I'm beating Scott at his sock game today.
Tanisha Carino:               Let's jump in because I know we don't have that much time with you. Over the course of the last 24 hours, we've heard a lot about the future of health. If there is one thing that has been a consistent theme throughout this summit, it's been that the greatest disruption and the greatest source of innovation is the idea of being patient-centered and really listening to what patients actually need.
Tanisha Carino:               You've been on three different tours of the FDA. Over the last few years, the FDA has been an incredible leader of what it means to be patient-centered. I'd love for you to first start by reflecting on how that's changed and what it means to the FDA and is this really a values-oriented strategy or how you think about that.
Scott Gottlieb:                 I think it certainly is a values strategy. I know Janet touched on some of those issues today about the changing orientation of the agency and the sense that the clinicians really go to the house that their medical officers working in medical endeavors in the pursuit of trying to improve outcomes of patients but I think we've done discrete things to try to incorporate patient perspectives into our regulatory decision making and inform our policy making, things like patient-reported outcomes, building them in as measures and as the outcomes in clinical trials.
Scott Gottlieb:                 But I think that the other big opportunity, in addition to trying to find discreet ways to bring patients into the dialogue, bring patient perspectives into the regulatory decision making process is some of the tools that we were going to have available to us to actually measure things that are meaningful to patients.
Scott Gottlieb:                 We're going to be promulgating in a couple of weeks a platform for how you can incorporate digital health tools in the context of the approval of a drug. You could get approval for a drug digital health tool delivery system, if you will. These are going to be digital tools that could help with patient compliance, could measure patient symptoms, or maybe could measure outcomes, measure physical performance in a way that it could be used for either post-market surveillance, post-market commitments, or future regulatory decision making, trying to collect information that can be used for supplement mental indications.
Scott Gottlieb:                 Right now, for example, we use a six-minute walk test as a standard measure of physical performance for a lot of clinical trials particularly for neurodegenerative diseases in children, mucopolysaccharide diseases. That's not a really great measure all the time of physical performance. Someone can come to the doctor and just have a bad day. They come in once a month or once every two weeks for a checkup and being measured on a walk test on a treadmill.
Scott Gottlieb:                 We know that, we have watches now that could potentially measure our physical performance on a daily basis and better track if we're improving, if our physical performance is improving. Obviously these need to be validated, these kinds of tools, but those are the opportunities I think that we're going to have and our goal is to try to build up out regulatory frameworks that enable these tools to come to market.
Scott Gottlieb:                 An example of looking at digital tools that could be used in conjunction with drugs, what we're looking to do there is incorporate the digital tools into the product labeling in a way that they could be regulated in the post market as part of the promotional labeling, as opposed to just having to be regulated as a pre-market medical devices and undergo pre-market review. This is going to help facilitate the development of more of these kinds of tools.
Tanisha Carino:               Scott, for us, we had a session yesterday on the future of data disrupting health. One of the aha moments was the transition from having what is unstructured data or some people called it dirty data into data that is going to be meaningful and useful. It sounds like this is the type of innovation from a regulatory perspective that's going to create more understanding of how to integrate this. Is that a fair way of understanding the path that you all are on?
Scott Gottlieb:                 I think it is. I think that if you look at a lot of the outcomes, there's objective outcomes like tumor shrinkage, objective outcomes in oncology but a lot of the measures that we use in clinical development measure physical performance or measure how patients experience symptoms.
Scott Gottlieb:                 I think that there was ways to incorporate technology that's going to allow us to gather that information in a much more objective fashion more reliably, perhaps more quickly so that if you have better tools that are more sensitive for measuring change in physical symptoms, of physical performance, you can potentially get an answer more quickly than if you have a tool that's measuring physical performance that's highly variable so you need a larger data set, you need more patients to account for the variability statistically. You need to follow them for long because you don't have a sensitive tools, so in order to measure a decline in function you need to you need to follow patients longer. Unfortunately, you're watching someone decline in function in the context of the clinical trial in order to prove something work.
Scott Gottlieb:                 I think that we're going to have more of these opportunities. If you look at what we did in Alzheimer's where we change the guidance to, before, in order to have a drug approved for Alzheimer's, you had to both have an effect on cognition as well as performance. The notion being that you had to decline enough in cognition so that it had an effect on your physical performance but the problem was, by the time your cognition declined in that setting, you were pretty advanced in the disease. By the time it declined enough to affect your physical performance, you were pretty advanced in the disease. It mitigated against trying to develop a drug for early Alzheimer's before the decline in cognition had an effect on physical performance. We put out new guidance that said that you only need to now in certain settings hit on cognition if you can measure changes in cognition.
Scott Gottlieb:                 One of the things that facilitated our ability to do that was the advent of better tools for measuring changes in cognition, not just better surveys that we used to measure cognition but better tools for actually collecting the data where we now had digital platforms for doing that, so you can collect them in a more reliable fashion and have better quality control in terms of how you collecting that information.
Tanisha Carino:               That's great. One of the questions that, for faster cures, we've been focused on this idea of patient engagement. We've been incredibly impressed with the FDA has done in terms of all of its listening sessions it's held over the last few years. Now, there's been a real surge in activity by almost every group that's out here from a patient perspective and wanting to really own the future of what is meaningful to them.
Tanisha Carino:               When you think about your review staff and you think about the talent that you have at the FDA and what's happened, do you see a meaningful difference and how they're really considering the patient and how has it really changed the culture even within the FDA?
Scott Gottlieb:                 I think the bottom line is it has changed the culture, the engagement, and the formal structures that we put in place to try to bring patients into the process has changed the culture of the agency.
Scott Gottlieb:                 The other thing we're trying to do with how we're restructuring the Office of New Drugs and the review process itself is, a medical reviewer right now at FDA does a lot of different things. They're not just doing clinical evaluation. They're not just trying to develop new guidance and be thought leaders within their fields. They're also, when the review package comes in to the FDA right now, they're the ones taking the raw data and putting it in charts and analyzing the data in statistical packages.
Scott Gottlieb:                 A medical officer shouldn't be doing that. We should be having experts who are expert in data analytics doing the initial analysis. We should have people who know how to format data, doing the formatting and assembling into standardized presentations that then the medical reviewer can use.
Scott Gottlieb:                 What we're trying to do with the reform that we're undertaking with the Office of New Drugs is actually free the Medical Review staff and create more cross-agency functions to do some of the technical aspects of the review process so that the clinical officers can be a clinical house staff so that they can focus on the clinical portion of the review so that they have more time to engage with patients and so that they have more time to engage as thought leaders within their fields.
Scott Gottlieb:                 That's the key element of what we're doing with the OND reform. I literally talked to reviewers who will explain to me that they spent a whole day trying to assemble data into a chart. There's a lot of people who know how to do that. Most of them are under the age of 25. We shouldn't have a physician doing that.
Scott Gottlieb:                 We're trying to create these kinds of cross-function units to do some of the structural work the analytical work that goes into the review. I think that's going to also ... We estimated that it's going to improve productivity by about 20% but what we're trying to do with that improved productivity now is facilitated development of many more disease-specific guidances. We committed to put out hundreds and do disease-specific guidances and also free our house staff to engage more with the external community. You're starting to see that now but that's the long-term vision.
Tanisha Carino:               I remember the days when you would never be able to meet or review stuff. Now, you have them spending all day at meetings about what's meaningful to patients in all sorts of different ways.
Tanisha Carino:               I want to touch on the fact that you spent 15 years as a resident fellow at the American Enterprise Institute. There's a long body of writing that you've done on the role of government in creating a transparency and being a force in creating efficient markets. One of the keys to transparency is the availability of information.
Tanisha Carino:               As the FDA Commissioner, how do you feel like you've moved forward in terms of the transparency the agency can have to be a good steward of what's happening in public health and inform the public of what's happening?
Scott Gottlieb:                 I actually published 826 articles. I know that only because the Senate collected them all at my confirmation hearing. The best part of my confirmation hearing was when I handed them all in, I had a hand in a list of every article I ever published. I inadvertently, and it was completely inadvertently, left three off and they found them.
Tanisha Carino:               Now, you have the exact number of what was that?
Scott Gottlieb:                 Now, I have them all.
Scott Gottlieb:                 One of the things that I talked about, the reform of the new drug approval process. One of the things that we're doing right now is creating a more structured review. You're going to start to see this first on the safety side in terms of how we review safety data coming into the agency. What we're trying to do is across all the different therapeutic divisions look at data the same way so when data comes in, we're going to have those cross-functional units analyze the data in a similar fashion, assemble the data in charts that are preferred.
Tanisha Carino:               Paper charts?
Scott Gottlieb:                 No. Digital charts that are preferred to FDA. What's facilitating this is the fully-electronic submission of data right now. We're now going to make more of the bottom line data from the review process available publicly. We started that as a pilot. Companies have been opting into it but we're going to try to make more of the bottom line data available as part of the review memo that gets ultimately promulgated.
Scott Gottlieb:                 But part of what a structure review is going to enable is that at the end of the review process, when a drug's approved, we're going to be able to make information about how and why we made our decision available much more quickly, within probably 48 hours. Then, it's going to be more of a problem-
Tanisha Carino:               Just for context for the audience, what is it now? Is 48 hours exponentially faster?
Scott Gottlieb:                 Yes. It's much faster. It could take weeks until you have that level of detail. There's some initial information for public but in terms of the level of detail we're going to make available, it's going to be much more quickly.
Scott Gottlieb:                 The review memo, so right now, the review process is you have a lot of people who are consultants to the review process, each developing their own review memos. It's a lot of cut and paste. If you look at different review memos as part of the standard review package, it's a lot of cut and paste. What we're going to do is have the different disciplines that are part of the review teams collaborating around one digital review template, much like you probably all do in your corporate jobs. We don't do that right now at FDA. We're going to have one review memo. The review memo's going to be more of a problem based review memo. What were the questions what were the critical questions we needed to answer as part of making a decision and how did we resolve those questions?
Scott Gottlieb:                 It's going to be a much more accessible document I think to providers to patients to people on the outside who want to review our work.
Tanisha Carino:               This will all be, at least part of it in the public domain [crosstalk 00:13:53]?
Scott Gottlieb:                 This is all going to be in the public domain and the place where we're implementing this approach first is around the safety portion of the review.
Tanisha Carino:               Mm-hmm (affirmative). In your past budgets, you've put in for a knowledge management system. Is this related knowledge management? Tell us a little bit about what that request really is about and how that plays into the notion of predictability or transparency.
Scott Gottlieb:                 Right. Right now, if someone came to me and asked me how many times has FDA approved an oncology drug based on a measure of effectiveness against this specific biomarker, the way I would answer that question is I'd try to assemble everyone in a room to ask them. I'd usually start with Bob Temple because he's been there the longest.
Scott Gottlieb:                 We don't have the ability to query our own decisions. We don't have the ability to look across our own decisions to try to extract from them where we made decisions based on common elements. All of our historical precedents is embedded in people's minds. Literally, when we're developing new guidance documents, where we're trying to consolidate collected knowledge over time, those are developed by bringing together big teams of people and trying to query them.
Scott Gottlieb:                 What we wanted to do is develop a knowledge management system that allows us to incorporate our own precedent and query it in a real-time fashion. This is something that a lot of knowledge enterprises have. We don't have it. We don't have it anywhere in the agency right now. We have a Google search bar. We want to develop this kind of a system. The budget request that we had in 2019 has a specific request for this. The requests were made in 2020 as well.
Tanisha Carino:               How urgent of an issue is this? Are you like most federal agencies that's looking at a tsunami of retirement. Now, you don't have those people in the room?
Scott Gottlieb:                 I'm worried about retirements because, like a lot of federal agencies, we have a cohort of people who came aboard at a certain time. A lot of our leadership is reaching retirement age but I'm not worried about retirements from this perspective. I think this is something that a lot of government agencies don't have but most government agencies are pass-through agencies, where we pass through very few dollars. Our money's spent at FDA developing intellectual capital and evaluating information.
Scott Gottlieb:                 We're an information enterprise. The fact that we don't have the ability to catalog and easily query our own decisions is a challenge especially as those decisions become more complex and we want to bring regulatory consistency across how we make decisions and ultimately if we built this tool, this would be a publicly accessible tool. There's companies on the outside, database companies that make a living developing databases that you can query around specific aspects of regulatory decision making. No one's built anything that would be a comprehensive tool on the order of what we want to develop.
Tanisha Carino:               For product sponsors as well as just patients, I would think that that's something that predictability is something we count on.
Scott Gottlieb:                 Yeah. The other thing is, on an ad-hoc basis, we, for example, will on certain safety reviews, consolidate safety datasets across multiple product reviews. This is a very touchy issue because companies claim that their data from their packages is commercial confidential information. If we're consolidating that into a data set to make regulatory decision making across a class, we need to be very mindful how we do that in a way that doesn't abrogate their intellectual property in those respects but we do do it. People know we've done it. We've been public about it but when we do it. those data sets aren't available publicly.
Scott Gottlieb:                 Where we are making decisions by putting queries against a consolidated data set that people on the outside can't query to see if we've made the right decision or to adjudicate their own decisions. That's not an enviable position. If we're going to be developing that kind of intellectual capital and property and make decisions based on it, we need to make that available.
Scott Gottlieb:                 This is another thing that, if you had a knowledge management system and help facilitate the consolidation of this information and make it publicly available, it's another opportunity that we'd be able to provide.
Tanisha Carino:               That's great. For those of you that don't often listen to the FDA Commissioner, one of the most important points to make is that Scott and his agency actually regulate, is it 20 cents on every dollar spent so 20% of the economy is regulated in some [formal way 00:18:33]?
Scott Gottlieb:                 It's a growing 20%.
Tanisha Carino:               Mm-hmm (affirmative). Tell us. There are all sorts of new areas of science. You just talked about areas that you're in selling gene-based therapies we've talked about. When people say you're moving towards a risk-based approach for a regulation, what does that mean and how are you thinking about how the regulatory frameworks keep up with the speed of the science?
Scott Gottlieb:                 I think we've always talked about trying to have a risk-based approach to regulation. I think when we think about that, that means trying to apply our regulatory tools in the areas of highest risk. The most palpable place to get a sense of what it means to be a risk-based system is in the post-market setting when you're doing foreign inspections or inspections of manufacturing facilities.
Scott Gottlieb:                 We inspect on the basis of risk and we have models to identify facilities that we think represent more risk based on where they're located, past history, the kinds of products they're manufacturing, what we found on past inspections, what we know about the proprietors of those facilities. We will go into certain facilities more often than other facilities. Some facilities we won't inspect on a regular schedules. Others we will. That's a risk-based model.
Scott Gottlieb:                 If you look at the Department of Agriculture, and this isn't to cast aspersions on my colleagues at the USDA, but the way they inspect meat and poultry is they have an inspector in every plant. We don't put an inspector physically in every plant. We go into facilities based on risk models. When we inspect those facilities, we're looking at things based on knowledge about where the points are that could create risk to consumers. That's the essence of what it means to be a risk-based model.
Scott Gottlieb:                 I think, as we advance those approaches, the opportunities come from having more information to better target how we do what we do.
Tanisha Carino:               Tell us how that philosophy has permeated even in things like your digital health strategy or things like cell and gene-based therapies.
Scott Gottlieb:                 Yeah. With respect to digital health, I think one of the things that marks this time in science and the regulatory challenges we face is that we are facing the advent of entirely new areas of technology and innovation where the old regulatory models don't apply real well when it comes to gene therapy, when it comes to regenerative medicine, when it comes to digital health tools, laboratory tests. If we tried to retrofit the existing regulatory paradigm onto these new areas, we would stifle innovation. In some cases it's going to require us to seek new legislative authority. You saw us provide about 70 pages of technical assistance to Congress around what we think would be an optimal approach to the regulation of laboratory-developed tests and diagnostics. In digital health, I think that that's a very palpable example where he had to come up with a new regulatory paradigm for how we would approach that.
Scott Gottlieb:                 Digital health tools are highly iterative. You're trying to make constant changes to them, to iterate them to improve those tools. You think about an app on your phone or on your smartwatch. If we required companies to come in and file five 10K supplement every time they wanted to push out an update on a medical app, it just wouldn't happen. We've taken what we're calling a firm-based approach the regulation of lower risk digital health tools, where tools that meet the definition of being a medical device and are subject to FDA's regulation, rather than regulating each specific product, what we're going to do is validate the underlying architecture of the software and allow firms that meet certain requirements in terms of their own SOPs to come to market with new products and new iterations of their existing products without requiring pre-market clearance each time. They'll be subject to post-market evaluation.
Scott Gottlieb:                 But when you look at the level of validation that some of these companies are engaging and I don't want to be company-specific but I've seen what some of these companies do. They're undertaking an enormous amount of validation. If we can go and inspect them, see their SOPs, understand the level of validation that they're requiring before they put their products in the market and it's an otherwise low-risk product, we can allow them to go to market and just be subject to post-market exercise of regulation.
Scott Gottlieb:                 If you look at the Consumer Product Safety Commission, this is how they regulate. They put out standards. People manufacture products subject to the standards. When a problem's identified in the marketplace, they issue a recall. They could take other remedies.
Scott Gottlieb:                 There's very few things in commerce that are subject to pre-market approval where you need pre-market approval before you can go to market with the product. Medical products are one, pesticides, aircraft engines. It's not a long list of things where you need the government's permission to be able to go to market. Putting the app on my watch on that list, we need to decide whether that meets the level of risk where we need to acquire that level of oversight. The answer has been more recently no. We don't we don't need to apply that level of oversight even if it's meeting the definition of being a medical device. We could think differently about how we apply regulation.
Tanisha Carino:               Right. I want to switch to the fact that FDA is now on track to approve more products than you did last year, which was a record year, where you'd approve 46 products. About 40% of products, it's almost holding steady year-over-year, have been really for orphan designations, orphan drugs.
Tanisha Carino:               Yesterday we had Harvard economist Amitabh Chandra come to us and talk to us a little bit about his research. One of the data points he gave us was that the public health needs and where R&D pipelines are are not a match.
Tanisha Carino:               Just as an example of that, in mental health, the burden of disease is 15%, whereas the R&D pipeline is less than 2%. Knowing that FDA isn't the only actor and what's needed for either pull or push incentives, what do you think is the role of the FDA to re-energize the innovation that needs to happen in these large big public health disease classes?
Scott Gottlieb:                 On the issue of the statistics, last year was a record year for the number of novel medical devices we approved, novel drugs and generic drugs will probably break all three records this year. We just approved the first new antiviral drug for flu in 20 years. I think that now we might have now surpassed the record number of approvals of all time. It's only October. We'll see how we do between now and at the end the year.
Scott Gottlieb:                 I have multiple concerns in this realm. I have a concern that it's become too hard to innovate in areas where there's so-called good enough generics and some of the primary care areas. I think that there's been such a focus on significant unmet medical needs that we've lost sight of the palpable public health improvement that comes from something that provides increments of additional efficacy for something that's an otherwise ordinary condition.
Scott Gottlieb:                 If you could shave 10 hours off of the common cold, when you aggregate 10 hours of relief from the common cold over a population of tens of millions of people who are going to get a cold each year, the overall public health impact of that could be enormous and productivity improvements and things like that.
Scott Gottlieb:                 I think that we've we've lost sight of the public health gains that come with small increments of improvement in therapeutics that treat otherwise common conditions that aren't necessarily life-threatening but where the symptoms do impact people's lives. It's become hard to run clinical trials in those settings because the expectation of safety has grown over time. I think there is nothing wrong with that.
Scott Gottlieb:                 I always say that if you gave me the choice between driving a 2002 Honda Accord with 200,000 miles on it or a 1980 Corvette that's been on cinder blocks and never driven, has zero miles on it, I would take the 2002 Honda Accord because it's a safer car. It has airbags. It has anti-lock brakes. It has dual suspension. I can go on. If I get into things I don't understand but the expectation for safety has grown over time. The regulatory process has built in more oversight to meet those expectations. I think that that's appropriate. As technology gets better, we should be using it to improve what our expectations are about both the benefits and the risks of the products we take but that can't come at the expense of creating inordinate hurtles.
Scott Gottlieb:                 I think in some therapeutic areas, the hurdles are very high right now. The clinical trial requirements are very big. That's where you have to look at I think the kinds of tools that can make it easier to collect information in a fashion that is just as rigorous but perhaps less onerous, things like collecting information through real world performance of people, every day measuring real world performance of, to measure lung function, to measure physical performance, things like that.
Scott Gottlieb:                 I'm also worried about, on the reimbursement side at the other end of that barbell, when you're looking at the drugs targeted to rare diseases, I think we have a highly nimble, highly innovative paradigm for drug development and medical product development and an ossified paradigm for reimbursement. There are places where I'm extremely worried that if we don't adapt the approach to reimbursement soon, we may foreclose therapeutic opportunities.
Scott Gottlieb:                 I was around and you were around too when we didn't get the reimbursement for the radiopharmaceuticals right. There were some good drugs at the time that worked and they're not used anymore. We not fully destroyed but we really shrunk the opportunity for radiopharmaceutical development in this country by underpaying the hospitals and forcing them to lose money on the application of radiopharmaceuticals.
Scott Gottlieb:                 We're doing the same thing with [Carty 00:28:17] right now. I think we have a window of opportunity to think about how we're going to reimburse that appropriately. There's things FDA can potentially do in terms of how we label the products but we're going to need to think about what an appropriate reimbursement approaches to some of these novel therapeutic areas if we want to see these sustained.
Tanisha Carino:               Many people observe that you're one of the few commissioners that has been very vocal about payment issues, drug pricing. Do you feel like that is something that is critical now that you're advancing science, you're on the forefront of knowing what's in pipelines, but the payer community by and large doesn't have the same point of view or the capacity to really be that in deep on every scientific advancement.
Tanisha Carino:               What is the FDA's role in bridging between the payers?
Scott Gottlieb:                 I think I've stuck to my knitting. I think that when you look at the issues of drug pricing. There's product competition. There's price competition. When it comes to price competition, you think about you know the reimbursement constructs that are exercised by Medicare or how PBMs and health plans are organized. When you think about product competition, you think about making sure there's timely generic entry when exclusivities and patents have lapsed on branded drugs and making sure their second and third to market innovation in some of these novel indications.
Scott Gottlieb:                 We've been focused on trying to bring more product competition to the market with the hope that if there's more products available within categories, the payment system is either going to use that to create price competition or hopefully adapt to figure out how to use that to create price competition in places where there's a lack of price competition based on product variety like in Medicare Part B, for example.
Scott Gottlieb:                 That's where we've been focused. I think one of the places you're going to see us focusing more going forward aside from high-value generic opportunities is second and third to market innovation in some of these highly novel classes. We now have data that we will put out soon that shows that the time in which it takes to get second to market innovation as some of these unmet medical needs, these novel orphan categories and third to market innovation is taking much, much longer. In many cases, we're seeing product categories remain monopolies in perpetuity.
Scott Gottlieb:                 I think that there is a sense in the product development community. I've been on the other side of this, on the venture side although I'm a little dated right now, having been out of it for a few years, but I think there is a sense that if you're going to be if you think you'll be third to market, you pull out. We're seeing less competition in these categories.
Scott Gottlieb:                 To the extent that innovators are having monopolies for longer periods of time or in perpetuity, that's not just creating an environment where prices stay higher longer but it's also foreclosing the opportunity for therapeutic variety within these categories. We know that therapeutic variety is important from a clinical standpoint. Patients sometimes have a differential response to drugs, even if they're largely similar molecules.
Tanisha Carino:               Right. I know we're almost out of time. I'm going to give you last question. Everybody who's been on the stage has been able to talk about what the future of health is. For you, what do you think is the most exciting thing that we have to look forward to in terms of health?
Scott Gottlieb:                 I'll tell you, the thing that I'm focused on right now the most is what we're doing on tobacco, quite frankly. I think that we have and I know it's not necessarily the answer that ties to some of these technology issues but I think that we have an opportunity when I came into FDA, we saw that smoking rates were declining in this country. I think if we get our policies right over a short period of time, we have the chance to dramatically accelerate the decline in smoking in this country. That's going to have a more palpable public health impact than perhaps any single product introduction that I can contemplate in any reasonable period of time.
Scott Gottlieb:                 It's going to require us, thank you, to more rapidly migrate adults off of combustible tobacco products onto products that have reduced risk. We have to embrace the concept of modified risk nicotine delivery products including e-cigarettes. It's also going to require us to make sure that those e-cigarettes, if we make them available for currently addicted adult smokers, don't become tools for addicting a generation of youth on nicotine. That's what we're seeing right now.
Tanisha Carino:               Got a lot of fans in the room for that.
Scott Gottlieb:                 We will have much more to say about this in about four weeks.
Tanisha Carino:               Thank you for spending your day with us, Scott, I know it's been a busy day. Please join me in thanking the commissioner.
Scott Gottlieb:                 Thanks a lot. Thank you.
Conrad Kiechel:               Thank you, everybody and good afternoon. Thank you also for your insights and your time to Scott Gottlieb.
Conrad Kiechel:               In just a few minutes, we're going to be serving the rest of lunch and our main course, which is our chairman Mike Milken's presentation 25 Years: The Quest to Create More Healthy, Lengthy, and Meaningful Lives, but before the meal we wanted to take a moment to give thanks.
Conrad Kiechel:               First, thank you for joining us here at the Milken Institute Future of Health Summit. We've had great feedback in the last couple of days from many of you. We hope that the insights and the inspiration connections you've garnered here will help inform and inspire the work that you continue to do.
Conrad Kiechel:               The Future of Health Summit would not have been possible without the work and commitment of many, many people, some of whom are in this room. In addition to our partners at the Baker Group, Vision Matrix and our host here at the Ritz-Carlton, please join me in thanking our dedicated wonderful staff at the Milken Institute. Thank you, staff. Thank you. Thank you for Mr. Milken.
Conrad Kiechel:               There are also some very important people, some of whom are in this room, some of whom are not. Those are our sponsors who make the work of the Milken Institute possible not just for the summit but year-round. We're having a special reel to thank them as well. Thank you and enjoy your lunch. Bye.