April 2019: Roger Klein in THE HILL on CMS Financial Outlook

On April 29, 2019, Roger Klein MD published an article in THE HILL on Medicare's grim overall cost outlook in future years and decades.

https://thehill.com/opinion/healthcare/441242-instead-of-revamping-it-congress-should-address-medicare-as-is

Overnight the op ed generated 39 comments and 152 shares.

new tech 201904

Inside CMS - 04/25/2019

CMS Proposes Add-On Pay Path For Breakthrough Devices

April 23, 2019

CMS proposes raising the add-on pay for new technology and providing a path for breakthrough devices approved by the FDA to access add-on payments as part of the proposed fiscal 2020 hospital inpatient pay rule released Tuesday (April 23) -- a move the medical device lobby praised for including several of the industry’s suggestions.

“Transformative technologies are coming to the private market, but Medicare’s antiquated payment systems have not contemplated these technologies,” CMS Administrator Seema Verma says in a press release on the proposed rule. “I am particularly concerned about cases that have been reported to the agency in which Medicare’s inadequate payment has led hospitals to curtail access to needed therapies. We must continually update our policies in response to the rapid pace of advancement in medical science.”

The agency proposes to increase the new technology add-on payment, as CMS believes setting the maximum add-on pay percentage at 50 percent for certain technologies may not be adequate. The agency proposes to increase the add on-pay, starting in fiscal 2020, to 65 percent.

“It is challenging to determine empirically a precise payment percentage between the current 50 percent and 100 percent payment that would be the most appropriate. We believe that 65 percent is an incremental increase that would reasonably balance the need to maintain the incentives inherent to the prospective payment system while also encouraging the development and use of new technologies,” the proposed rule says.

To qualify for an add-on payment for new services and technologies under the inpatient pay system, devices and services must be new; “be costly such that the DRG rate otherwise applicable to discharges involving the medical service or technology is determined to be inadequate;” and demonstrate a substantial clinical improvement over existing services or technologies, according to CMS.

In the proposed rule, the agency proposes an alternative new technology add-on payment pathway for medical devices that receive FDA marketing authorization and are part of an FDA expedited program for medical devices -- currently the Breakthrough Devices Program. If a device was approved as part of that program, the agency would consider the product new and not substantially similar to an existing technology, meaning that it would only need to meet the cost criterion to receive the add-on pay, CMS says in a fact sheet. The change would begin with applications for fiscal 2021 add-on payments.

The agency says in its press release that real-world data on outcomes for breakthrough devices are often limited when they are approved, and “it can be challenging for innovators to meet the requirement for evidence demonstrating ‘substantial clinical improvement’ in order to qualify for new technology add-on payments.”

“Therefore, CMS is proposing to waive for two years the requirement for evidence that these devices represent a ‘substantial clinical improvement,’” CMS’ press release says. “Waiving this requirement would provide additional Medicare payment for the technologies for a period of time while real-world evidence is emerging, so Medicare beneficiaries do not have to wait for access to the latest innovations.”

The proposed rule notes that CMS is not proposing a similar alternative path for drugs at this time.

The medical device lobby praised CMS for adopting several of the industry’s proposals.

“We are pleased that this rule reflects several of AdvaMed’s priorities, including increasing funding for new technology add-on payments (NTAP), and making it easier for breakthrough technologies to access the Medicare program. This will help ensure that hospitals and patients have access to breakthrough innovations and care, and further incentivize transformational technologies,” Advanced Medical Technology Association President and CEO Scott Whitaker said in a statement. “We look forward to reviewing the proposed rule in more detail, and working closely with CMS on additional ways to facilitate greater efficiencies in the coverage and payment process.”

CMS also says stakeholders have indicated more guidance on what constitutes a substantial clinical improvement would be helpful. The agency says it is considering potential changes to the substantial clinical improvement criterion under the new technology add-on payment policy and the outpatient pay system transitional pass-through pay policy for devices. The agency wants feedback on the kind of detail and guidance stakeholders would find useful, and says it plans to use that feedback to inform future rulemaking.

The agency also seeks comments on changes or clarifications to the substantial clinical improvement criterion CMS could consider including in the final rule “to provide greater clarity and predictability.”

The proposed rule includes 17 new applications for the new technology add-on payment in fiscal 2020 and the agency proposes to continue payments for 10 of the technologies currently receiving the add-on pay. Three other technologies currently getting add-on pay will no longer be within their three-year newness period come fiscal 2020. -- Michelle M. Stein (mstein@iwpnews.com)

2019.04.24 Pierian Webinar on Illumina TruSight Oncology 500 Test (RUO/LDT)

https://info.pieriandx.com/comprehensive-genomic-profiling-an-optimized-approach-for-rapid-implementation-and-reporting?hsCtaTracking=06677217-a32d-4000-a350-fb640f8305ed%7C899a2883-cbb6-4e86-b808-99c641b85d6c

Enabling Comprehensive Genomic Profiling with TruSight™ Oncology 500

Wednesday, April 24, 11:00 AM EDT (10:00 AM CDT, 9:00 AM MDT, 8:00 AM PDT)

Enabling Comprehensive Genomic Profiling with TruSight™ Oncology 500

guest Rakesh Nagarajan, MD, PhD

host: Josh Forsythe

In this educational webinar, 

we explore how your laboratory can rapidly validate, implement, 

and report using the pan cancer profiling assay.

Enabling Comprehensive Genomic Profiling with TruSight Oncology™ 500

Clinically-optimized for rapid implementation and 
evidence-based reporting

Thanks to larger, more comprehensive NGS assays, there are more opportunities than ever to improve care and outcomes for cancer patients. The newest assays provide comprehensive coverage of variants and immunotherapy biomarkers (including tumor mutational burden and microsatellite instability) and enable the tracking of mutational signatures over time. Now that these assays are commercially available for the first time, laboratories have the option of running them in-house, which can positively impact quality and crucial turnaround time.

In this educational webinar, we explore how your laboratory can rapidly validate, implement, and report using the pan cancer profiling assay.   

By attending this webinar, you will:

• Understand the utility of comprehensive panels and drivers behind larger DNA- and RNA-based assays
  
• Learn how additional knowledge management and other curation approaches can increase clinical management yield, (i.e., the number of cases where one or more clinically relevant variants is found)
  
• Learn strategies for optimizing reporting and sign-out to reduce turnaround times and maximize clinical utility
  
• Learn how to reduce validation costs while maximizing specificity and sensitivity of all variant types
  
• Acquire strategies for assessing your assay’s overall performance

Extra PGX Pubs (2019 04 20)

Zip at
https://drive.google.com/file/d/11pseAkR1B5lVTsX1w9_2Q8UteMPvBz0x/view?usp=sharing

Short link to this webpage here
https://tinyurl.com/ExtraPGxPubs20190420

File names


2014 CIA 9_2079 Alhawassi Review of ADR in Elderly in Acute Settings 8p.pdf
2015 BJCP 80_796 Davies ADR in the Elderly.pdf
2016 Lancet Psychiat 3_585 Bousman Comm PGX tests in Psychiatry CDS 6p.pdf
2016 PLOS One 11_30165757 Nair Prediction ReHosp ADR Elderly (Preventable).PDF
2017 BMC Psych 14_250 Perez Psych RCT PGX.pdf
2018 AJGP 26_125 Abbott PGX and Late Life Depression 8p.pdf
2018 BMC Ger 18_135 Lenssen Prevent ADR Readmissions in Elderly (Germany).pdf
2019 CPT Epub Bousman Labyrinth of Commercial PGX Test Options 4p.pdf
2019 CPT Epub Derendorf DDI Progress and Future 3p.pdf
2019 CPT Epub Huddart Are RCT Necessary for PGX in Clinic 3p.pdf
2019 CPT Epub Tornio Clinical Studies for DDI Pitfalls Methods 17p.pdf
2019 CPT Epub Venkatakrishnan Dance_ Transforming DDI 7p.pdf
2019 Curr Opin Psychiat 32_7 Bousman Five Gene REcommend Panel Psychiat 9p.pdf
2019 Genomeweb 0401 Geneticure PGX HT FDA RCT.pdf
2019 Genticure Clin Trials Gov 02988245 HT PGX.pdf
2019 Lancet Psych BOUSMAN Op Ed CYP2d6 3p.pdf
2019 Lancet Psych JUKIC Clin Study Respir CYPd26.pdf
2019 Pharmacogenomics 20_37 Bousman Meta Anal Favorable for CYP in Depression 11p.pdf
2019 Pharmacopsychiatry Epub Fan Bousman Commerc Psych Tests Do PRN w SUPP 5p.pdf
2019 Pharmacopsychiatry Epub Fan Bousman Commerc Psych Tests ORIG LOCKED 5p.pdf
2019 Pharmacopsychiatry Epub Fan Bousman Commerc Psych Tests SUPP ONLY 10p.pdf
2019 Translational Software_s White Paper zPGX 17p.pdf

2019 UJME 21_1246 Kelley PGX in HT Econ Value 7p.pdf

FT, Wellcome Fnd Author on Antibiotic Crisis. Similar in THE HILL by IDSA, Pew.

Depending on whether I google this on my phone, on a laptop, via different keywords, this article comes up variably behind - or not behind - a firewall at Financial Times.  This is a clipping from a non firewall access path.

See also a UN announcement covered in the NYT, and an article in Business Week.  Here, here.

See articles in The Economist here, here, podcast series here, March 2019 London conference here.  (With the conference see also an "Economist Article Kit," here.)
See article in MedCity News here.


https://www.ft.com/content/4da1c6e4-603d-11e9-9300-0becfc937c37

See also:

Axios, October 2018: The Antibiotics Market is Falling Apart
     https://www.axios.com/antibiotic-drug-resistance-market-c712e575-0d34-4c9e-b81c-ce1844f59242.html


Pew Foundation, April 2019:  The Antibiotic Market is Broken - And Won't Fix Itself
Jointly with IDSA.  Appeared at this link, and also in THE HILL.
     https://www.pewtrusts.org/en/about/news-room/opinion/2019/04/10/the-antibiotic-market-is-broken-and-wont-fix-itself

Pew to Congress, re ABx, February 2019
     https://www.pewtrusts.org/en/research-and-analysis/speeches-and-testimony/2019/02/05/groups-urge-congress-to-act-on-antibiotic-resistance

Pew "Drug Spending Initiative" (not specific to Abx), 2017/2018
https://www.pewtrusts.org/en/projects/archived-projects/drug-spending-research-initiative

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We ignore the disaster in the antibiotics market at our peril

Financial Times   April 21, 2019

There is no viable path for new drugs, however valuable they are to society 

 To go beyond early-stage research and initial small-scale trials, biotechs need private capital to step in 

  Jeremy Farrar  - Wellcome Trust


 Achaogen is not a company most people have heard of. It is not a household name, barely has an international presence and most of us have never used one of its products. The few who have probably don’t know the company either, even if its product saved their life. And yet its recently announced bankruptcy is one of the most significant — and worrying — corporate failures of this decade.


 In the global struggle against superbugs, Achaogen is a biotech at the front line. Its failure is the latest symptom of an ailing antibiotics market. Decades of disinvestment have left perilously few companies active in antibiotic development. Those remaining are often dependent on support from philanthropic or public funders — such as the Wellcome Trust, the medical research charity, or the US government.


 This support has catalysed an exciting crop of biotechs that are now driving antibiotic innovation. But to go beyond early-stage research and initial small-scale trials, they need private capital to step in. Without external investment, small biotechs cannot carry prospective drugs through the complex and expensive later-stage trials they must pass.


 Against the odds, Achaogen appeared to have succeeded. Its antibiotic, plazomicin, was approved by the US Food and Drug Administration in 2018 for treating complex urinary tract infections caused by drug-resistant bacteria. It is a vitally needed drug and just one of the many new antibiotics we need to replace drugs that are rapidly losing their effectiveness against superbugs.


 Achaogen was a leading example of what could be achieved by a smart start-up working in partnership with government and philanthropic funders. (Wellcome supported Achaogen with early research and development for the plazomicin programme, was later an investor in its IPO and still indirectly holds a small amount of stock). A handful of other companies followed, some making it all the way to market.


 Private investors backing such companies counted on revenues being buoyed either by growing need for their products or by governments responding to the calls to fix the market. Instead, a disaster is unfolding. With limited resources behind it, and facing a tough marketplace, plazomicin has struggled to gain market share even as an alternative to colistin, a cheap, 50-year-old antibiotic with severe side-effects.


 In the year before filing for bankruptcy protection last week, Achaogen’s stock lost more than 95 per cent of its value. It closed research and development programmes, laid off staff and even auctioned lab equipment online in desperate efforts to stay afloat. It is not alone. Ten of 12 antibiotics launched in the US in the past decade (not all of them breakthrough products) are achieving US sales of less than $100m a year. This barely covers the cost of keeping them on the market, let alone recouping investments. 


 The tragedy is not that investors have lost their money. Rather, it is the signal that there is no viable route to market for new antibiotics, however valuable they may be to society. Capital-starved smaller companies will fold. Innovation will die on the vine. Money already invested by governments and charities will be squandered.


 Despite agreement about what is wrong, our political leaders have chosen not to address the problem. This is unacceptable. Governments must send an immediate signal to companies and investors that the future is not as bleak as the present. The UK government has announced a limited new pricing model pilot, paying developers upfront for antibiotics used by the National Health Service, based on the value of the drugs to the service rather than quantity. But there will be a two-year delay before it is implemented and possibly no fresh funding from the Treasury.


 We need real change now. The amounts that need to be injected are high — about $1bn per drug. That’s a hard sell for politicians, but it reflects the value of antibiotics. The cost could be shared among countries: for the UK to contribute 5 per cent, for example, of an additional $1bn a year for antibiotic development would cost about 6p per person per month. This is a problem we can afford to solve.


 If leaders are afraid to deliver these types of market incentives, we need to offer a plan B — finding creative new models to stabilise the antibiotics market and stimulate private sector innovation without exposing public funders to all the risk. If we do not act, the biggest losers will not be the investors, staff and shareholders of companies such as Achaogen, but patients and the public.

 The writer is director of the Wellcome Trust


###
###
https://thehill.com/opinion/healthcare/438165-the-antibiotic-market-is-broken-and-wont-fix-itself

The antibiotic market is broken and won't fix itself

BY ALLAN COUKELL AND HELEN BOUCHER, OPINION CONTRIBUTORS — 04/10/19 07:30 AM EDT

PEW & IDSA

Over the past year we’ve watched two troubling trends escalate. First, patients increasingly face — and their doctors struggle to treat — infections that do not respond to existing antibiotics. Second, major pharmaceutical companies are backing away from developing new antibiotics. Last July, Novartis became the third major pharmaceutical company in 2018 alone to announce that it would end antibiotic research and development. Other companies that haven’t eliminated antibiotic R&D have significantly reduced it.

In short, just as the world needs new and novel antibiotics, the research needed to find them is shrinking in size and scope.

Antibiotics play a central role in modern medicine — one that goes beyond treating infections that were routinely fatal before the drugs came on the scene in the mid-20th century. The discovery of penicillin and the drugs that followed also made possible surgery, chemotherapy, dialysis, and other procedures that we now take for granted. They also help protect at-risk patients such as children, seniors, and people who have underlying conditions, such as cystic fibrosis, that weaken the immune system.

The Pew Charitable Trusts and the Infectious Diseases Society of America, together with more than 20 leaders in antibiotic research and development, have joined forces to push for swift action in the face of the growing challenge posed by the combination of increasing resistance to antibiotics and decreasing efforts to find urgently needed new antibiotics. In a letter recently sent to members of Congress, our organizations called on policymakers to commit to advancing a set of economic incentives to make antibiotic development viable again.

Providing economic incentives to pharmaceutical companies might seem like a hard sell at a time when high drug prices are a top concern of the public and policymakers. Our two organizations share this concern, and Pew has been working to address the problem. However, antibiotics are unlike other drugs. As companies reduce or eliminate their research efforts, the antibiotic market is less likely to produce new therapies to meet the threat of increasingly resistant infections, and physicians will have fewer tools to cure infections and protect patients as they undergo medical procedures.

At least nine authoritative commissions and reports have come to the same conclusion, recommending “pull incentives” to reward the development of urgently needed antibiotics by increasing the return on a drug once it comes to market. We believe that the U.S. approach to pull incentives must:

1. Address the greatest public health needs

The focus should be on stimulating the development of novel antibiotics to treat the most dangerous threats.

2. Align with efforts to improve stewardship and surveillance

Antibiotics must be used appropriately so as not to accelerate the emergence of resistance.

3. Provide predictability

Any incentive needs to be structured in a way that ensures it will be available when an antibiotic developer meets eligibility criteria.

4. Stabilize the market

Measures must be put in place to ensure that companies that have recently developed promising new antibiotics, often with the support of public funds, have an incentive to continue producing them.

According to one estimate, to fix the antibiotic market, a package of pull incentives needs to be worth an additional $1 billion in revenue for each qualifying antibiotic that a company develops, on top of sales earnings. The full amount would not need to come from the U.S., nor would it need to come exclusively from public sources. The U.S. contribution should be proportionate to the U.S. share of the global pharmaceutical market.

At a time when the cost of pharmaceuticals in the U.S. is already high, we do not suggest pull incentives lightly. But the antibiotic market is different from other drug markets in two important ways. When a new antibiotic is approved, public health requires that it be held in reserve as long as possible to slow the development of resistance.

This means that new antibiotics often face anemic initial sales. For example, Allergan’s Teflaro — one of the very few drugs effective against multidrug-resistant pathogens that have been identified by the Centers for Disease Control and Prevention as serious threats to public health — garnered average annual sales of $130 million from 2016 through 2018. Contrast that figure with the $1.4 billion in sales generated last year by the 20th best-selling cancer drug, and it isn’t surprising that Allergan announced plans last year to divest its anti-infectives business unit. 

The second way in which antibiotics differ from other medications is that as bacteria develop resistance, existing drugs lose their effectiveness, making it inevitable that new antibiotics will be needed. When antibiotic development stands still, the options for patients go backward.

CDC data show that tens of thousands of Americans die each year because of resistant infections. The situation could quickly become exponentially worse if new, resistant mutations emerge. Indeed, alarming new types of antibiotic resistance  have recently been identified — enabling pathogens to evade some of our most powerful antibiotics and spread quickly.

Private-sector investment in antibiotics has been falling for several decades, and there is no indication that the trend will slow. Public programs that support aspects of antibiotic development before they reach the market (often referred to as “push incentives”) — such as those of the Biomedical Advanced Research and Development Authority, the National Institutes of Health, and the Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) program — have been important steps forward, but not enough. We need to fundamentally change how the market for new antibiotics works.

We cannot afford to wait or hope any longer. Ensuring that the U.S. has effective antibiotics is not a luxury; it is a necessity for protecting medical advances, preventing the spread of drug-resistant pathogens, and saving lives. Congress must act now. 

Allan Coukell directs health programs for The Pew Charitable Trusts. Helen W. Boucher, M.D., is director of the Tufts Center for Integrated Management of Antimicrobial Resistance at Tufts Medical Center, professor of medicine at Tufts University School of Medicine and a member of the Infectious Diseases Society of America board of directors.

Peter Bach and Mark Trusheim: Upending Biosimilar Pricing

Two blogs on why biosimilar pricing doesn't work, and why it won't work, and why we should treat the first biosimilar as a strongly regulated monopoly when it's patent expires.  Health Affairs, Blogs, April 2019.

https://www.healthaffairs.org/do/10.1377/hblog20190405.396631/full/

https://www.healthaffairs.org/do/10.1377/hblog20190405.839549/full/

Herper and Silverman discuss the plan in STAT

https://www.statnews.com/2019/04/15/peter-bachs-latest-crazy-idea-give-up-on-biosimilars-regulate-drug-prices-instead/

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In 2017, Bach and Trusheim suggested it was cheapest for US Govt to buy Gilead.

https://www.forbes.com/sites/sciencebiz/2017/01/17/the-u-s-government-should-buy-gilead-for-156-billion-to-save-money-on-hepatitis-c

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For earlier work by Trusheim, see Berndt & Trusheim, 2017, article on precision medicine and game theory, here or here.  (Quinn has ms. PDF).   This is a chapter in a 2019 book, "Economic Dimensions of Personalized and Precision Medicine," Univ Chicago, here.   Etextbook, $123 here.  Release date 4/22/2019.