From the desk of David Whitrap
ICER proposed updates to our 2020 Value Assessment Framework this week – including new emphasis on real-world evidence, a revised approach to voting on contextual considerations, and a formal process for re-evaluating evidence report 12 months after our reports are published. These proposals are based on ICER’s experience in methods development, benchmarking with other HTA groups around the world, and ongoing public feedback, including suggestions from nearly 100 organizations and individuals who submitted letters during a recent Open Input period. These new proposals will be discussed during a September 4 webinar, and public comment is now open through October 18.
This week’s updates include:
- ICER in the News: More on our 2020 Value Assessment Framework and on value frameworks in general, our review of treatments for sickle cell disease, President Trump’s call for $0 Spravato, and the FDA’s rejection of a potential treatment for Duchenne muscular dystrophy we recently reviewed.
- Pharmaceutical News: The continued saga around Novartis’ Zolgensma, an approval and a price for AbbVie’s upadacitinib, a slowdown in drug-price increases, a case for generics, and also a case for pursuing price controls instead of biosimilars.
ICER in the News
The Pink Sheet takes a deep dive into the updates we’re proposing for our 2020 Value Assessment Framework, focusing specifically on our use of real-world evidence, as well as our willingness to incorporate into our reports a broader discussion of alternative assumptions suggested by manufacturers.
A paper in the Journal of Clinical Pathways examines various cost-effectiveness frameworks that have been applied to oncology treatments. The authors note that, “ICER has achieved the most notoriety over the last few years… ICER panels are diverse and include payer, manufacturer, and third-party stakeholders. The overall approach results in reports that are complex but also applicable to the changing health care landscape.”
The Evolution of Value Frameworks and What Is Next
(Journal of Clinical Pathways)
Sickle Cell Anemia News covered our ongoing review of treatments for sickle cell disease and emphasized ICER’s approach to gathering patient feedback to “help make sure patients can access, and afford, the most effective treatments.”
ICER Will Compare Lead Candidate Therapies
Crizanlizumab and Voxelotor for Efficacy, Value
(Sickle Cell Anemia News)
While speaking to an audience of veterans on Wednesday, President Trump urged Johnson & Johnson to “make a contribution” by giving Spravato away for free to the Department of Veterans Affairs. In its coverage, Endpoints News notes ICER’s conclusion that the list price for J&J’s reformulated ketamine nasal spray would require a 25-52% discount to reach common thresholds of cost-effectiveness.
Last week, ICER published a final evidence report for Duchenne muscular dystrophy therapies, focusing on the tensions around Sarepta Therapeutics’ Exondys 51, a million-dollar-per-year treatment that, still three years after FDA approval, lacks adequate evidence of efficacy. This week, the FDA rejected the application of Sarepta’s second DMD therapy, Vyondys 53. In a column speculating that the rejection of Vyondys 53 may be connected to Sarepta’s delay in conducting a comfirmatory trial for Exondys 51, STAT’s Adam Feuerstein concludes: “Vyondys 53… the delinquent effort to confirm Exondys 51’s treatment benefit… and the criticism of the price and value of its medicines by the watchdog group ICER are all weighing on Sarepta.”
As most readers of this newsletter will know, ICER found that Zolgensma, Novartis’ gene therapy for spinal muscular atrophy, could be cost-effective even at its eye-popping $2.1 million price tag. A heartbreaking story in Bloomberg Businessweek chronicles the different paths – from crowdfunding to immigration – that families are now taking to try to gain access to the treatment. Thankfully, as the article notes, several major insurers are now expanding their initial policies to now cover all patients who match the FDA’s label for Zolgensma.
When a $2.1 Million Drug Could Cure
Your Child’s Fatal Disease
While Zolgensma appears to be cost-effective, controversy continues to swirl around data manipulation in the pre-clinical animal studies associated with the treatment. This week, the Novartis CEO continued to defend the company, the scientist associated with the controversy denied any wrongdoing, and perhaps most interestingly, an FDA top official shared his overarching concern: “[While the manipulated data don’t cast doubt on Zolgensma’s benefits], we’re making sure that the whole ecosystem understands that when people are working on these things that are highly technically complex, that they have to work truthfully and accurately. Because that’s the foundation upon which the trust that patients put in these products is built.”
Novartis CEO Battles Fallout From Data Manipulation
(The Wall Street Journal)
So far in 2019, prescription drug prices have increased by a median of 5%, compared to 9-10% in recent years. But before we celebrate the success, note that one drug maker just replaced an old treatment and hiked the price by 1,300%. (Later this year, ICER will be publishing our list of the industry’s most significant price hikes that aren’t supported by new clinical evidence.)
Brand-name drug prices rising at
slower pace, lower amounts
Last Friday, AbbVie received FDA approval for its new treatment for rheumatoid arthritis, Rinvoq (upadacitinib) and immediately set an annual price of $59,000 — nearly matching the price for which AbbVie currently sells its existing RA blockbuster, Humira. ICER will be publishing our draft assessment of upadacitinib and other JAK inhibitors on September 18.
In a WSJ op ed, Peter Bach and Mark Trusheim reflect on the barriers that limit biosimilar competition in the US market, proposing that the government simply forget about biosimilars and instead impose price caps on the original biologics once their patent protection expires.
Time to Throw In the Towel on Biosimilars
(The Wall Street Journal)
A study from the Annals of Internal Medicine finds that Medicare could have saved $17.7 billion over the past decade by encouraging use of generic medications instead of brand name drugs.
Gilead’s Truvada may not actually have exclusivity in the US, which means that generics competing against Truvada could become available soon. Gilead has encountered significant criticism for Truvada’s $20,000 annual price.
Gilead did not seek US exclusivity on PrEP with Truvada
(The Financial Times)
Endo and Allergan are in the process of negotiating opioid-related settlements with counties in Ohio worth a total of $15 million. Purdue Pharma, Teva, and J&J are still scheduled to face trial in October.
Two Drugmakers Closing in on Opioid Settlements
(The Wall Street Journal)