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ICER will be attending ISPOR 2024 next week, and we’re excited to dig into HEOR issues like the CMS drug negotiation process, the employer perspective in value assessment frameworks, outcomes-based agreements for cell and gene therapies, and more.
Click here for the full lineup of ICER speakers. We hope to see you there!
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Let's see what happened this week... | |
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ICER’s Chief Medical Officer David Rind, MD authored a JAMA Viewpoint piece on the FDA’s accelerated approval pathway and gene therapies for DMD.
Dr. Rind stated:
“Although the FDA has made it clear that some therapies granted accelerated approval are expected to ultimately be shown not to have net benefits, FDA has stretched the meaning of a ‘surrogate outcome’ when it comes to SRP-9001. Flexibility around accelerated approval can benefit patients. However, the integrity of accelerated approval will be called into question if FDA grants subsequent full approval to therapies that have failed confirmatory trials.”
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ICER recently released a Draft Evidence Report assessing the comparative clinical effectiveness and value of 3,4-Methylenedioxymethamphetamine-assisted psychotherapy (MDMA-AP; Lykos Therapeutics) for the treatment of post-traumatic stress disorder (PTSD). That draft was open to public comment until mid-April, and we’ll be releasing a revised Evidence Report on May 14th.
The Washington Post highlighted ICER's report:
“ICER researchers also said trials may have been skewed by therapist participants who are boosters of MDMA as a mental health treatment, as well as large numbers of subjects who had previous experience taking MDMA. The report cited concerns that some participants that received MDMA felt ‘pressured to report good outcomes and suppress bad outcomes,’ and flagged a documented case of therapist misconduct that raised safety risks.”
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Pink Sheet's Sue Sutter and ICER's Vice President of Research Foluso Agboola, MBBS, MPH discussed ICER's new clinical trial diversity tool, which is part of ICER's 2023 update to our Value Assessment Framework.
"As an HTA organization, ICER often finds itself trying to answer questions not only about clinical and cost effectiveness, but also about ethical and contextual issues related to how new drugs are developed, Agboola said in explaining why ICER became interested in assessing clinical trial diversity...ICER developed the Clinical trial Diversity Rating (CDR) framework to provide an objective and transparent approach to evaluating trial diversity. Agboola was lead author of a 22 February article in the Journal of Clinical Epidemiology that described the framework’s development and testing."
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We will assess the comparative clinical effectiveness and value of tabelecleucel (“tab-cel”, Pierre Fabre) for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD).
Consistent with ICER’s process for announcing new assessments, we have spent the past five weeks engaging with targeted stakeholders, including relevant patient groups and consumer advocates, the manufacturers of the treatment being assessed, and clinical specialists. Based on this preliminary cross-stakeholder engagement, ICER has posted a Draft Scoping Document outlining how we plan to conduct this assessment, which will be open to public comment until May 22nd.
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Researchers from Tufts CEVR, Harvard’s PORTAL, and the CHOICE Institute at the University of Washington, Seattle developed a framework to guide CMS as they design the processes for drug pricing negotiations. The framework covers comparator selection, value assessment, other considerations in addition to value, and the importance of a public and transparent process. They stated:
“Our approach is borne of a conviction that negotiation for the sole purpose achieving the lowest possible price will send the wrong signals to industry and the wider public about the benefits of pharmaceutical innovation, and that the appropriate tool for negotiation is an ascertainment of the value these drugs provide."
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Pfizer plans to allow payers to use a “warranty” approach to pay for its new gene therapy. The company received FDA approval last Friday for Beqvez (fidanacogene elaparvovec-dzkt) for the treatment of adult patients with moderate to severe hemophilia B.
In a statement Friday, Pfizer announced it will be “launching an innovative warranty program based on durability of patient response to treatment.” The wholesale acquisition cost for the treatment will be $3.5 million.
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On Monday, a New Jersey federal judge dismissed cases by Johnson & Johnson and Bristol Myers Squibb against the Inflation Reduction Act (IRA). In a summary judgment, Judge Zahid Quraishi ruled the IRA provisions don’t constitute a physical taking of property or a violation of speech, the two constitutional claims at the core of the drugmakers’ arguments. The opinion could impact similar cases in other courts across the country.
Separately, PhRMA and AstraZeneca are each appealing their cases against CMS drug pricing negotiations.
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Skinny labeling refers to a move by a company that seeks regulatory approval to market a generic or biosimilar medicine for a specific use, but not for other patented uses for which the brand-name drug is prescribed. By doing so, the company seeks to avoid lawsuits in which the brand-name manufacturer could claim patent infringement. In short, this is a so-called carve-out.
Researchers examined 15 brand-name drugs where a generic with a skinny label approval occurred from 2015 to 2019. From there, they culled Medicare Part D spending data 2015 to 2021 to compare actual spending on each brand-name drug and the “skinny label” generics with projected spending had those not been introduced. Among the 15 brand-name drugs, the researchers found the “skinny label” generics generated competition that was 2.5 years sooner, on average, than if these generic alternatives had not become available. And the actual use of these generics was, on average, 13.5% higher than what might have otherwise occurred without “skinny label” alternatives, according to their projections.
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California will soon begin selling its own generic version of Narcan — the drug that can save someone’s life during an opioid overdose — under a deal announced Monday by Democratic Gov. Gavin Newsom as part of his effort to offer less expensive, state-branded options of medication.
The New Jersey-based Amneal Pharmaceuticals will sell naloxone to California for $24 per pack, or about 40% cheaper than the market rate. California plans to give away many of the packs for free to first responders, universities and community organizations through the state’s Naloxone Distribution Project.
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Want to work for a fast-paced, mission-driven organization? Look no further! ICER is looking to expand our team, which includes a diverse set of clinicians, researchers, and policy experts. | |
Health Technology Assessment
Program and Operations
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5/9: MDS -- Draft Evidence Report
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5/14: PTSD -- Revised Evidence Report
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5/30: COPD -- Revised Evidence Report
Learn more about ICER’s ongoing and recently completed reports:
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