Conveying Value of Accelerated Approval Drugs May Be Challenge for Pharma Firms

That is the title of an article in Spotlight on Market Access published by MMIT Radar. The article examines how FDA decisions to expedite getting treatments to market will be received by payers. The Healthcare Economist is quoted extensively in the article:

Payers have two key concerns, maintains Jason Shafrin, Ph.D., senior director of policy and economics at Precision Xtract: “(1) Will the benefit from the clinical trial translate into health benefits for patients in the real world, and (2) how will new treatments affect budgets?”

On the first question, Shafrin points to an article published in 2017 in the International Society for Pharmacoeconomics and Outcomes Research journal Value in Health on which he was lead author that examined how patients and physicians assessed durable survival gains. That research, he tells AIS Health, “found that the surrogate endpoints used in most accelerated approval trials were good but imperfect proxies of overall survival. Specifically, effectiveness observed in the real world was 16% lower than what was observed in clinical trials when trials used surrogate endpoints such as progression-free survival or time to progression. This ‘adjustment factor’ for surrogate endpoints varied depending on tumor type, the size of the clinical trial, treatment line of therapy and other factors.”

On the issue of drug prices, Shafrin points to research showing that the price of a new oncology drug in 2015 was about three times the price of a new cancer drug in 1997. Payers, however, should put data about the rising cost of cancer drugs into context,” he says, noting that same research “shows that most of these new cancer treatments are targeted therapies that are indicated for smaller patient populations. In fact, the indicated population for a typical cancer drug in 2015 was 90% smaller than the indicated population of a cancer drug in 1997. Further, total payer spending on a given new cancer drug has actually fallen over time.”

…“Payers are increasingly looking for new cancer treatments that are not only safe and effective but also provide good value for money,” he says. “The advent of new value assessment groups in the United States such as the Innovation and Value Initiative and Institute for Clinical and Economic Review are gaining prominence. These groups use advanced economic models to monetize the net benefits of treatments less their cost.

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