Liberal withdrawal of accelerated approvals will have negative ramifications for oncology patients, says GlobalData

The Food and Drug Administration’s (FDA) accelerated approval program has recently come under fire due to high-profile withdrawals of immune checkpoint inhibitors (ICIs) from the oncology market. Conservative withdrawal of drugs marketed under accelerated approval has more upside for cancer patients with few available options compared to liberal withdrawal, says GlobalData, a leading data and analytics company.

The FDA’s accelerated approval program has allowed a large number of cancer patients of high unmet need to gain access to innovative treatments swiftly. The majority of accelerated approvals by the FDA since then included an oncology indication, with other therapy areas such as genetic and infectious diseases having far fewer such approvals.

An FDA advisory panel recently voted against withdrawing the label of immunotherapy drugs approved under the accelerated pathway in four out of six indications examined. The vote reflects the opinion of US-based physicians interviewed by GlobalData, who often comment that in cancers with few available options, new drugs need not be evaluated with the same stringent criteria used for well-served indications.

Sakis Paliouras, PhD, Senior Oncology Analyst at GlobalData, comments: “The controversy stems from the fact that a number of experts only accept an overall survival benefit as a sufficient measure of clinical benefit, as opposed to progression-free survival or response rate data. A good example is Roche’s Tecentriq in bladder cancer, which withdrew its label for treatment following chemotherapy in March 2021. In the first-line setting, Roche still hasn’t provided confirmatory data of clinical benefit in patients ineligible for chemotherapy following the IMvigor210 trial (cohort 1). However, the conditional approval was voted to be upheld based on a lack of other options for this particular patient group.”

Within the EMA, the equivalent pathway is conditional marketing authorization, and between 2006-2016, the EMA gave the nod to 30 such drugs, 17 of which were in oncology and nine in infectious diseases. While Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) and China’s National Medical Products Administration (NMPA) have accelerated/conditional programs, they have used them parsimoniously, to the detriment of cancer patients who in most indications have access to far fewer agents than their western counterparts. For both regulatory and strategic reasons, out of 88 oncology drugs approved by the FDA since 2016, only 26 have made it into Japan’s market and 12 in China’s, according to GlobalData.

Paliouras adds: “Accelerated approvals have significantly contributed to the US being the dominant country in patient access to innovative drugs among the eight major markets (8MM*). While it is clear that in cases of no clinical benefit an accelerated approval should be withdrawn, there are arguments in favor of maintaining the authorization if there are no (or barely efficacious) alternatives in a given cancer patient population.”

*8MM: US, Germany, France, Italy, Spain, the UK, Japan and China

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