Opinion: STAT+: There’s a better way to get drugs on the market: progressive approval
A progressive approach to drug approval would balance the reality of drug safety and efficacy research with the desire to get innovative products to patients faster.
The public and pharmaceutical companies are calling for changes in drug regulation and the costs of medicines. They are right to do so. Companies want to see products approved faster for financial reasons, and patients want medicines that can improve their lives. The two are inextricably tied, and realizing those goals requires tossing out the broken system we have in exchange for a superior system that is richer in data and evidence. It also means that we all have to embrace the simple truth that taking these chemicals will always come with risk.
The U.S. regulatory system evolved to be more risk-averse as the FDA takes live fire every time an approved medication leads to severe adverse side effects and deaths. The most significant recent changes to drug regulation, the accelerated approval program, has brought about justified concern and increasingly complex scientific debates over the perceived public benefit of these programs. For certain, manufacturers have benefited by charging very high prices for unproven medications and the process to withdraw an ineffective medicine is unnecessarily complex.
In 2018, the Food and Drug Administration Safety and Innovation Act (FDASIA) authorized the FDA to apply an accelerated approval mechanism more broadly and created the breakthrough therapy designation, both of which would likely have been considered faster horses by Henry Ford. The winners have clearly been industry, and the losers are the vast majority of patients for whom the debate on value rages on.
