New Approach to Expedited Drugs Needed: New York Times
The New York Times editorial board says that while the goal of getting new medicines to desperate patients as quickly as possible is a worthy one, the uncertainty surrounding expedited drugs has strained the public’s trust and made it easy for insurance companies to deny coverage for such drugs. The editorial notes the recent FDA approval of Zolgensma, a single-dose gene therapy that has the potential to cure spinal muscular atrophy. However, it says, Novartis announced the drug would cost about $2.1 million per patient, believed to be more than any one medication has ever cost.
“Pharmaceutical companies like Novartis receive a number of government incentives to develop treatments for rare and neglected diseases such as spinal muscular atrophy,” the Times says, “including speedier drug approvals, generous tax breaks, and extended patent protections. But because lawmakers have not tied such incentives to any price controls, and insurance may not cover all or any of the cost, families who desperately need Zolgensma are struggling to afford it.”
According to the editorial board, rapid drug reviews, which used to be the exception, are becoming the rule, with at least 60% of all new drugs being approved through such pathways over the past five years. One effect of the expedited pathways, it says, is that, on balance, more drugs are being approved with less scrutiny than in the past. “The benefit to drug makers is clear: fewer, shorter clinical trials mean a faster, cheaper regulatory process with a much higher probability of success,” the Times says. “The value to patients can be more difficult to evaluate.”
The editorial recommends that lawmakers and regulators consider:
- limiting expedited drug approvals to companies without a record of “bad behavior”;
- · capping prices for unproven medications by amending the 1983 Orphan Drug Act and the 21st Century Cures Act; and
- demanding more data by penalizing companies that don’t keep up with post-marketing studies.