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Recent FDA Decisions Signal Shift, with Troubling Implications for Patients and Medical Innovation

Drug development has always required a surfeit of intestinal fortitude. It can take hundreds of millions of dollars and a decade or more to bring a new drug to market, and each phase of development—from structuring a clinical trial to securing reimbursement from insurers—carries substantial risk. No entity plays a more central role in the process than U.S. Food and Drug Administration (FDA), which is charged with evaluating the safety and efficacy of new drugs, as well as determining the indications and patient populations for which a drug may be prescribed.

While perception of the FDA ranges from trusted gatekeeper to beleaguered bottleneck, in recent years the biopharma industry has been encouraged by a sense of growing transparency and consistency in interactions with the FDA—an environment in which drug developers can better assess and mitigate risks, which in turn leads to improved processes, better outcomes for patients, and a greater appetite for investment in innovation. Yet two recent decisions by the agency are deeply at odds with that sentiment, with troubling implications for both drug developers and patients.

Last week’s approval of Lynparza for maintenance treatment of recurrent ovarian cancer, viewed alongside the FDA’s eteplirsen approval in Duchenne Muscular Dystrophy late last year, suggest a disturbing shift in the agency’s approach to evaluating new treatments and expanding indications of existing drugs, characterized by (1) a reduced standard for evidence required to establish efficacy; and (2) an increased potential for industry/sponsor influence to impact regulatory decisions.

These new standards have the potential to lead to more product approvals and label expansions which may, in the short term, benefit patients and increase competition. However, these new standards will also result in reduced scientific rigor and increased reliance on all forms of direct and indirect influence on FDA decision making. In addition, these new standards leave payors and prescribing physicians in difficult positions with drugs having the same indication yet widely disparate levels of evidence. 

The long-term implications for patient care and for the healthcare system are quite negative given the reduced evidence of benefit required to introduce expensive pharmaceutical products, and the increased reliance on lobbying, activism and influence pedaling over scientific rigor. There are also numerous near-term implications for drug development and regulatory strategy at many companies because the risk to reward ratio for high-risk drug development and hypothesis testing has been fundamentally altered.

In light of these new FDA standards, there is strong incentive to reduce innovation and drug development risk taking, and to rely instead on short cuts, existing clinical precedents rather than attempting to establish new ones, and on political pressure and influence rather than regulatory and scientific rigor. This is a disservice to patients and to the healthcare system.