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    FDA Drug Approval and Drugs that Work

    In October 2023, MEHP Online’s Assistant Faculty Director Holly Fernandez Lynch, JD, MBE appeared before the United States Senate Special Committee on Aging to deliver expert testimony on the Promising Pathways Act. In the hearing, titled “Unlocking Hope: Access to Therapies for People with Rare, Progressive, and Serious Diseases,” she made a strong argument that ethical innovation and regulation of pharmaceuticals must integrate accessible, rigorous, ongoing testing.

    Promising Pathways Act, introduced by Senator Michael Braun of Indiana, would speed access to certain drugs under investigation for rare and serious diseases by creating a new category of FDA approval—provisional approval—for drugs intended for those diseases’ treatment or prevention. Provisional approval could be granted if early trials:

    • Conclude that the drug is safe,

    • Demonstrate relevant early evidence of a positive therapeutic outcome,

    • And demonstrate early evidence that the drug is at least as effective as existing treatments.

    Drugs would need to be reviewed and reapproved after a 2-year period, with the possibility of 3 additional 2-year periods, and then must either be fully approved or have their approval rescinded. In addition, the bill would create a patient registry to collect observational data about drugs that have received provisional approval.

    Core to Prof. Fernandez Lynch’s testimony [PDF] is that while the intention of the Promising Pathways Act is noble, and while the need for new drugs to treat life-threatening conditions is urgent and real, we “must be wary of unintended consequences in efforts to promote access to unproven drugs.” The goal of the FDA is not to facilitate getting more drugs onto the market, but rather, “getting patients more drugs that work.” And the Promising Pathways Act may complicate that endeavor.

    Specifically, Prof. Fernandez Lynch told the committee that although the 2-year renewal schedule is an important step forward, the bar for provisional approval is too low. The language used in the bill—"relevant early evidence” and “positive therapeutic outcomes”—is not well defined. And the patient registry—the mechanism proposed to track the effectiveness of provisionally approved drugs—would not generate high-quality evidence because it cannot facilitate blinded, randomized controlled trials.

    The consequences of this, she states, are that the Promising Pathways Act would:

    • Make it harder to determine whether drugs are effective to actually treat people with rare and serious diseases.

    • Make it harder for patients and their physicians to make informed decisions about treatment.

    • Interfere with the development of other, possibly more effective, drugs when so many resources are directed to those that are currently on the market.

    As an alternative, Prof. Fernandez Lynch reiterates the need for measures she has called for elsewhere in her scholarly work:

    • Clarifying that the FDA has the authority to require and enforce post-market efficacy studies when it approves drugs of uncertain benefit, to ensure that drugs available on the market are in fact effective treatments.

    • Promoting and funding measures to make clinical trials more accessible, so that every patient who wishes to participate in a trial is able to do so.

    Ultimately, Prof. Fernandez Lynch’s testimony points to a different, more effective approach to addressing rare, progressive, and serious disease. The sad reality, as she tells Congress, is that the lack of good treatment options is “not because FDA is blocking access or refusing… to approve new drugs. Instead, it is because in too many areas, the science has not caught up to the need.” The imperative, then, is not to revise the regulatory process. Rather, it is to fund rigorous, targeted research trials that are accessible to patients and provide specific, actionable evidence to investigators attempting to solve the problem.

    Additional Resources

    Learn more about the ethics and regulation of clinical research in:

    And learn more about Holly Fernandez Lynch’s work on the regulation and approval of drugs with uncertain benefit by reading: