Retooling the FDA is overdue. The pace of drug and device approvals is glacial, and the rules prohibiting discussion of off-label uses are heavy-handed. Just recently, we saw the agency turn down Avedro’s NDA for collagen crosslinking—even though it had been recommended for approval by the FDA’s advisory committee and similar technology has been used in Europe for a decade. Did that benefit, or constrain, the public good? No one wants to ease up on safeguards, but there are clearly ways in which the current system falls short.

Fixing the FDA is but one aspiration of ambitious new legislation called the 21st Century Cures Act introduced in late April. It’s loaded with admirable goals—some big, some small (see sidebar). But delivering on them is far from guaranteed; how likely is improved EHR interoperability, for instance?

Highlights of the Act

  • Increase funding for the NIH and provide student loan forgiveness for its researchers.
  • Build a global pediatric clinical trial network.
  • Accelerate “the discovery, development, and delivery” of therapy for rare diseases via a partnership among FDA, NIH and industry.
  • Improve patient access to drugs for compassionate use.
  • Expand the FDA’s Patient-Focused Drug Development  program, which takes patient’s real-world experiences into account. 
  • Encourage development of biomarkers.
  • Improve the design of clinical trials and access to trial data.
  • Improve EHR interoperability.
  • Encourage antibiotic drug development to combat bacterial resistance.
  • Many FDA approval revisions (see text).

Specific to the FDA, the package contains many good ideas for prodding the agency to accelerate new drug approvals. Three notable ones: (1) allow more use of intermediate study endpoints that suggest viability of an investigational drug, (2) collaborate with European regulatory agencies and (3) streamline review of an existing approved product that’s being considered for a new indication (this last one, while a great idea, is limited to cancer treatment.) Medical device approvals would also get a boost, as the bill would allow priority review for breakthrough technologies deemed genuinely new and broaden the definition of “valid scientific evidence” to include, among other things, international data, which is often in abundance.

But the bill’s authors backed off from one long-hoped-for update: less Draconian rules for discussion of off-label uses. An earlier draft had a placeholder for efforts to encourage “responsible communication of scientific and medical developments” beyond label indications, but it has been cut. The current draft would allow manufacturers to inform insurers about the economic benefits of off-label uses, but not doctors about clinical benefits—a telling choice of priorities by legislators. I don’t see how you can have the one without the other. The instinct to prevent bias or weak science from influencing the discussion of medical products is admirable, but we shouldn’t let that justify a gag rule that keeps doctors in the dark. Still, there are many welcome “cures” here—if Congress is willing to write the Rx.