Jim Gearhart

by Jim Gearhart

Possible Next Steps for 21st Century Cures Act: “Advancing Medical Innovation for a Healthier America”

On July 10, the House of Representatives sent what it called the 21st Century Cures Act to the Senate. Now the bill, with a wide-ranging list of changes for medical research, is in the hands of the Senate’s HELP (Health, Education, Labor, and Pensions) Committee chaired by Lamar Alexander. Two weeks ago, a report came out that Senator Alexander said could influence his committee’s response to the 21st Century Cures Act. The Bipartisan Policy Center, a think tank founded by four former senators in 2007, released “Advancing Medical Innovation,” which lays out a set of recommendations for reforming medical research and the FDA. Since Senator Alexander said that the BPC’s suggestions could end up in the 21st Century Cures Act, it could be worth a look to see what those suggestions are.

The BPC’s report focuses on the functioning of the FDA, and has four sections:

  1. Improving the Medical Development Process
  2. Increasing Regulatory Clarity
  3. Strengthening the FDA’s Ability to Carry Out Its Mission
  4. Increasing Investment in Medical Products to Address Unmet and Public Health Needs

The report echoes many themes and suggestions that appeared in the 21st Century Cures (The BPC also released a table comparing their recommendations with the bill), but it also travels over some new ground. Here are some highlights of what could become law from each section of the report.

Improving the Medical Development Process

Both 21st Century Cures and the BPC report call for a greater reliance on clinical data for new drug approvals. Like 21st Century Cures, the BPC report recommends using information from medical practice for approving new indications. The BPC adds to this idea, calling for a change to the FDA’s established Phase I – III approval process. The report endorses a move away from relying solely on randomized, controlled trials to including studies that replicate clinical treatments, using “populations where care is provided under more typical settings.”

The report includes a packet of recommendations about publicizing off-label uses of medicines. In presenting the report, former Senator (and surgeon) William Frist said that drug sponsors know what off-label uses work and that clinicians deserve to hear about them. The BPC’s report suggests a framework where pharmaceutical companies can provide, “truthful, non-misleading and scientific” data about alternate uses of approved treatments. Senator Frist insisted that current restrictions on those communications were dated and unnecessary.

The BPC report also called for a greater use of biomarkers and patient-reported outcomes, again showing a parallel with themes prominent in 21st Century Cures.

Increasing Regulatory Clarity

The BPC offered a series of suggestions for better defining who should have responsibilities over what at the FDA. A major topic here, as in the 21st Century Cures Bill, was limiting the regulation of health-related software. The FDA should have very little oversight of health apps, the BPC report. Just as 21st Century Cures set boundaries for what programs should fall under the FDA’s jurisdiction, the BPC says the fewer restrictions on new programming the better.

The BPC report took aim at one new area: the FDA’s regulatory framework around combination products. The report said a product that uses both a device and a drug to work (such as artery stents or asthma inhalers) faces confounding guidance about which FDA division should review it. To accelerate approvals, and to ensure the United States remains competitive, the BPC calls for updating and simplifying these regulations.

Strengthening the FDA’s Ability to Carry Out Its Mission

This section recommended changing the compensation rules for the FDA in order to improve recruitment and retention. It also called for new guidelines around when and how FDA personnel interact with the private sector (such as at conferences).

Increasing Investment in Medical Products to Address Unmet and Public Health Needs

Like the 21st Century Cures Act, the BPC report calls for increased attention to developing new antibiotics. It reinforces the bill’s call for less restrictive boundaries around making experimental treatments available to patients. The BPC also recommends new routes for approving what it calls “dormant therapies.”

Conclusion

It will be interesting to see in the coming months how many of these policy suggestions make it into the next iteration of the 21st Century Cures bill. It stands to reason that those areas of intersection with the current bill – such as monitoring of health IT programs and the use of ‘real-world’ data for approving additional indications – have a good chance of making it through.

Links

BPC Press Release

C-span video of Lamar Alexander’s speech

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