There’s more to the 21st Century Cures Act, which passed the full House of Representatives with overwhelming bipartisan support, than $9 billion in new funding for the National Institutes of Health (NIH) to pick up the pace of medical research.
The legislation also includes a number of provisions that would speed up the Food and Drug Administration review process for new drugs and medical devices, including so-called high-risk devices. While the increased funding for NIH has met with near universal support, the FDA provisions have engendered criticism that the Senate will encounter when it takes up the legislation.
Among the provisions that would change the way the FDA reviews applications for drugs and biological agents are:
- A patient-focused drug development approach that would require the FDA to consider patient experiences, such as case studies and testimonials, along with clinical trials in the new drug review process.
- Mandate a framework for the use of biomarkers, along with other drug-development tools, and ease the process for applicants to obtain accelerated approval plans.
- For agents designated for rare conditions, the FDA would have to consider data previously submitted for a different agent that uses the same or similar underlying approach.
- Require a program to include clinical experience when considering a new indication for a previously approved agent.
- Create a streamlined program to accept clinical data summaries in support of a new indication for agents for treatment of cancer or other diseases.
- Compel manufacturers of investigational agents to make basic information on their expanded access policy available to the public.
- Require expedited guidance to give drug sponsors clarity regarding adverse events patients experience in an expanded access program.
- Create incentives for orphan drug extensions for limited populations, including extending exclusivity for six months for an agent that receives approval for a new rare-disease indication.
For device oversight, the law would make the following changes in the FDA process:
- Provide priority review for breakthrough devices.
- Allow the use of FDA-authorized third-party vendors to certify companies’ quality systems in the pre-market review process.
- Allow sponsors to submit registry data, studies published in peer-reviewed journals and data collected in foreign countries with device applications.
- Allow the FDA to grant humanitarian device exemption to diseases and conditions that affect up to 8,000 people in the United States.
- Remove the limitation on the use of non-local, or central, institutional review boards in medical device trials.
As critics line up to challenge the FDA provisions in the Senate, Sen. Lamar Alexander (R-Tenn.), chair of the Senate Health, Education, Labor and Pensions Committee that will take up the bill, has made it clear that he thinks the medical innovation process in the United States has bogged down.
“America is home to the sharpest scientists and boldest researchers who are every day finding cutting-edge cures, treatments, and devices to help make sick people well or make life easier for the disabled,” Alexander said in a statement earlier this year when releasing a report titled Innovation for Healthier Americans. “The challenge is that our process for developing and reviewing these products is out-of-date and unable to match the pace of discovery, so American patients are waiting too long for relief.”