The 21st Century Cures Act Will Be Implemented Piecemeal

fdaBy Jason Liu

As technology and medicine advance, the need to streamline and regulate medicine will increase. One can visit a virtual doctor, connect medical devices to the internet, and access cutting-edge gene therapy precision medicine. However, government agencies work with laws that never considered these innovations. To update these laws, the House passed the 21st Century Cures Act in 2015. The Act currently sits in the Health, Education, Labor and Pensions Senate (HELP) committee. Congress may also break the bill into smaller pieces of legislation.

Lamar Alexander (R-Tenn.), the leader of the HELP committee, recently stated that the panel will divide the 21st Century Cures Act into smaller pieces of legislation. The Act has stalled in the Senate because Democrats and Republicans disagree on how to fund the bill. Beginning Feb. 9, the committee will vote on at least seven bipartisan bills ranging from expediting therapies for rare diseases to improving electronic health records.

Why is the 21st Century Cures Act important? 

The Act proposes broad changes to medical community and regulatory pathways. Passed in 2015, the Act covers medical research, practice, funding, FDA approval processes, and Medicare. The 21st Century Cures Act proposes the following:

An accelerated FDA approval process: The Act will allow evidence in some FDA approval processes that do not have to come from Randomized Controlled Trials. This will alleviate the typical 20-to-40 year time period between a discovery and widespread acceptance of a new treatment.

A new biomarker research framework: The use of biomarkers for research will better facilitate research. For example, patients with phenylketonuria are known to test positive for the neurotoxin phenylalanine. This toxin causes a reduction in IQ. All research projects require a clinical “endpoint,” which is the studied abnormality that constitutes one of the target outcomes of the trial. However, IQ is a difficult endpoint to measure. If IQ is used as the endpoint, the progress of research will slow down, and it would be difficult to develop new treatments. However, if phenylalanine is used as a biomarker to create surrogate end points, research progress can be much faster.

The inclusion of a Limited Population Antibacterial Drug (LPAD) pathway: This change allows the FDA to approve new antibiotics and antifungal drugs that address unmet medical needs for limited populations of patients. Drugs approved under this pathway may take less time to develop than current expedited FDA pathways. This change is in response to the rising threat of antibiotic resistance to drugs.

“Easier” FDA requirements for medical devices: The FDA will be able to approve medical devices more quickly. Although medical devices vary wildly, medical devices may take 3-7 years from concept to market release. The assessment of devices will depend more on post-market studies, and case studies rather than full clinical trials will be accepted in the approval process.

For more information, you can view the FDA website on current medical device regulation and guidance.


In his press release, Chairman Alexander revealed that the senate committee will implement the Act’s proposed changes. But the committee should critically review its’ priorities in the Act. As with all legislation, the committee wants to implement the parts of the bill that garner bipartisan support. These parts include: improved health records, medical device accountability, and therapies for rare diseases.

However, a brief look over the piecemeal bills reveals that some of the major changes of the 21st Century Cures Act have yet to be addressed as separate bills. The changes not addressed include the accelerated FDA process and the biomarker framework for research. The first piecemeal bills do not appear controversial because there is bipartisan support. But it is difficult to predict how quickly or in what order the committee will institute these changes. The committee might also choose to leave some things out.

Regulators should keep a critical eye on developments. Advocates for the 21st Century Cures Act should create a checklist (of what) from the original act to see if the committee left anything out.

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