The 21st Century Cures Act passed with overwhelming bipartisan support in both the House of Representatives (by a vote of 392-26) and the Senate (94–5), and now has been signed into law by the president of the United States. This represents a substantial step forward for everyone who wants to see safer and more effective medicines developed faster and more efficiently. It also represents an example of Congress working together in a bipartisan manner toward achieving something very important.
There has been a lot of talk lately about restoring American greatness. However, one of the areas where the United States clearly leads the world, and has done so for a long time, is in the area of biomedical innovation.
Even though we are leading the way, there are still many diseases or conditions where there are no safe and effective treatments currently available, and areas where we can improve efficiency. One area where the 21st Century Cures legislation will help is by providing more support to the FDA, in the form of $500 million in additional funding for the agency.
Perhaps just as importantly, this landmark bill provides greater operational flexibility for the FDA to hire highly qualified staff, so the agency can fill needed positions in a more efficient manner. Having an appropriately staffed FDA is critical to ensuring that new medicines are effectively evaluated, and where appropriate, approved in a timely and efficient manner. When safer and more effective medicines are made available faster, it benefits patients, their families and society as a whole by improving quality of life and clinical outcomes for patients, reducing the overall healthcare burden and the corresponding economic burden.
Another important focus of the legislation is on promoting specific areas of highly promising innovation, like regenerative medicine, including stem cell therapies and related approaches. Relative to this area, the legislation addresses several important issues:
- It reaffirms FDA authority over these types of promising technologies and treatments – and that helps to ensure patient safety and well-being. FDA oversight is needed to protect against clinics that operate illegally and charge patients for unapproved “treatments”, without providing any legitimate evidence of safety, effectiveness, or quality control – and without obtaining truly informed consent from patients or their family;
- It explicitly authorizes the FDA to use mechanisms such as priority review and accelerated approval to expedite development and facilitate the regulatory process for qualified regenerative medicine treatments that are focused on serious unmet medical needs where current standard of care is inadequate;
- For appropriately qualified therapies, it also enables the potential use of supplementary data such as patient registries and other sources of real world evidence, like electronic medical records. These can be used to compliment data from clinical trials and provide additional evidence of safety and effectiveness for innovative therapies relative to current standard of care;
- It also mandates the establishment of clear standards for these types of products through the National Institute of Standards and Technologies (NIST) and provides funding for the creation of those standards.
These represent meaningful steps forward for the field of regenerative medicine, by helping to create a more efficient development path, yet doing so in the context of helping to ensure patient safety and well-being. That means a victory for everyone.
Dr. Gil Van Bokkelen is the chairman and CEO of Athersys, Inc., a biopharmaceutical company based in Cleveland, Ohio. He also serves as the Chairman of the Board of Governors for the National Center for Regenerative Medicine (NCRM).
The views expressed by authors are their own and not the views of The Hill.