Written by Jeffrey C. Wu, Ph.D. and Dan Hart, Ph.D.

I. Overview

The December 8, 2016 passage of the 21^st Century Cures Act by Congress is expected to lead to sweeping changes to the biotech, medical device and health industries and streamline the regulatory system for approval of both drugs and medical devices. The Obama-supported bill was years in the making and is predicted to provide strong benefits to the biotech, pharmaceutical, and medical device industries. It received overwhelming bipartisan support with the Senate voting 94-5 and the House voting 392-26 in support of the bill. President Obama is expected to sign the bill before the end of the year.

Some key aspects of the bill include regulatory changes to expedite drug and medical device approval at the FDA, a “Eureka prize” competition to advance biomedical research, federal agency directives to require equal insurance coverage for mental health illnesses relative to physical illnesses, and billions of dollars in new funding for the NIH. The funding for the NIH includes three major projects: $1.8 billion in funding for the “Cancer Moonshot” project, $1.4 billion for the Precision Medicine Initiative to collect genetic data on one million American volunteers to enhance personalized medicine, and $1.6 billion for the BRAIN initiative to enhance understanding of the human brain. The Precision Medicine Initiative further includes the addition of a new FDA drug category: the “Precision Drug or Biological Product” category.

II. Expedited Drug and Medical Device Approval at the FDA

The changes to drug and medical device approval at the FDA include streamlined clinical trial requirements and a streamlined data review program, expedited patient access to drugs designated as “breakthrough therapies,” facilitated approval of certain drugs such as antibiotics or orphan drugs for use in a limited population of patients, expedited review for vaccines, the reauthorization of the rare pediatric disease priority review voucher incentive program, expedited medical device review, and a general accelerated approval development plan.

Although all biotech and pharmaceutical companies should benefit from the accelerated approval development plan, companies with projects specifically focused on vaccines, rare pediatric diseases, and “breakthrough therapies” should take special note of the regulatory changes provided in the new Cures Act.

III. National Cancer Moonshot Project and “Patents 4 Patients” at the USPTO

The National Cancer Moonshot project and the accompanying “Patents 4 Patients” at the USPTO (also known as the “Cancer Immunotherapy Pilot Program”) should provide significant benefits to companies developing cancer therapies and seeking patent protection for such therapies. Patents 4 Patients aims to cut the time it takes to review cancer immunotherapy applications significantly by issuing final decisions in one year or less after filing at the USPTO.

Companies seeking to take advantage of the “Patents 4 Patients” program must file applications containing one or more claims to a method of treating a cancer using immunotherapy and file a grantable petition using the patent electronic filing system. The program does not require an additional fee. The application must not currently be under a final rejection in prosecution at the USPTO.

Please see this USPTO link for additional information on the Patents 4 Patients program: here.

IV. Precision Medicine Initiative and Precision Drugs or Biological Products Category Created

The Precision Medicine Initiative, which seeks to review the genetic data of one million Americans, is expected to provide significant breakthroughs in the understanding of genetic diseases. However, biotech and pharmaceutical companies may find the creation of a new drug category pertaining to personalized medicine to be of particular interest. The 21^st Century Cures Act creates a new category for precision drugs or biological product therapies that target specific biomarkers for a therapeutic response. The new category requires the development of new regulations regarding the design of studies to demonstrate the validity of a biomarker as a predictor of drug/ biological product response and to evaluate the benefit and risks of clinical trials limited to patient population subsets identified using such biomarkers. Companies developing such personalized medicine therapies would be well-advised to review the development of such FDA regulations closely.

V. Conclusion

The passage of 21st Century Cures Act is expected to produce landmark changes to the FDA, biotech, medical device, and health insurance industries. Companies developing personalized medicine products, cancer immunotherapies, antibiotics, orphan drugs, or medical devices should take careful notice of new benefits and regulatory changes provided in the Cures Act. Overall, the passage of the Cures Act appears likely to provide a significant boost to the biotech and pharmaceutical industries.