New US Law Provides Opportunities for Drug and Device Developers
By Michael J. Werner, Partner, Holland & Knight, LLP
In early December, the US Congress will enact the 21st Century Cures Act (the Act), a bi-partisan effort to streamline regulatory review for drugs and devices, especially those targeted at rare diseases and unmet medical needs. The Act also has provisions to specifically support new technologies such as regenerative medicine and genomics. Biotech, pharmaceutical and medical device companies seeking to get products approved in the US will have new opportunities for success with the passage of the Act.
The Act contains many provisions that will optimize drug and medical device review and approval. Some of these include:
- It creates a regulatory pathway for new biomarkers;
- It clarifies the authority of the FDA with regard to genetically targeted drugs for rare diseases; and
- It reauthorizes the pediatric rare disease priority review voucher program allowing sponsors of products for pediatric rare diseases to get special treatment at FDA.
In addition, it allows sponsors of genetically targeted or variant protein targeted drugs to rely on data for the same or similar technology from previously approved applications by the same sponsor.
The Act improves the regulation of combination products – such as those products that contain both a drug and device– by requiring that FDA meet with sponsors and agree early in development how to best study the combination product to meet the standard for approval.
Regarding regenerative medicine, the Act creates a new designation for a “regenerative medicine advanced therapy” product. These are cell therapy, regenerative medicine, and tissue engineered products for serious and life threatening conditions. This designation provides the sponsor with a guaranteed early meeting with FDA as well as access to priority review and accelerated approval programs. In addition, the Act facilitates the development of standards to support the development, evaluation, and review of regenerative medicine and related products.
Two other key provisions are:
- The Act establishes a breakthrough medical device pathway, providing sponsors of such devices with additional regulatory support from FDA to speed review; and
- The establishment of a priority review voucher program to encourage the development of drugs and vaccines for agents that present national security threats. This voucher will provide sponsors of countermeasures with priority review at FDA.
The Act modernizes FDA’s approach to clinical trials by requiring the agency to hold a public meeting and issue guidance to assist sponsors in incorporating adaptive designs and novel statistical modeling into new drug applications. It also requires the FDA to evaluate the use of real world evidence (such as from patient registries) that could support the approval of a new indication for a previously approved drug and to help support or satisfy post-approval study requirements.
Regarding medical research and genomics, the Act supports the National Institute of Health’s “Precision Medicine Initiative”. This program is designed to “leverage advances in genomics, emerging methods for managing and analyzing large data sets while protecting privacy, and health information technology to accelerate biomedical discoveries”. The Initiative contains many research and health information technology elements. A key component is collection of biological samples from one million Americans in an attempt to “fuel the development of new treatments for disease, and catalyze a new era of data-based and more precise preventive care and medical treatment.” The Act authorizes funding to implement this program.
The legislation contains many other provisions relating to research, drug discovery and development. Once enacted, the various US government health agencies – FDA, NIH, and other DHHS agencies – will be developing regulations, guidance, and programs to implement the Act’s provisions. Companies should monitor these events closely.