Patient-Focused Drug Development

Additional information focusing on the patient’s experience while using an investigative drug will be included in 505b submissions.   Input on the experience will be derived from any person, including patients, family members and caregivers of patients, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers. Methodologies to collect such information will be issued in a guidance from the FDA.   Clearly, the FDA will be seeking more input regarding the experience of the patients, other than the manufacturer when determining the decision to approve a drug.

Advancing New Drug Therapies

There are new provisions in the Act which will allow the Secretary to establish a process for the qualification of drug/biologic development tools such as a biomarker, a clinical outcome assessment and any other method, material or measure that aids drug development and regulatory review.  Such submissions for the qualifications will have a transparent review process for all to see on the FDA website.  The purpose of qualifying development tools to encourage the development of genetically targeted drugs or biologics for rare diseases.   Other areas of interest will be drugs for serious or life-threatening diseases and a program will be developed to encourage treatments for rare pediatric diseases. Grants and vouchers for priority review of drugs for such conditions will be expanded by the FDA and other federal agencies.

 Modern Trial Design and Evidence Development

The Agency will consider sources for data regarding the usage or potential benefits or risks of a drug derived from sources other than randomized clinical trials. A framework for collecting this information shall be established with the consultation of industry, academia, medical professional organizations, representative of patient advocacy groups, consumer organizations, disease research foundations and other interested parties.  The goal is to consider other sources when determining regulatory approval and conditions for use of the drug/biologic.

Patient Access to Therapies and Information

Manufacturers of investigational therapies for serious diseases shall make available policies for inclusion of patients on a publicly available website. Certainly applications for regenerative advanced therapy under 505(b)(1) will be eligible for priority and accelerated review meeting certain criteria.  This will apply to cell therapy, therapeutic engineering products, human cell and tissue products, and combination products using such therapies or products.  By opening the door in this area the FDA will be issuing guidance for devices used in the recovery, isolation or delivery of regenerative advanced therapies.

Combination Products

To assist and streamline the review of all combination products consisting of a drug, device or biologic product, the Agency shall assign a Center to regulate these products.  The purpose is to streamline the review of these products and help focus on the “primary mode of action” and administer any requests for review by the sponsor of an application if the “primary mode of action” is in question and general shepherding the review process to approval.

Antimicrobial Innovation and Stewardship

The Agency shall continue to monitor, along with other federal agencies, those antimicrobial drugs which become resistant to humans. In a limited population, the FDA may approve an antibacterial or antifungal drug, alone or in combination with one or more other drugs, in a limited population those that are intended to treat a serious or life-threatening infection. Such drugs may be approved notwithstanding a lack of evidence to fully establish a favorable benefit-risk profile in a population that is broader than the intended limited population. Promotional material for such drugs will require preapproval 30 days prior to dissemination. The Agency will develop appropriate susceptibility test interpretive criteria and provide the information on its website.

Medical Device Innovation

A program will be established for expediting approval for breakthrough technologies for which there are no comparable technologies. In doing so, the Agency will work with the sponsor to help with a development plan. Certain criteria will be developed for determining if a device meets this designation.

Another area of importance and very much needed is the requirement for the Agency to clarify medical device software regulations.  With the evolving technology of medical devices, this is very much needed.

Note:  The implementation of these programs have varied timeframes from the passage of the 21st Century Cures Act on December 13, 2016