12/16/2019: FDA announced the issuance of a draft guidance entitled "Qualification Process for Drug Development Tools--Guidance for Industry and FDA Staff." Public commentary is requested and may be submitted electronically by February 14, 2020.

7/25/2019: FDA announced the establishment of a public docket to receive comments from interested parties on the taxonomy for the classification of biomarkers for use in drug development. Public commentary is requested and may be submitted written or electronically by September 23, 2019.

12/12/2018: The FDA announced a draft guidance on considerations to address when developing a biomarker, entitled "Biomarker Qualification: Evidentiary Framework." Public commentary on the guidance is requested and may be submitted written or electronically by February 11, 2019.

11/13/2018: The FDA announced a public meeting titled "Drug Development Tool Process under the 21st Century Cures Act and PDUFA VI" that will provide an opportunity for public input regarding the drug development tools qualification pathway for animal models, biomarkers, and clinical outcome assessments. Public commentary is requested and may be submitted written or electronically through the public docket until January 31, 2019.

10/30/2018: The FDA is seeking feedback on the surrogate endpoint table (SE table) to help determine its utility and to assist in developing future versions of the table. Comments must be submitted written or electronically by December 31, 2018.

7/25/2018: The FDA has fulfilled a requirement of Section 3011 with the publishing of the Surrogate Endpoint Table. This table is a list of "surrogate endpoints which were the basis of approval or licensure (as applicable) of a drug or a biological product" under both accelerated and traditional pathways. The endpoints can be used instead of clinical outcomes in some clinical trials, which has helped expedite drug development.

7/30/2019: FDA announced the availability of a draft guidance entitled "Rare Pediatric Disease Priority Review Vouchers," providing information on the rare pediatric disease priority review voucher program. Comments on the draft guidance may be submitted electronically by September 30, 2019.

12/7/2017: FDA announced the availability of draft guidance entitled "Pediatric Rare Diseases-A Collaborative Approach for Drug Development Using Gaucher Disease as a Model"

FasterCures will periodically assess this item as appropriate.

Complete.

9/30/2018: FDA initiated a pilot program to help drug developers use complex innovative trial designs, an element of the most recent PDUFA agreement.

8/29/2018: FDA launched a pilot program, the "Complex Innovative Designs Pilot Meeting Program," which provides drug and biologic companies the opportunities to discuss the use of novel complex innovative trial designs (CID) with agency staff for their clinical development programs.

3/20/2018: FDA held a public meeting entitled, "Promoting the Use of Complex Innovative Designs in Clinical Trials" with meeting materials and information now available on the FDA website. 

FasterCures will periodically assess this item as appropriate.

7/9/2019: FDA announced a public meeting entitled "Incorporating Alternative Approaches in Clinical Investigations for New Animal Drugs" to be held on July 16th. The meeting will discuss the incorporation of real-world evidence in proposed clinical investigation protocols and applications for new animal drugs.

6/10/2019: The Robert J. Margolis, MD, Center for Health Policy at Duke University announced the convening of a public workshop to consider key challenges and opportunities to establish a more systematic approach to collecting and submitting real-world data to FDA as part of evidence packages.

5/9/2019: FDA announced the availability of a draft guidance for industry entitled "Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics." Comments on the guidance may be submitted electronically until July 8, 2019.

12/6/2018: FDA released a new strategic framework for their real-world evidence program, fulfilling a requirement for this section.

11/6/2018: FDA launched the MyStudies App to collect real-world evidence from patients' mobile devices to help inform regulatory decisions around medical products.

7/31/2017: FDA announced a public workshop, "A Framework for Regulatory Use of Real-World Evidence” convened by the Duke-Robert J. Margolis, MD, Center for Health Policy at Duke University and supported by a cooperative agreement with FDA. The purpose of the public workshop is to bring the stakeholder community together to discuss a variety of topics related to the use of real-world data (RWD) and real-world evidence (RWE) in drug development and regulatory decision making.