FDA is currently using 3 programs to expedite development of COVID-19-related therapies, with the goal of making new treatments available to patients in the US as quickly as possible. Note that the need and ability to utilize these programs will be dependent upon the status of the COVID-19 pandemic at the time your drug product is available and whether you are in a position to potentially engage with FDA in these programs.
At a high level, there are basically 3 (inter-related) programs that FDA has in place to make drug product available for COVID-19 patients in an expedited manner or under an “emergency” use status for products that are ready to initiate clinical studies, are already undergoing clinical development prior to approval of a new drug application, or are already approved in other indications:
The CTAP program is a special emergency program created by FDA, announced on 31 March 2020, to expedite their review of clinical study protocols and development programs for possible therapies to treat COVID 19. The overarching goal is to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful. This program may be worth considering for your product if it has strong scientific merits as a COVID-19 therapy, is at an appropriate stage of development for clinical studies, and can be identified as a possible priority product in US Government documents. The CTAP Program has been rapidly triaging requests from product developers with potential COVID-19 therapies and providing interactive input on development plans. Many protocols are being provided review within 24 hours of submission. FDA is also working to expedite quality assessments on these products and mitigations for potential supply disruptions. It should be noted that, for products still in very early development, FDA is continuing to recommend submission of a Pre-IND request to the appropriate Division at FDA. The Division of Antivirals has provided some guidance for this process (https://www.fda.gov/emergency-preparedness-and-response/mcm-issues/covid-19-therapeutics-general-information-interested-stakeholders). FDA’s press release FDA Combating COVID-19 with Therapeutics contains many helpful links for interested product developers.
Emergency Use Authorization (EUA) gives FDA the authority to allow unapproved medical products or unapproved uses of approved medical products to be used in an emergency to diagnose, treat, or prevent serious or life-threatening diseases or conditions caused by chemical, biological, radiological, and nuclear (CBRN) agents when there are no adequate, approved, and available alternatives. For COVID-19, FDA has already granted EUAs for a range of medical devices (diagnostics, personal protective equipment, ventilators) and chloroquine phosphate and hydroxychloroquine sulfate. To qualify for an EUA, a product generally needs to be in an advanced phase of development or already approved and on the market, with submission of a request for an EUA and full supporting data for the proposed use. FDA’s Guidance for Industry and Other Stakeholders: Emergency Use Authorization of Medical Products and Related Authorities provides more information about EUAs and the process for making a request. This guidance recommends that any sponsor considering an EUA request be in contact with the appropriate FDA Center prior to submitting any formal request. FDA expects product development to continue in parallel with an EUA.
Expanded Access provides a potential pathway for a patient with an immediately life threatening condition or serious disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Expanded Access includes expanded access for individual patients including for emergency use, expanded access for intermediate-size patient populations under a treatment protocol submitted to an existing IND, and expanded access for widespread treatment use through a treatment IND or treatment protocol designed for use in larger patient populations. For COVID-19, under the CTAP program, FDA has prioritized the review of single patient expanded access requests to occur around-the-clock, generally within 3 hours of request.
Thus far, these programs are focused on quickly delivering potentially effective COVID-19 therapies to patients as quickly as possible or expediting the review of development programs and protocols to test potentially effective COVID-19 therapies. The CTAP program or other FDA communications have yet to address whether there will be additional expedited processes available for review and approval of marketing applications, beyond potential priority review, for therapies that demonstrate effectiveness for the treatment of COVID-19 in clinical trials.
Rho is continually evaluating new information released from FDA regarding the Agency’s response to the COVID-19 pandemic, and our regulatory experts are available to consult with anyone attempting to determine if any of these pathways might be applicable to their product development program.
Kevin Barber, PhD, RAC, PMP, Vice President of Regulatory Strategy & Submissions, has more than 20 years of experience in regulatory affairs and product development, working for both sponsor companies and CROs, across all stages of development from pre-clinical through product launch and post-approval life cycle management. He has led the preparation and execution of integrated regulatory strategy and clinical development plans for drug, biologic, and medical device products in therapeutic areas including dermatology, nephrology, urology, women’s health, CNS/neurology, cardiovascular diseases, virology, oncology, immunology, infectious diseases, blood products, and gene therapy. Dr. Barber has significant experience preparing and filing regulatory submissions, including more than 45 US INDs and more than 40 marketing applications in the US, Canada, Europe, Latin America, Australia, and New Zealand. He also has experience with medical device and in vitro diagnostic development programs and regulatory submissions including pre-IDE meetings, IDEs, 510(k)s, and PMAs.
Theresa Zucchero Scocca, PhD, RAC, Research Scientist, manages and contributes to multiple integrated product development programs at Rho and has over 18 years of experience in research, scientific and regulatory writing, and project management. In addition to leading programs ranging from the preclinical through the marketing application stages, her experience includes authorship of multiple regulatory and clinical documents, including draft product labels, briefing packages, protocols, clinical study reports, and marketing application modules such as the Clinical Overview and integrated summaries of safety and efficacy. She has also participated in multiple FDA meetings at various stages of development. Her management and regulatory authorship experience spans drug, biologic, medical device, and combination products and a broad range of therapeutic areas, including CNS, infectious disease, gastrointestinal diseases, osteoarthritis, analgesia, asthma, dental products, ADHD, inner ear disorders, and ophthalmology.