North America Legal and Regulatory Affairs Watchdog Update June 2023

Recommend

Darshan Patel, MS
Fred Hutch Cancer Center
United States

FDA Seeks Input on Boosting Data and Technology Capabilities:

The US Food and Drug Administration (FDA) is requesting stakeholders’ inputs on how it can advance its data and technology capabilities under the data modernization framework. These responses will be used to “define and shape” its data and technology strategic plan covering fiscal years 2024 to 2027.

According to the agency, “This plan will define and shape the future course of FDA’s data and technology capabilities, building on the existing FDA Modernization Framework.” This plan is scheduled to be published by 30 September 2023.

The agency’s modernization framework, developed by the Office of Digital Transformation (ODT) includes three modeled implementation plans -

Technology Modernization Action Plan (TMAP),
Data Modernization Action Plan (DMAP), and
Leadership Modernization Action Plan (LMAP)

The TMAP aims to enhance the agency’s hardware and software technologies to better accommodate novel sources of data, such as real-world data. This plan was announced back in September 2019.

The FDA said the new Data and Technology Strategic Plan will build on the modernization framework. The agency is taking the iterative approach in developing the plan by first collecting public input, releasing a draft version for comment, and publishing a final version after considering any feedback it receives. “Stakeholder input is crucial for developing a comprehensive plan that best meets the needs and goals of industry and the agency,” said the FDA.

The agency said it wants input in several specific areas, including what outcomes stakeholders think the plan can help them achieve; challenges stakeholders face in achieving those outcomes, and areas in which the FDA could strengthen its data and technical capabilities. Input is also being sought on potential opportunities and risks the agency could face with its plan, industry changes and trends that could affect the plan; and how the agency can best communicate and engage with stakeholders.

.

FDA and Industries are Taking Steps on Streamlining Cell Therapy Regulations

Since the inception of Cell Therapy, there’s been little consensus on what a streamlined regulatory pathway might look like for cell and gene therapies, but in recent discussions, stakeholders seemed to agree on several ideas, such as allowing sponsors to extrapolate data from earlier trials for new product iterations; taking a platform approach to potency assay requirements; moving toward real-time communication between regulators and sponsors; and facilitating greater alignment between international regulatory agencies.

During a roundtable discussion with the FDA, academia, and industry, the sponsors and researchers sought clarity from the FDA, particularly on where regulators draw the line in determining when a new iteration of a cell therapy becomes an entirely new product.

In the recent annual meeting of the American Society of Gene & Cell Therapy (ASGCT), Peter Marks, the director of the FDA's Center for Biologics Evaluation and Research (CBER) commented on questions regarding the current cell and gene therapy regulations and what the future holds.

Given the bespoke nature of many cell and gene therapies, particularly autologous cell therapies derived from a patient's own biological material, regulators and drugmakers often describe the treatment as a process and not merely a product.

During the roundtable event, Peter Marks commented on the subject "What changes are acceptable before you have a new product? We're trying to help define that, yes, we have a definition, but the question is, 'Where should that boundary be, and what could we do to allow ourselves to bootstrap from one product to the next without having to go through another full series of clinical trials?' I don't know that we have all the answers, but I think we're going to have to find our way through them."

Given that drug development often happens at a global scale, the agency acknowledged the need to better support drugmakers struggling to navigate different countries' regulatory standards in seeking to make their cell and gene therapies available to patients outside the US. As the agency indicated, one of the potential solutions could be applying an approach like the FDA Oncology Center for Excellence's Project Orbis, which aims to make it easier for drugmakers to share and submit data to regulators from other countries who participate in the project.

Since the regulatory pathway for cell and gene therapies remains in flux and can differ case-by-case, sponsors, and the FDA both appreciate the importance of transparent communications as early as possible in the drug development process.
Going forward, be it through a Project Orbis-type approach or other strategies, the FDA underscored the importance of aligning global regulatory frameworks, to not only ease the path for sponsors but also ensure that patients around the world have better access to these therapies including in low- and middle-income countries.

As to forthcoming guidelines that could further explain the FDA's regulatory thinking, the agency added that CBER is trying to get these documents out faster since they can take so long for the agency to draft, revise, and publish that drugmakers sometimes worry the final version might not even reflect the agency's current thinking.

.

References:

Data and Technology:

https://www.raps.org/news-and-articles/news-articles/2023/4/fda-seeks-input-on-boosting-data-and-technology-ca

Precision Medicine Online:

https://www.precisionmedicineonline.com/regulatory-news-fda-approvals/fda-industry-taking-steps-streamline-cell-therapy-regulation-though

.

Updates and Regulatory News

Health Canada

What’s new in Biologics, radiopharmaceuticals and genetic therapies can be found at:

https://www.canada.ca/en/health-canada/services/drugs-health-products/biologics-radio-pharmaceuticals-genetic-therapies/what-new-biologics-radiopharmaceuticals-genetic-therapies-health-canada.html

Information for health product manufacturers and distributors in relation to COVID-19:

https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry.html

Health-related consultations:

https://www.canada.ca/en/services/health/consultations.html

FDA

Cellular, Tissue and Gene Therapies Advisory Committee Meeting: Biologics License Application (BLA) 125781 from Sarepta Therapeutics, Inc. for delandistrogene moxeparvovec – May 12th, 2023

https://www.fda.gov/advisory-committees/cellular-tissue-and-gene-therapies-advisory-committee/2023-meeting-materials-cellular-tissue-and-gene-therapies-advisory-committee

FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa – May 19th, 2023

https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa

Regulatory Education for Industry (REdI) Annual Conference 2023 – June 5th to 9th, 2023

https://www.fda.gov/drugs/news-events-human-drugs/regulatory-education-industry-redi-annual-conference-2023-06052023

RDEA Pilot Program 2023 Public Workshop – June 7th to 8th, 2023

https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/rdea-pilot-program-2023-public-workshop-06072023

OTP Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls – June 8th, 2023

https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/methods-and-approaches-capturing-post-approval-safety-and-efficacy-data-cell-and-gene-therapy

Vaccines and Related Biological Products Advisory Committee – June 15th, 2023

https://www.fda.gov/advisory-committees/advisory-committee-calendar/vaccines-and-related-biological-products-advisory-committee-june-15-2023-meeting-announcement

What’s new for Biologics including Approval and Determination Letters:
https://www.fda.gov/vaccines-blood-biologics/news-events-biologics/whats-new-biologics