July 26, 2024

Dear Colleague,

While the FDA continues to focus on protecting the public’s health, using science to guide our decisions, and facilitating access to critical medical products, we also recognize the work of our public health partners. As public health advocates, we know that your work to better our country is invaluable. Your partnership, knowledge and engagement in the public health space are appreciated.

Updates

• July 26, 2024 - FDA alerts health care providers, compounders and patients of dosing errors associated with compounded injectable semaglutide products
• July 25, 2024 - FDA Announces National Academies Study of Supply, Market Competition, and Regulation of Infant Formula in the U.S.
• July 25, 2024 - FDA Public Health Alert for Additional Ground Cinnamon Product Due to Presence of Elevated Levels of Lead
• July 23, 2024 - FDA Roundup

FDA Voices

FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients

By: Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research

Recent rapid advances in the identification of promising drug targets and development of gene therapies offer momentum and potential to meet the needs of patients with rare diseases. In 2023, over half of all the novel drugs and biologics approved by the FDA’s Center for Drug Evaluation and Research (CDER) and the FDA’s Center for Biologics Evaluation and Research (CBER) were to prevent, diagnose or treat a rare disease or condition.

The Hub will leverage the activities of the CDER Accelerating Rare disease Cures (ARC) program and CBER Rare Disease Program and will enhance existing cross-center collaborations. In addition, a newly created senior leadership position, Director of Strategic Coalitions for the Hub (Associate Director for Rare Disease Strategy), will act as a single point of connection and engagement with outside parties on behalf of the Hub on cross-cutting rare disease-related issues.

From Awareness to Action: Tackling the Rising Burden of Common Chronic Diseases

By: Robert M. Califf, MD, Commissioner of Food and Drugs

Since graduating from medical school in 1978, I’ve seen medicine and healthcare change in unimaginable ways for the better. However, one of the most alarming changes is the recent decline in life expectancy in our country, largely due to the cumulative impact of common chronic diseases (CCDs), combined with excessive rates of suicide, drug overdose and gun violence, and, of course, COVID-19. How can it be that our prowess in technology is not translating into better health and longevity for the American public?

Guidance Documents

CVM GFI #100 (VICH GL18 (R2)) Impurities: Residual Solvents in New Veterinary Medicinal Products, Active Substances and Excipients (Revision 2)
The objective of this guidance is to recommend acceptable amounts for residual solvents in pharmaceuticals for the safety of the target animal as well as for the safety of residues in products derived from treated food-producing animals. The guidance recommends use of less toxic solvents and describes levels considered to be toxicologically acceptable for some residual solvents.

Providing Over-the-Counter Monograph Submissions in Electronic Format
This guidance is intended to assist submitters by describing the electronic over-the-counter (OTC) monograph submissions requirement in the Federal Food, Drug, and Cosmetic Act (FD&C Act) and providing recommendations and other information on how to send such OTC monograph submissions to FDA in electronic format.  This guidance finalizes the draft guidance of the same title issued on September 28, 2022.

Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products
FDA is issuing this guidance as part of its Real-World Evidence (RWE) program and to satisfy, in part, the mandate under the Federal Food, Drug, and Cosmetic Act (FD&C Act) to issue guidance about the use of RWE in regulatory decision making. This guidance is intended to provide sponsors and other interested parties with considerations when proposing to use electronic health records (EHRs) or medical claims data in clinical studies to support a regulatory decision for effectiveness or safety. This guidance finalizes the draft guidance of the same title issued on September 30, 2021.

Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products Questions and Answers
This guidance provides answers to commonly asked questions from applicants and other interested parties (collectively referred to as applicants throughout this guidance) regarding postapproval manufacturing changes (referred to as manufacturing changes throughout this guidance) made to licensed biosimilars and licensed interchangeable biosimilars. This question-and-answer (Q&A) guidance is intended to inform prospective and current applicants of the nature and type of information that applicants should provide in support of manufacturing changes to licensed biosimilars and licensed interchangeable biosimilars in different reporting categories.

View all Official FDA Guidance Documents and other Regulatory Guidance
You can search for documents using key words, and you can narrow or filter your results by product, date issued, FDA organizational unit, type of document, subject, draft or final status, and comment period.

ICYMI!

Men’s Health is Falling Behind – Some Thoughts About Why

As I described in my previous blog, the trajectory of men’s health in the U.S. is headed in a negative direction. As we develop approaches to reversing these trends of declining life expectancy, excess chronic disease and disproportionate risk taking, it behooves us to examine the potential underlying reasons for this decline.

Webinars and Virtual Workshops

2024 PDA/ FDA Joint Regulatory Conference

Day 1: Mon, Sep 9; 7:15 a.m. - 5:00 p.m. ET
Day 2: Tue, Sep 10; 7:15 a.m. - 5:00 p.m. ET
Day 3: Wed, Sep 11; 7:15 a.m. - 5:00 p.m. ET

FDA and Parenteral Drug Association (PDA) will host the 33rd Joint Regulatory Conference to emphasize the role of effective quality systems in ensuring an ongoing state of control throughout the product lifecycle.

Electronic Drug Registration and Listing (eDRLS) Using CDER Direct - 2024

September 12, 2024; 8:45 a.m. - 4:45 p.m. ET

This annual event will provide:

• A demonstration on how-to submit establishment registration and drug listing data using CDER Direct.
• An overview on registration and listing regulatory requirements and compliance framework.
• Case studies for an interactive learning experience at the end of the day.

Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases

September 20, 2024; 11:00 a.m. - 4:30 p.m.

The objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations.

Join us for a #virtual listening meeting on 9/20 to share perspectives on safety considerations for approved #GeneTherapy treatments for #RareDiseases and participation in long-term studies after receiving gene therapy.

Register to speak by 8/30:https://bit.ly/3XUEQrP

View Upcoming FDA Meetings, Conferences and Workshops
Public meetings involving the FDA: Upcoming events, past meetings, meeting materials, and transcripts

About Us

The Stakeholder Engagement Staff resides within the Office of the Commissioner and falls under the Office of External Affairs. We aim to build stronger relationships with health professional organizations, consumer groups, trade associations, patient advocacy organizations, think tanks/academia, and other stakeholders, in order to better inform our policy making process, identify policy hurdles or stakeholder misconceptions, and create strategic collaborations. For more information, please contact us at: FDAStakeholderEngagement@fda.hhs.gov.