Drug Development and Approval: A Conversation with the FDA
Manage episode 320976300 series 2927358
Dr. Scott Winiecki is the Director of the Safe Use Initiative at the FDA. He completed his M.D. at the University of Maryland and his pediatric training at the Children’s Hospital of Philadelphia. After 12 years in private pediatric practice, he joined the U.S. Food and Drug Administration in 2011. After working on immune globulin products and vaccines for five years, he joined the Center for Drug Evaluation and Research (CDER) in September 2016. In his current role at the FDA, he is working to reduce preventable harm from medications by collaborating with both public and private groups within the healthcare community.
In this episode, Dr. Winiecki explains the role of the FDA in getting a drug to market. He talks about the different stages a drug has to go through before it reaches the FDA and the phases of clinical trials in assessing a drug’s suitability for public use. Dr. Winiecki gives us an inside look at the intricacies of ensuring the safety and efficacy of a drug, as well as how patients can work with the FDA in terms of drug development. Tune in to find out more.
Disclaimer: The information provided in this podcast is designed to support, not replace the relationship that exists between listeners and their healthcare providers. Opinions, information, and recommendations shared in this podcast are not a substitute for medical advice. Decisions related to medical care should be made with your healthcare provider. Opinions and views of guests and co-hosts are their own.
[1:51] Education partner: Bristol Myers Squibb
[1:54] Mary Jo introduces the guest for this episode, Dr. Scott Winiecki.
[3:14] Dr. Winiecki shares a bit about his background and his role at the FDA.
[3:58] What are some of the steps a drug typically goes through before it reaches the FDA for approval?
[5:04] What are the phases of a clinical trial and what is being assessed in each phase?
[8:01] How does the process differ if the drug is for rare diseases, or if the drug is already approved for some other use case, or approved in other countries?
[9:15] How does the FDA engage with patients that are enrolled in a clinical trial?
[9:45] What protections are in place for people participating in a clinical trial?
[10:46] One concern patients may have in deciding whether to participate in a clinical trial involving investigational drugs is whether they will be able to access that medicine after the trial ends. Dr. Winiecki explains how decisions are made regarding post-trial access to a drug.
[11:50] The FDA has a variety of different designations it gives to drugs. What does each of these designations mean?
[16:55] What are some circumstances that might cause a delay in the approval process?
[17:56] On average, how long is the development timeline for a drug to come to market?
[18:25] Who decides which disorder a drug will be approved for?
[20:20] How does it determine if a medicine would be made available over the counter or by prescription?
[20:58] What is the FDA involvement after a drug is approved?
[22:27] How would patients report side effects that they believe might be caused by a drug?
[23:19] What are some considerations in the labeling of allergens used as inactive ingredients in drugs?
[24:10] What is the role that patients play in drug development? What is the value of having the patient and families engaged and involved?
[26:41] What are some ways patients and families can engage with the FDA, apart from the PFDD meetings?
[27:40] What are some FDA programs that might focus on rare disease?
[29:23] For more information and resources, visit the FDA website.
Mentioned in This Episode:
American Partnership for Eosinophilic Disorders (APFED)
APFED on YouTube, Twitter, Facebook, Pinterest, Instagram
Call FDA — 1-888-INFO-FDA (1-888-463-6332)
Clinical trials for eosinophil-associated diseases
EOS Connections Online Community
This episode is brought to you thanks to the support of our Education Partner Bristol Myers Squibb.
“Pre-clinical research can answer basic questions about drug safety, but they’re not a substitute for ways the drug can interact in the human body.” — Dr. Winiecki
“The FDA continues to look at benefits and risks of a therapy throughout its lifecycle even after a drug is approved.” — Dr. Winiecki
“It’s also noteworthy that most rare diseases do not have any FDA approved treatments.” — Dr. Winiecki
“The FDA has a really important role in protecting patients enrolled in clinical trials.” — Dr. Winiecki
“The FDA estimates it takes about 8.5 years to study and test a new drug before it can be approved for the general public.” — Dr. Winiecki
“Patient involvement is very important and perhaps even more important in the context of rare diseases because obviously, no one knows the disease as well as the patient and what sort of outcomes or benefits would be important.” — Dr. Winiecki