Educational Resources for Patients, Advocacy Groups, Research Investigators and Drug Developers regarding Rare Diseases

The US Food and Drug Administration (FDA) has recently published a list of “Educational Resources for Patients, Advocacy Groups, Research Investigators and Drug Developers”.  These resources are available from the FDA’s “Developing Products for Rare Diseases & Conditions” page, in the “Resources for You” section.  Rare Disease research is supported by the Orphan Drug Designation program (OOPD), and provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.  Rare Disease Day 2014 was celebrated on February 28th, 2014, and was established to raise awareness about rare diseases, the challenges encountered by those affected, the importance of research to develop diagnostics and treatments, and the impact of these diseases on patients’ lives.     

 The goal of posting these recorded webinars is to educate Patients & Advocacy Groups, Research Investigators, and/or Drug Developers about Rare Disease issues.  Listed below are the available short presentations, including a short description.  They are best launched from the Rare Disease website.  (Note: all webinars are Adobe Connect presentations)


Essentials of Interacting with the FDA

                Overview of the meeting process between regulators and study sponsors/staff

                Types of Meetings (A/B/C)

                Phases of Development, timelines

Regulatory Application for Products Intended to Treat Rare Diseases

                Standards for Approval

                Regulatory Path (Pre-IND Consultation, IND Application, NDA/BLA, Approval, Post-Marketing)

                Adverse Events and Exemptions

Introduction to Patient Focused Drug Development

                Several modules on FDA background, drug development process

                Patients are consulted regarding symptoms and daily impacts

Frequently Asked Questions about Expanded Access

                Expanded Access – facilitate availability of IND’s to patients

                Serious/life threatening

                Benefits outweigh risks

FDA & NIH Science of Small Clinical Trials Course

                Overview of the clinical trials process

                Rare diseases and small populations

Workshop on Natural History Studies in Rare Diseases:  Meeting the Needs of Drug Development and Research

                2 days of conference proceedings

Sponsor-Investigator Roles and Responsibilities in Clinical Investigations for Drug and Biological Orphan Products

                Sponsors Roles

                Investigators Roles

                FDA Oversight

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