fda – EJP RD – European Joint Programme on Rare Diseases https://www.ejprarediseases.org Mon, 28 Aug 2023 08:05:56 +0000 en-GB hourly 1 https://wordpress.org/?v=5.9.9 https://www.ejprarediseases.org/wp-content/uploads/2020/11/cropped-index-32x32.png fda – EJP RD – European Joint Programme on Rare Diseases https://www.ejprarediseases.org 32 32 Announcement of New Grant Funding Opportunity for Rare Disease Research https://www.ejprarediseases.org/announcement-of-new-grant-funding-opportunity-for-rare-disease-research/?utm_source=rss&utm_medium=rss&utm_campaign=announcement-of-new-grant-funding-opportunity-for-rare-disease-research Mon, 28 Aug 2023 08:05:53 +0000 https://www.ejprarediseases.org/?p=17458 The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2023 – FY 2025 to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)
Receipt Dates: October 24, 2023; October 22, 2024

FOA Number: RFA-FD-23-001

• Purpose: To fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions.

• Focus: Efficiency, innovation, impact, collaboration, leveraging patient input, infrastructure and financial resources

• Efficiency: Encourage efficient and innovative clinical trials in all phases of product development through adaptive and seamless trial designs, basket, umbrella and platform trials studying multiple rare diseases/products and the use of data modeling and simulations. Allow additional total costs up to $250,000 per year for studies using efficient and innovative designs.

• Contact: Katherine Needleman, Director, Orphan Products Grants Program

E-mail: katherine.needleman@fda.hhs.gov

• Additional Information: OOPD Website

]]> FDA Rare Neurodegenerative Disease Grant Program https://www.ejprarediseases.org/fda-rare-neurodegenerative-disease-grant-program/?utm_source=rss&utm_medium=rss&utm_campaign=fda-rare-neurodegenerative-disease-grant-program Fri, 21 Oct 2022 08:38:48 +0000 https://www.ejprarediseases.org/?p=13961 In addition to the Orphan Products Grants Program that the Office of Orphan Products Development (OOPD) currently administers, a new grant program was established this year by the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS) named the FDA Rare Neurodegenerative Disease Grant Program. This new program will be administered by OOPD to promote medical product development for rare neurodegenerative diseases such as ALS.
More information about the programme : Rare Neurodegenerative Disease Grant Program | FDA

To plan for future RFAs for this new program, FDA recently announced a call for comments on current funding needs in the rare neurodegenerative disease space that could be supported by grants from the Office of Orphan Products Development. If you have interest in this space, please consider providing comments to the open docket. The comment period closes on November 28, 2022.

]]> NIH/FDA Virtual Workshop on Regulatory Fitness in Rare Disease Clinical Trials https://www.ejprarediseases.org/nih-virtual-workshop-on-regulatory-fitness-in-rare-disease-clinical-trials/?utm_source=rss&utm_medium=rss&utm_campaign=nih-virtual-workshop-on-regulatory-fitness-in-rare-disease-clinical-trials Fri, 08 Apr 2022 16:22:30 +0000 https://www.ejprarediseases.org/?p=12026 The National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) and the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) are hosting a jointly sponsored virtual workshop to explore Regulatory Fitness in Rare Disease Clinical Trials.

The virtual workshop will take place over two days on May 16th – 17th.

Academic investigators, patient groups, and small or emerging pharmaceutical and biotechnology companies play a critical role in rare disease drug development and are often the sponsors for rare disease drug development, but they may lack regulatory experience. This workshop focuses on academic investigators and those looking to learn how to bridge the gap between academic investigation and the regulatory aspects of drug development.

This workshop will:

  • Provide lessons on the fundamentals of drug development and discuss rare disease case studies shared by academic investigators
  • Share experiences and expertise from multi-disciplinary stakeholders
  • Discuss regulatory considerations for rare disease clinical trials
  • Explore topics such as:
    • Adequate and well-controlled trials
    • Core principles for clinical trials
    • The nuts and bolts of Investigational New Drug (IND) applications and additional considerations
    • Additional pathways to interact with the FDA
    • Case studies

Registration is mandatory to attend the workshop.

More information and registration here: https://events-support.com/events/CDER_NCATS_Regulatory_Rare_Disease_Workshop

]]> FDA announces 2022 grant funding opportunity for rare disease research https://www.ejprarediseases.org/fda-announces-2022-grant-funding-opportunity-for-rare-disease-research/?utm_source=rss&utm_medium=rss&utm_campaign=fda-announces-2022-grant-funding-opportunity-for-rare-disease-research Fri, 07 Jan 2022 15:36:30 +0000 https://www.ejprarediseases.org/?p=10710 The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for to support natural history studies for rare diseases and conditions. These studies are intended to provide acceptable data to the FDA that will substantially contribute to the approval of new products, or new indications for already marketed products. In the FDA OOPD Orphan Products Grants Program, products for rare diseases and conditions (orphan products) are defined as drugs, biologics, medical devices, and medical foods indicated to treat or diagnose a rare disease or condition with a prevalence of fewer than 200,000 people in the United States.  

Apply here: Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01)

  • Receipt Date: February 15, 2022 and February 13, 2024
  • FOA Number: RFA-FD-22-001

Purpose: To support efficient and innovative natural history studies that advance medical product development in rare diseases/conditions with unmet needs. 

Focus: Natural history studies with high quality and interpretable data elements

Contact: Katherine Needleman, Director, Orphan Products Grants Program

More information here

]]> FDA Upcoming Grant Funding Opportunity for Rare Disease Research https://www.ejprarediseases.org/fda-pcoming-grant-funding-opportunity-for-rare-disease-research/?utm_source=rss&utm_medium=rss&utm_campaign=fda-pcoming-grant-funding-opportunity-for-rare-disease-research Thu, 30 Sep 2021 13:23:57 +0000 https://www.ejprarediseases.org/?p=9100 The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce availability of funds for fiscal years (FY) 2021 – FY 2022 to support innovative and efficient clinical trials for rare diseases and conditions.  These studies are intended to provide acceptable data to the FDA that will substantially contribute to the approval of new products, or new indications for already marketed products. In the FDA OOPD Orphan Products Grants Program, products for rare diseases and conditions (orphan products) are defined as drugs, biologics, medical devices, and medical foods indicated to treat or diagnose a rare disease or condition with a prevalence of fewer than 200,000 people in the United States.  

Apply here: Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (RO1)

  • Receipt Date: October 5, 2021
  • FOA Number: RFA-FD-21-001

•    Purpose: To fund well-controlled studies in support of a new indication or change in labeling of products to address unmet needs in rare diseases or conditions. 

•    Focus: Efficiency, innovation, impact, and added focus on leveraging patient input, infrastructure and financial resources

•    Innovation: Inclusion of a new optional innovative demonstration project

•    Contact: Katherine Needleman, Director, Orphan Products Grants Program

    E-mail: katherine.needleman@fda.hhs.gov

More information here

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