In the United States, an estimated 30 million individuals are affected by rare (orphan) diseases or conditions, yet historically, the development of new treatments for these has been limited. This is primarily due to the economic challenges associated with recouping the costs of drug development for diseases that affect a smaller number of people. To address this, the Orphan Drug Act was enacted by Congress in 1983 to encourage the development of drugs, biologics, devices, or medical foods that show promise for diagnosing and/or treating rare diseases. Under this act, a disease is considered "rare" if it affects fewer than 200,000 people in the U.S., or if the cost of developing a drug for the disease would not be recovered by expected sales. In this blog we will share an overview of an ODD and helpful steps to application.
Overview
Orphan Drug Act Overview Since its inception, the Orphan Drug Act has led to significant advancements in rare disease treatment. Over 4,500 orphan drug designation requests have been approved, and more than 730 drugs and biological products have been developed and approved for over 250 rare diseases. This is a stark contrast to the period between 1973 and 1983, when fewer than 10 orphan drugs were marketed.
Incentives of Orphan Drug Designation Recognizing the financial hurdles in developing treatments for a limited patient population, the FDA offers several incentives to encourage pharmaceutical companies to invest in orphan drug development:
7-Year Marketing Exclusivity: Approved orphan products receive seven years of market exclusivity.
Federal Tax Credit: Companies can receive a tax credit for clinical research expenses incurred within the United States.
Flexible Tax Credit Application: These credits can be carried back to the prior year or carried forward for up to 20 years.
PDUFA Fee Waiver: Prescription Drug User Fee Act fees are waived for orphan drugs.
Competitive Grants: Eligible companies can compete for research grants from the Office of Orphan Products Development to support clinical studies.
Regulatory Assistance: Sponsors can receive FDA guidance on designing comprehensive drug development plans.
These incentives make it financially viable for companies to develop drugs for rare diseases, fostering innovation and providing hope to patients with limited treatment options.
Preparing an ODD Application: Helpful Steps
1. Determine Eligibility
Rare Disease Definition: Verify that the drug targets a disease affecting fewer than 200,000 individuals in the United States or a disease with a higher prevalence that would not be profitable to treat without incentives.
No Existing Treatments: Ensure there are no adequate treatments available, or the drug must prove clinically superior to existing treatments.
2. Pre-Application Research
Clinical Data: Gather preliminary data to support the drug's efficacy and safety in treating the specific rare condition.
Comparative Data: If treatments are available, collect comparative data demonstrating superiority in efficacy or safety.
Scientific Rationale: Prepare a scientific rationale based on preclinical or clinical data supporting the drug's use for the rare disease.
3. Prepare the Application
Form FDA 3921: Complete and compile the necessary forms for the FDA, specifically Form FDA 3921 for Orphan Drug Designation.
Detailed Proposal: Include a detailed description of the disease or condition, the population affected, and the potential market for the drug.
Supporting Documentation: Attach all supporting scientific arguments and empirical evidence.
4. Submit the Application
Electronic Submission: Submit the application electronically through the FDA’s Electronic Submissions Gateway.
Ensure Completeness: Double-check the application for completeness and compliance with FDA requirements to avoid delays in the review process.
5. FDA Review Process
Initial Review: The Office of Orphan Products Development (OOPD) conducts an initial administrative review to check for completeness.
Evaluation: The application is then reviewed for substantial medical plausibility and eligibility criteria.
Request for Additional Information: Be prepared to provide additional information if the FDA requests further clarification or more data.
6. Receiving Designation
Approval Notification: If approved, receive the formal Orphan Drug Designation notification.
Public Listing: The designation details will be listed in the public database of the Office of Orphan Products Development.
7. Post-Designation Compliance
Annual Reporting: Submit annual reports on the drug’s development status as required by the FDA.
Maintain Eligibility: Continue to demonstrate eligibility through ongoing clinical trials and development activities.
8. Leverage Designation Benefits
Tax Credits and Grants: Apply for tax credits for clinical testing and investigate available grants.
Market Exclusivity: Upon approval, the drug will receive seven years of market exclusivity in the U.S.
9. Prepare for Marketing Approval
Clinical Trials: Plan and conduct the necessary clinical trials to obtain data for a New Drug Application (NDA) or Biologics License Application (BLA).
Regulatory Strategy: Develop a comprehensive regulatory strategy for FDA approval, leveraging the benefits and incentives associated with the Orphan Drug Designation.
Conclusion
Obtaining Orphan Drug Designation is a detailed and strategic process that requires careful planning and execution. The designation not only facilitates drug development with financial and regulatory support but also aligns with ethical goals to provide treatments for underserved patient populations. Contact Trinity M Consulting to learn more how we can help prepare your ODD application.
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