In the United States, a Rare Disease is usually defined as a disease or condition that affects less than 200,000 people. This is usually an estimate and may change over time. Here are the top 5 reasons for conducting clinical trials on Rare Diseases:
Reason 1: Government Programs & Financial Aids
There are different programs offered to encourage research on Orphan or rare disease. Some of which include:
- Orphan-Drug Designation Program
- Humanitarian Use Device Designation Program
- Orphan Product Development Grants
Reason 2: Academic-Industrial Partnerships
The combination of academic intellect and infrastructure provided by the industry may help in the successful development of a new drug product that could turn out to be a lifesaving treatment.
Reason 3: Patient Advocacy Groups for Rare Diseases
Advocacy groups are often formed by patients with rare diseases as way of providing encouragement and sharing knowledge that supports research and also helps others patients with similar needs. Such groups can serve as a patient recruitment pool and also help with research funding.
Reason 4: Clinical Trial Design
Though the design of a Rare Disease clinical trial has its own challenges, the sample size is usually much smaller when compared to other clinical trials.
Reason 5: Higher Rates of Regulatory Success
There appears to be fewer hurdles to approval when compared to other trials and the Food and Drug Administration (FDA) has developed the three successful approaches to make drugs for rare diseases available as rapidly as possible:
- Priority Review
- Accelerated Approval
- Fast Track
Despite the smaller patient pool for rare disease R&D and other challenges the above factors have helped in making progress in the field of rare diseases.