Orphan diseases are diseases affecting fewer than 200,000 individuals (US FDA), although this number can vary depending on the regulatory authority (e.g., Europe, Australia, Japan). Because these are rare diseases, developing drugs for these indications can be challenging, both from a clinical and a financial perspective.
On the clinical side, study enrollment, retention, and patient logistics are key concerns given the limited pool of potential study subjects. In addition, rare diseases are often understudied and many have the potential for variable disease manifestations. These factors can make both designing studies and interpreting pharmacokinetic and pharmacodynamic (PK/PD), safety, and efficacy results more challenging. Also, because of the nature of many rare diseases, there are ethical concerns that may require a finer balance between patient assessments and patient burden.
Developing drugs for orphan indications can also be financially challenging. The high cost of development coupled with a small patient population can result in a lower return on investment relative to indications with a larger potential patient pool. To encourage the development of rare disease therapies, the FDA supports incentives for companies developing orphan drug products, including tax credits, grant funding, assistance for clinical research, and extended marketing exclusivity. This support can both expedite the drug development process and improve profitability after drug approval.
Because of the many challenges (and opportunities) associated with orphan drug development, it is important to have individuals on your team who can provide sound strategic advice and who are knowledgeable about both the clinical and regulatory aspects of drugs for rare diseases.
Nuventra’s Orphan Disease Experience
Nuventra can perform any of our wide range of services for drugs to treat Orphan Disease indications. Our experience includes drugs, biologics, and combination products.
Some of our recent project experience in this area includes:
- BLA Submission
- Clinical Development Plan
- Clinical Pharmacology & Pharmacokinetics
- Data Management
- EMEA Scientific Advice
- IND/BLA/NDA Submissions
- Orphan Application Preparation
- Overall Program Strategy
- Phases 1-3 Clinical Studies
- Preclinical Studies
- Preparation of FDA Meeting Materials
- Program Oversight
- Regulatory Interactions with FDA
- Regulatory Strategy, Writing and Submissions
- Strategic Advice
Visit our services page to see our full range of services and to learn more about what our drug development consultants can offer your program.
Orphan Disease Indications
Nuventra has experience with a number of Orphan Disease indications, including:
- Allergy/Immune Conditions
- Eosinophilic Esophagitis
- Diffuse Large B Cell Lymphoma
- Pancreatic Cancer
- Genetic Disorders
- Duchenne Muscular Dystrophy
- Gaucher’s Disease
- Sickle Cell Disease
- X-linked Adrenoleukodystrophy
- Hereditary Inclusion Body Myopathy
- Hypereosinophilic Syndrome
- Lysosomal and Glycogen Storage Disorders
- Fabry Disease
- Metabolic and Endocrine Disorders
- Cushing’s Syndrome
- Brittle Type 1 Diabetes
- Niemann Pick Type C
- Nonalcoholic Fatty Liver Disease (NASH)
- Viral, Bacterial, And Parasitic Indications
- Recurrent C. difficile