RARE & ORPHAN DISEASES
SMALL PATIENT POPULATIONS
When developing therapeutics for small patient populations, the right leadership needs to be in place to set your therapies up for success. According to the NIH:
- In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people.
- Other countries have their own official definitions of a rare disease. In the European Union, a disease is defined as rare when it affects fewer than 1 in 2,000 people.
- There may be as many as 7,000 rare diseases. The total number of Americans living with a rare disease is estimated at between 25-30 million.
- Efforts to improve and bring to market treatments for rare diseases are coordinated by the Food and Drug Administration (FDA). The Office of Orphan Products Development (OOPD) provides incentives for drug companies to develop treatments for rare diseases. Between 1973 and 1983, fewer than 10 treatments for rare diseases were approved. Since 1983, the OOPD program has helped develop and bring to market more than 400 drugs and biologic products for rare diseases.
Ashton Tweed’s Life Sciences Talent Bank is ready to provide your company with interim and permanent professionals, creating the flexible workforce that rare disease companies require in order to survive the competitive industry landscape. Together, we can overcome issues such as:
- Funding difficulties resulting from narrow target markets.
- Clinical research and trial obstacles due to small patient populations.
- Regulatory setbacks affecting your critical timelines.
- Trouble connecting with external resources needed for approval.
- Challenges attracting and retaining top specialized talent.
With the patients in mind, we can work with your company towards the same goal of improving their quality of care. Review the case studies below to see how we’ve helped this industry sector in various ways:
A global rare disease company wanted to conduct in-licensing deals in the US to grow their portfolio. To do this, they needed a senior business development professional to evaluate and close both developmental and commercial assets…
A biopharmaceutical company specializing in orphan drugs, was looking to advance their therapies through a fast-track model in order to begin phase III trials. With this ambitious program, they needed help hiring a VP of Worldwide Regulatory Affairs…
An orphan drug company was acquired by two larger companies in quick succession. Due to employee turnover, they had lost a critical member of their due diligence team and decided to bring in a strong interim Chief Medical Officer…
Ashton Tweed has provided talent for multiple rare disease companies and enjoys the challenge of this valuable and rewarding work. Our team understands both the difficulties and the need to select specifically qualified professionals for your open roles. See our services below and speak to a partner to discuss which hiring strategy is best for you!
Ashton Tweed’s executive talent bank is a unique service matching members to companies through retained executive search. This ensures companies and candidates alike work with the best and brightest.
Interim executives are the perfect solution for your company when in need of just-in-time talent to solve critical business needs. Convert fixed hiring costs into a variable expense as your business gains a fresh perspective.
Speak to an Ashton Tweed Partner
Use the button below to discuss your rare disease company’s needs or call 610-725-0290 today!