Rare disease research and orphan drug development needs to start and end with the patients with unmet medical need. Arguably more than any other sector of the healthcare industry, the rare disease patient group voice is individually loud and collectively legislation changing.
As many of the most successful orphan drug developers will say, the patient organisations and rare disease sufferers play an integral role in ensuring the successful development, approval and reimbursement of an orphan drug. In fact, some rare diseases patient organisations with exceptionally strong resources and funding are now taking it upon themselves to drive research and discover treatments for their rare disease.
In the following video, Sue Millman, CEO, Ataxia UK outlines how patient organisations can assist in raising disease awareness at a national level, influence healthcare authorities when it comes to reimbursement and also how patients themselves view the premium prices of orphan drugs and the struggle to gain access to potentially life changing treatments.
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If you're looking to move into the orphan drug market, why not see if your candidate is right for orphan drug designation?
Or if you're well into orphan drug development, take a look at the challenges companies are facing in late stage development