Posted by Michael Wonder on 12 Apr 2017
Why drug appraisal methods need to take into account treatments for rare diseases
12 April 2017 - The UK government needs to adapt its drug and technology appraisal methods to prevent patients with rare diseases being left out in the cold.
Patients with a rare disease continue to face unmet need in the UK. Only 5% of rare diseases have a licensed treatment option and one in three children with a rare disease will die before the age of 5 years.
Rare diseases are still being discovered, so finding innovative medicines for these conditions is increasingly important. However, small patient populations present unique challenges in the research, development, and reimbursement of these medicines.
