Using the UK’s Innovative Medicines Fund to Accelerate Rare Disease Drug Development and Access

January 27, 2023

The UK’s Innovative Medicines Fund (IMF) was started in collaboration with the NHS England and National Institute for Health and Care Excellence (NICE) to provide novel medicines with unresolved uncertainties to patients with rare non-cancer diseases that have little treatment options. In doing so, the program is intended to incentivized development of drugs for rare diseases, or orphan drugs.

According to PharmaPhorum, “The IMF is a new opportunity for patients outside of the oncology space to access treatments when the evidence base is uncertain, if these treatments have the plausible potential to be cost-effective in time. It builds on the Cancer Drugs Fund (CDF), an early access mechanism for many cancer medicines in England. The opportunity is welcomed by the rare and ultra-rare disease community as their situation remains difficult. A rare disease is defined in the UK as a condition affecting less than 1 in 2000 people, but collectively, rare diseases are not rare. One in 17 people is impacted by a rare disease, yet around 95% of the between 7,000 and 10,000 rare diseases have no effective treatment. Even when treatments are developed, they come with high uncertainty.”

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