The initiative could change the lives of people with rare diseases by giving them access to cutting-edge treatments
The NHS has announced a new ‘Innovative Medicines Fund’ to fast-track potentially lifesaving new drugs to patients who need them.
Following the model of the existing Cancer Drugs Fund, the Innovative Medicines Fund will allow patients access to drugs that have yet to be approved as cost-effective by the National Institutes of Health and Care excellence (NICE). The new initiative will include treatments for rare and genetic diseases.
“We want NHS patients to continue to be the first in the world to benefit from cutting-edge treatments,” said Health and Social Care secretary, Sajid Javid. “This vital new initiative extends the successful Cancer Drugs Fund model to other patients, bringing equal access to the best treatments for all, regardless of the condition.”
The Cancer Drugs Fund
The current Cancer Drugs Fund (CDF) was established in 2016 and offers patients the opportunity to access treatments not approved for routine NHS use that could help save or extend their lives. Over the last five years, over 64,000 people have accessed treatments through the fund that would otherwise not have been available for months or years.
The fund works by offering NICE a third option when it evaluates a treatment: as well as recommending a drug for routine commissioning or not, it can also recommend a drug for use within the CDF.
For a drug to qualify for the CDF, it must have “plausible potential […] to satisfy the criteria for routine commissioning.”
Rare diseases are – as the name suggests – individually rare, but when looked at collectively they are common. Around 1-in-17 people in the UK will be affected by a rare disease at some point in their life. The majority of rare diseases have a genomic basis, and they can affect people of all ages including young children.
Approving treatments for these conditions can be challenging, however. As the number of patients affected by a single rare disease is small, it is often difficult to gather enough evidence for NICE to recommend it for routine NHS use.
The new Innovative Medicines Fund offers a potential solution to this problem, giving patients the opportunity to have promising treatments fast-tracked, and, in turn, increasing the amount of data available to NICE for future decisions.
“In the last year, NHS England has successfully negotiated deals for a range of new treatments, including drugs which may allow toddlers with spinal muscular atrophy the chance to walk thanks to the ‘world’s most expensive drug’, as well as giving cystic fibrosis patients the latest medicines against their debilitating disease,” said NHS chief executive, Sir Simon Stevens.
“This new fund will build on these successes, offering hope to even more patients.”
For more on the Innovative Medicines Fund, visit the NHS website