UK patients living with rare diseases are not being offered many of the life-changing medicines being developed, mainly due to poor reimbursement criteria and weaker commercial opportunities.
A survey, published by the Association of the British Pharmaceutical Industry (ABPI) and the Bioindustry Association (BIA) on Rare Disease Day (28 February), found that most pharmaceutical companies developing medicines for rare diseases do not expect their treatments to reach UK patients.
Companies were asked last year to consider their pipeline rare disease assets with global launches within five years and assess how many would make it to the UK – a region where 3.5 million people live with a rare disease. From the 18 respondents in the survey, 11 companies said that less than 75% of their pipeline is expected to launch in the UK over the next five years.
The number of rare disease drugs in a company’s pipeline varies and may not always be publicly reported. Sarepta Therapeutics, a company specialising in rare diseases, has disclosed it has four candidates in the clinic with more at the discovery and preclinical stage. However, it is harder to ascertain the number of candidates in a big pharma company as not all programmes are publicly disclosed.
According to ABPI, pharmaceutical companies’ low confidence in a UK launch stems from financial considerations. First, there is a low likelihood of a positive reimbursement decision from the National Institute for Health and Care Excellence (NICE). Treatments for rare diseases are generally more expensive than non-rare disease counterparts due to the small target patient population. Research has shown that orphan products for rare diseases are disadvantaged by NICE’s current appraisal model for new technologies.
There is also the UK’s “increasingly unfavourable commercial environment”, as per ABPI, that puts off pharmaceutical companies. Rebate rates for medicines, the percentage of sales that drug developers are required to pay back to the NHS if sales exceed a pre-set target, are currently at 22.9%. The average between 2014 and 2022 was around 6.8%, with ABPI saying that rates are now “rocketing and unpredictable”.
ABPI survey results indicate the knock-on effect – 16 out of 64 rare disease medicines approved for license in the UK were not submitted to NICE for evaluation.
The result is that whilst there are more than 7,000 recognised rare conditions, only one out of 20 diseases has an approved NHS treatment or medicine.
Call to arms
The ABPI called for a greater ambition to make it easier to develop and launch medicines in the UK by making the region “a more attractive place”.
The UK Government’s Rare Disease Action Plan 2024 highlighted the ongoing progress to advance the region’s rare disease capabilities, including a £14m investment into research platforms over the next five years.
The document also details a framework comprising national priorities to raise awareness and care, increase diagnosis, and improve access to treatment and drugs. Initiatives by the government, however, have been far and few between, with researchers calling for better inclusion of rare disease strategies in public health strategies.
Although welcoming the latest action plan, ABPI chief executive Dr Richard Torbett said: “Unless we address the UK’s historic and ongoing disinvestment in medicines, we will continue to see NHS patients with rare conditions missing out on the latest innovations.
“This means fixing the current skyrocketing repayment rates under the Voluntary Scheme for branded medicines and making sure that the way NICE evaluates medicines for rare diseases does not prevent companies from bringing them to the UK.”
