UK Faces Barriers to Accessing Rare Disease Medications, Survey Reveals

The survey highlights pharmaceutical companies' concerns over UK reimbursement standards, limiting access to rare disease treatments.

According to a survey conducted by the Association of the British Pharmaceutical Industry (ABPI) and the Bioindustry Association (BIA) on Rare Disease Day (February 28), most pharmaceutical companies developing treatments for rare diseases do not expect their products to reach UK patients. Less than 75% of the pipeline is expected to be introduced in the UK within the next five years, according to 11 of the 18 businesses that replied to the survey.

Rare illness specialist Sarepta Therapeutics has revealed that it has four clinical candidates and more in the discovery and preclinical stages. However, because not all programs are made public, it is more difficult to determine the number of candidates in a large pharmaceutical corporation.

Pharmaceutical companies' lack of trust in a UK launch is attributed to financial concerns, according to ABPI. First, the National Institute for Health and Care Excellence (NICE) is unlikely to make a favorable reimbursement judgment. Due to the tiny target patient population, rare disease treatments are typically more costly than their counterparts in non-rare diseases. According to research, NICE's current appraisal model for innovative technologies disadvantages orphan goods for uncommon diseases.

Additionally, pharmaceutical companies are discouraged by the UK's "increasingly unfavorable commercial environment," according to ABPI. The current rate of medicine discounts, which is the proportion of sales that pharmaceutical firms must return to the NHS if sales surpass a predetermined threshold, stands at 22.9%. The ABPI reports that rates are currently "rocketing and unpredictable," with an average of roughly 6.8% from 2014 to 2022.

According to the results of the ABPI survey, 16 of the 64 rare disease medications that were granted licenses in the UK were not submitted to NICE for review. As a result, only one out of every 20 disorders has an NHS-approved treatment or medication, despite the fact that there are over 7,000 identified uncommon conditions.