OBJECTIVES:

To consider the eligibility of orphan medicinal products (OMPs) for either the National Institute for Health and Care Excellence (NICE) highly specialised technology evaluation (HST) programme or single technology appraisal (STA), and the potential impact a referral to either programme may have on patient access to new medicines.

METHODS:

MAP reviewed all 46 treatments designated as OMPs by the European Medicines Agency (EMA) since 2015 to ascertain what proportion have been/will be assessed by either NICE’s HST or STA programmes. MAP also surveyed its members to understand what impact they envisage an inappropriate STA referral would have on their company and access to new treatments for patients with rare diseases.

RESULTS:

Since 2015, only 7 OMPs have been referred for HST indicating that the majority of OMPs are at risk of an unsuitable referral for STA. The HST review has much greater flexibility than the current STA process in terms of evidence requirements and a variable cost-effectiveness threshold of between £100,000 to £300,000 per quality-adjusted life-year (QALY). Non-cancer OMPs are subject to a maximum cost per QALY of £30,000 which does not take into account the development costs and evidence profile of the majority of OMPs. Unsurprisingly, MAP members are concerned about the impact on patient access and are considering their future launches in England as a result.

CONCLUSIONS:

MAP’s previous research has underlined the increasing number of OMPs and the impact that these are likely to have on health systems. Given the number of OMPs that do not meet NICE’s HST criteria, NICE will need to adapt its STA process in order to assess all orphan treatments appropriately. Failure to do so may act as a disincentive for manufacturers who may choose to deprioritise product launches in England, particularly in the context of Brexit.