A report published by market access consultancy MAP BioPharma is pushing for the National Institute for Health and Care Excellence (NICE) to be more ‘flexible’ with its work with NHS England and charities for rare diseases – deeming assessment methods to be ‘inappropriate’.

The report, titled “Access to Orphan Medicines: A Case for Change” ultimately highlights that NICE’s Single Technology Appraisal (STA) does not deliver equal access for patients.

The European Medicines Agency (EMA) defines a ‘rare disease’ as one that affects less than 5 in 10,000 people. Today there are estimated to be around 7,000 rare diseases affecting 3.5m people in the U.K. with 80% of the diseases being genetic.

MAP BioPharma clearly underlines the point that of 24 completed STA reviews of rare disease medications between 2013 and 2017, hardly any (13%) were recommended for the full eligible population. Additionally during this time-frame 50% of rare disease medications were limited or given ‘restricted recommendation’ compared to 21% medications combatting non-rare diseases.

NICE
National Institute for Health and Care Excellence

NICE’s Highly Specialised Technology (HST) appraisal has led to numerous innovative pricing and reimbursement deals for orphan and ultra-orphan drugs – however most rare diseases cannot be assessed using this pathway and instead must be examined under STA.

One of the most prolific examples of this inequality is Biogen’s Spinraza (nusinersen) – a first-in-class treatment for Spinal Muscular Atrophy (SMA) – the drug was approved in Europe in 2017, but is still not available to patients on the NHS in England.

The drug was in fact rejected by NICE last year on the grounds of its high cost – £450,000 for first year and then £225,000 subsequently. The rejection came despite solid evidence of its efficacy in many patients.

The report itself goes on to say clearly that STA is not fit-for-purpose in the department of rare diseases and suggests manoeuvring to similar arrangements held in Scotland and Wales.

MAP Biopharma CEO, Christian Hill, said: “MAP’s research illustrates that the current processes are not fit for purpose and we urge NICE, NHS England and the Government to work with us and the rare disease community to review how they can improve patient access to rare disease medicines, and give careful consideration to the case for change and recommendations set out in the report.”