NICE accused of inflexible, inappropriate assessment methods

MAP BioPharma have published a report suggesting that when it comes to appraisals of rare disease drugs, NICE’s assessment methods are so “inflexible” and “inappropriate” that it leads to access to new medicines being delayed or completely prevented. The report offers recommendation to create a better system with faster outcomes. NICE use a standard Single Technology Appraisal process to assess rare diseases, rather than its Highly Specialised Technology appraisal, which has resulted in several innovative pricing and reimbursement deals closed to bring orphan and ultra-orphan drugs to NHS patients. According to the report, in the period from 2013 to 2017 just 13% of the 24 completed STA reviews of rare disease medicines were recommended for the full eligible population compared to a full recommendation for more 66% of other medicines, and 50% of rare disease medicines were given a restricted recommendation versus only 21% of non-rare disease medicines.

Biogen’s Spinraza (nusinersen) for Spinal Muscular Atrophy, is a prime example. Europe approved it in June 2017 yet it is still not available on the NHS in England. NICE said it was too expensive at £450k for the first year of treatment and £225k for each year thereafter, even though there is robust evidence of its effectiveness in some patients.

Christian Hill, CEO of MAP BioPharma, 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.”

CEO of Genetic Alliance UK, Jayne Spink, said: “This report helps to show the systemic failings in our decision-making systems for access to rare disease treatments. This system will continue to fail people living with rare diseases until the fundamental challenges of assessing rare disease treatments are better addressed. We at Genetic Alliance UK, our 220 patient organisation members, and our community look forward to ensuring patient voice plays a crucial role in making positive changes.”

A NICE spokesperson said: “We share its (the report’s) ambition to ensure innovative treatments in areas of high unmet need that are clinically and cost-effective can reach the people who need them. We will review the report’s recommendations carefully and to that end we will be meeting with its authors to further explore the report’s conclusions ahead of our own forthcoming review of our methods for evaluating new treatments.”