NICE stands for the National Institute for Health and Care Excellence in England. This organization is responsible for evaluating the cost effectiveness of all medications as they come to the market.
MAP BioPharma has recently completed a report which analyzes NICE’s approach regarding medications for rare disease. They conclude the organization’s appraisal policies are inflexible and inappropriate for the rare population. This is demonstrated by the extreme delays that rare patients can experience when trying to access the medications they need. In some cases, they aren’t able to obtain them at all.
MAP urges NICE to engage with not only their own organization, but also rare disease charities, the English government, and the National Health Service of England in order to consider changing their approach.
As part of their recommendation, MAP suggests the adoption of five different “flexibilities” which address data requirements, value assessments, and other factors which could ultimately provide rare patients easier access to treatment.
Single Technology Appraisal (STA)
The STA process is utilized by NICE when the disease does not qualify for their Highly Specialized Technology appraisal. Most rare diseases fall into this category. According to MAP’s report, this approach is inflexible and inappropriate for these conditions.
One of the rare disease treatments negatively impacted by this form of appraisal was Spinraza for the treatment of spinal muscular atrophy (SMA). Spinraza has been proven to have life-altering affects for some patients with SMA, allowing patients to live a practically normal life. This treatment was approved in Europe in June of 2017. However, NICE rejected the treatment, stopping it from being available within England’s National Health System, in August of 2018 because they said it was too expensive. As a result, there are SMA patients in England who are being kept from a treatment which could essentially eliminate their symptoms.
Contrarily, Spinraza was approved in Scotland as an accessible medication in their National Health System.
Ultimately, MAP’s report shows that STA criteria do not properly take into account unmet need, evidence, or cost for rare diseases.
Between the years of 2013 and 2017, based on 24 STA reviews MAP found:
- 50% of medications for rare diseases were offered “restricted recommendation”
- 21% of medications for non-rare diseases were offered “restricted recommendation”
- 13% of medications for rare diseases were recommended for the full eligible patient population
- Over 2/3rds of medications for non-rare diseases were recommended for the full eligible patient population
Endorsement by the Genetic Alliance UK
The Genetic Alliance UK is an organization composed of 220 patient charities in the UK. They have endorsed the study conducted by MAP, saying that it aids in illuminating the systemic failings of the NICE system. Genetic Alliance UK further explains that these failings for rare disease patients will continue to occur until they are specifically addressed.
They promise that they will ensure the rare patient voice is included in the changes made in England’s policy.
NICE’s Response
Thankfully, NICE’s response to this report has been positive. They have already planned a meeting with the authors of the MAP study in hopes to discuss its findings more in depth. Following this meeting, they plan to review their methods of evaluating new rare disease treatments.
A NICE spokesperson has stated that the organization shares MAP’s ambition for ensuring clinically effective and cost effective treatments reach the patients who so desperately need them.
Hopefully, this response means that we will begin to see reform soon.
You can read more about this evaluation of NICE policies here.
