UK NICE Processes Not Fit For Orphan Drugs, Says New Report
Executive Summary
UK health technology appraisal body NICE says it will carefully review a new report that has found its processes block access to orphan drugs.
Processes used in England by health technology appraisal body NICE to evaluate rare disease treatments are not fit for purpose and should be reformed, claims a new report from the market access consulting firm MAP Biopharma.
The report, which involved input by several pharma companies, suggests five “flexibilities” including interim funding recommendations and more room to consider higher cost-effectiveness thresholds, which would deliver a faster and fairer system.
The report’s authors are urging NICE, England’s National Health Service and the government to work together with the rare disease community to improve access to rare disease treatments and consider the recommendations outlined. They say that the need to do so is ever more pressing as NICE is set to take on a greater work load, in line with the latest pharmaceutical pricing agreement agreed between NHS England, the government and the pharmaceutical industry. Under the revised PPRS, NICE will evaluate all new medicines. (Also see "New UK Price Deal To Save NHS £930m & Speed Up Access To Innovation" - Pink Sheet, 26 Nov, 2018.)
"We will... further explore the report’s conclusions ahead of our own forthcoming review of our methods for evaluating new treatments" – NICE
It looks as if NICE could consider the recommendations through an upcoming review of the way it evaluates medicines. A NICE spokesperson said the institute also wants to ensure that clinically and cost-effective treatments in areas of high unmet need reach the patients 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,” the spokesperson said. The institute told the Pink Sheet that this review is in its early phases but was unable to give further details about any time lines.
STA Unfit For Orphans
According to the report, the Single Technology Appraisal process, which NICE uses to evaluate the majority of new medicines that it looks at, has become the “default referral route” for most orphan drugs, rather than the Highly Specialised Technology program.
The HST is better able to accommodate rare disease treatments as the allowable incremental cost-effectiveness ratios (ICERs) are higher, ranging between £100,000 and £300,000 depending on the magnitude of benefit. In addition, the HST program applies a broader range of decision-making criteria, for example greater consideration of benefits beyond health care. Meanwhile, the maximum ICER for the STA is usually around £30,000, or £50,000 for end-of-life treatments and there are no further adjustments for orphan drugs under the STA. “The STA criteria fail to take into account the nature of rare disease medicines both in terms of the evidence base, unmet need and cost,” says MAP.
Nevertheless, the majority of orphan drugs do not qualify for the HST program and go through the STA process, putting them at a disadvantage, the report says. Of 24 STA reviews of rare disease treatments between 2013 and 2017, only 13% won recommendations for their full eligible population, in line with their marketing authorization. In comparison, more than two thirds of other medicines received a full recommendation.
In addition, the report highlights that none of the non-oncology rare disease medicines reviewed under the HST were recommended in line with the full marketing authorization. It also points out that more rare cancer treatments going through the STA were able to benefit from the Cancer Drugs Fund than other cancer medicines. “This demonstrates how rare disease medicines are more likely to require special considerations in their appraisals,” says MAP. Through the CDF, treatments can receive interim recommendations for funding while more evidence is gathered to support routine uptake.
The report says that the number of orphan products being authorized is growing and this will only add pressure to the system. Health systems around the world are tackling the issue, and “as a global leader in health technology assessment, NICE should be facing this challenge head-on,” it adds. The report notes that NICE’s counterparts in Scotland and Wales, the Scottish Medicines Consortium and the All Wales Medicines Strategy Group, have modified their systems for dealing with orphan drugs.
According to the report, the forthcoming review of NICE’s STA methods provides an opportunity to remedy the situation and consider the following five flexibilities:
- Introduce formal changes to STA evidence requirements for orphan drugs. Flexibilities introduced should be informed by stakeholders who have experience in developing or assessing orphan medicines.
- Consider the introduction of a sliding ICER scale for orphan medicines up to £100,000, drawing from HST methodology.
- Consider adapting the Evidence Review Group brief for orphan treatments within the STA program, and explore a role for the Rare Disease Advisory Group when orphan medicines are appraised by the STA process.
- Provide opportunities for negotiations between companies and NHS England for orphans assessed within the STA work program.
- Consider introducing an interim recommendation for orphan drugs, in line with such recommendations that can be made through the CDF and from the SMC to support real-world evaluation of impact of treatments.
Background To The Report
Many of MAP’s clients are small companies with a particular focus on orphan medicines (rare disease medicines). Based on short-term research projects, and anecdotal evidence and feedback from patient groups, clinicians and companies, MAP convened a Steering Group of MAP Online members to review the data and policy environment for orphan medicines, and to make recommendations for improvement. The new report sets out the group’s findings and recommendations.
The Steering Group members are Amicus Therapeutics, AveXis, bluebird bio, Chiesi, Gilead Sciences, Kyowa Kirin International and Santhera Pharmaceuticals. Each company made an equal financial contribution to support the work involved with the report.
From the editors of Scrip Regulatory Affairs.