UK Politicians Want NICE To Use Conditional Reimbursement
Executive Summary
A UK parliamentary report has called for the HTA body NICE to consider the use of conditional reimbursement recommendations and more flexible cost-effectiveness thresholds in its ongoing methods review.
NICE, the health technology appraisal institute for England and Wales, should consider the greater use of conditional reimbursement and more flexibility when appraising medicines for severe disease and unmet needs, says a new report from the UK’s All-Party Parliamentary Group on Access to Medicines and Medical Devices.
The report, published on September 13, sets out what the group thinks should be included in NICE’s methods review. The review is as part of the 2019 Voluntary Pricing and Access Scheme, which replaced the previous Pharmaceutical Pricing Regulation Scheme (PPRS) at the beginning of this year.
NICE told the Pink Sheet that it is considering the report’s recommendations, which also call for greater patient involvement in its processes and reforms to the highly specialized technologies (HST) appraisal process.
The review was considered necessary because of changes in the types of medicines being developed and because of their potential to deliver benefits over a longer time line. Products such as gene therapies bring new challenges to HTA bodies because they promise to be curative but the data to support such claims are limited.
To compile its recommendations, the APPG collected evidence from more than 50 individuals and organizations. Of 53 written submissions, 26 came from industry and four from trade associations or industry groups. 17 came from patient organizations, four from think tanks and two from clinicians.
Recommendations
The biggest issue with NICE’s methodologies that respondents raised was uncertainty surrounding cost-effectiveness, says the report. To remedy the problem, many submissions called for an “interim conditional approval mechanism” that would function in a similar way to the Cancer Drugs Fund for “plausibly” cost-effective drugs.
The APPG backs such a mechanism because of the success of the CDF, which it says has broad stakeholder support. The mechanism would be triggered for drugs that were plausibly cost-effective, when NICE identified areas of uncertainty, and if modelled scenarios and sensitivity analyses showed that the ICER (incremental cost-effectiveness ratio) would not exceed the cost-effectiveness ceiling. The company would have to collect data for a set period of time and then resubmit the product for appraisal.
QALYs
There should also be more flexibility when it comes to QALY (quality adjusted life year) modifiers. The report recommends that NICE should expand the current end-of-life ICER threshold so it applies to treatments that address a clear unmet need in a severe condition. The cost-effectiveness thresholds NICE considers reasonable generally fall between £20,000-30,000 per QALY. Higher thresholds of up to £50,000 per QALY can be considered for end-of-life drugs if certain criteria are met.
“This will add much needed flexibility to the system, and will primarily benefit products that have been developed to treat severe diseases, which are often rare,” says the report. However, orphan designation alone does not warrant a QALY modifier, it adds.
Patients
Meanwhile, the APPG suggests that NICE should find a better way to incorporate the patient voice into the appraisal process. Taking part in committee meetings can be intimidating for patients, particularly if they have not experienced a NICE appraisal before and are unfamiliar with the formal setting and technical language, says the report.
NICE should look to Scotland where the Scottish Medicines Consortium uses the Patient and Clinician Engagement (PACE) process to gather input from patients in HTAs, it says. Under this process patients and clinicians meet to develop a summary statement on a condition and possible treatments. A similar process could be introduced to NICE’s STA (single technology appraisal) process for orphan treatments when there is a clear unmet need.
The report also suggests how NICE could rethink the way products are referred to the HST process. The institute has already said that it will be looking at the criteria used for admitting products to this program. (Also see "UK NICE Review Will Look At Criteria For Appraising Highly Specialized Products" - Pink Sheet, 8 Apr, 2019.)
According to the report, parts of the routing criteria for referring a product to the HST process are unclear and too stringent, and this has led to specialized treatments for very small patient populations being referred to the STA process instead.
The STA “was not set up to assess ultra-orphan conditions and therefore struggles to take into account characteristics of ultra-orphan treatments, with patient populations of around 500 or fewer in England,” the report explains.
Under one requirement, described as “misguided” by several respondents, a product must be delivered through a highly specialized service to qualify for an HST evaluation. The report recommends this requirement be removed and says NICE could also clearly state patient number thresholds for the HST process. In addition, the report recommends there should be a right of appeal if a treatment is rejected on budget impact grounds.
Other recommendations include changes to the way NICE approaches discounting. This refers to the way economists calculate and value a product’s benefits and costs that might occur in the future. In the public sector, it means that costs and benefits with different time spans can be compared on a common present value basis.
The report recommends that NICE should use the discount rate of 1.5% as set out in the Treasury’s Green Book. This is guidance issued by the treasury on how to appraise policies, programs and projects and applies to all proposals that involve public spending. NICE currently uses a discount rate of 3.5%.
“Greater use of the 1.5% rate would fairly place value on long term effects on length and quality of life in the context of our healthcare system, as in other parts of society, such as road traffic accidents,” says the report.
The APPG would also like to see NICE take a clearer and more transparent approach to publishing the timelines and deliverables for the review.
And to improve transparency, NICE should publish a bi-annual breakdown of its recommendation rates for each pathway, including how many products have been recommended within their marketing authorization.
NICE response
NICE said it was taking stock of the report. “We are currently considering the suggestions from the All-Party Parliamentary Group on Access to Medicines and Medical Devices as to the topics for inclusion in NICE’s review of its methods and processes, and will respond to the chair of that group in due course,” a spokesperson said.
Following engagement with stakeholders, the institute has already put together a short list of topics to be considered in the review. “For each, a case for change will be explored, and proposals presented for public consultation in summer of 2020,” it noted.
The topics for consideration include the modifiers used in decision making and how to quantify and reduce uncertainty, for example in the evidence that is available or in the assumptions that are made in economic modeling.
Other topics to be considered are incorporating quality of life into economic analyses, and technology specific issues such as the challenge of evaluating treatments that target tumors according to their genetic make-up rather than where they originate in the body.
The Ethical Medicines Industry Group believes the review is unlikely to be far reaching and has called for a wider discussion on the problems companies face when bringing medicines to the UK market. (Also see "UK NICE Review Needs To Be More Than A ‘Bureaucratic Exercise’" - Pink Sheet, 20 Jun, 2019.) The group represents companies, patient organizations and academics from the life sciences sector.