New joint NICE/NHS proposals look to significantly change future appraisal and funding of treatments

By: Mapi’s Health Technology Assessment, Strategy & Communication team, Real World Strategy and Analytics group

The National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) England proposed changing the way treatments are appraised in joint proposals released on the 13th October 2016, with a particular focus on high value treatments, treatments with potentially high budget impact, and highly specialised technologies (HSTs). According to Sir Andrew Dillon, NICE’s Chief Executive, “By further streamlining our processes we will ensure treatments that clearly offer exceptional value for money will be available to the patients who need them faster than ever before.”

Given recent concerns regarding access to new treatments for patients in the UK, these new proposals may speed patient access to high value treatments. Mapi welcome any changes to the appraisal processes that will increase the timeliness of patient access to a broader range of new and effective treatments. However, there is a lack of clarity in the methodologies utilised to determine the potential new thresholds, as well as uncertainty in how some of the proposed changes widen patient access to new, cost effective treatments. Mapi look forward to obtaining further clarity on these proposals during the consultation period and to understand how these changes may impact global HTA processes.

The three proposals described in the NICE consultation document focus on the implementation of a budget impact threshold, potential fast track review for highly cost-effective treatments, and utilisation of a cost-effectiveness threshold for HSTs.

The first proposal focuses on treatments which may have a budget impact of £20 million or more, in a manner similar to other European HTA bodies. Treatments recommended by NICE under their current appraisal process that are expected to have a high budget impact will require commercial agreements between the manufacturer and NHS England. In cases where NHS England and the manufacturer are unable to come to an agreement, NHS England may ask NICE to extend the period of phased introduction beyond the standard 90 days to lessen the budget impact. NICE and NHS England expect that less than 20% of treatments will exceed the proposed threshold. However, in recent years a number of treatments have implemented patient access schemes in order to satisfy cost-effectiveness requirements and obtain NICE approval. As such, Mapi expects that a higher proportion of appraised treatments than that indicated by NICE and NHS England are likely to exceed the proposed budget impact threshold and will require special commercial agreements with NHS England.

The second proposal unveiled plans to introduce a “fast-track” option for technologies found to be highly cost-effective. According to the NICE press release, treatments costing less than £10,000 per quality-adjusted life-year (QALY) “with a high degree of certainty” would be assessed under a “lighter touch” process. Treatments falling below the proposed threshold and those with a budget impact of less than £20 million will be made available to patients within 30 days of publication of the final NICE guidance. The “fast-track” option is designed to streamline the assessment process for highly cost-effective drugs with strong evidence and a low degree of decision uncertainty, in order to quickly allow patient access to treatments that offer exceptional value for money. However, the proposal does not provide additional clarity regarding how a “reasonable degree of uncertainty” will be defined.

The third and final proposal was specific to HSTs indicated for the treatment of patients with very rare diseases. The proposal would implement a cost-effectiveness threshold of £100,000 per QALY for those treatments assessed under the HST programme. Treatments exceeding the cost per QALY threshold would have another opportunity to be considered through the NHS England’s process for prioritising other HSTs. The programme currently does not have a cost-effectiveness threshold due to limited availability of data associated with very rare disease and small patient populations. Currently, HSTs are assessed under a cost-consequence analysis which considers other aspects beyond cost-effectiveness. It will be interesting to see if the interim methods guide changes to adopt a full cost-utility analysis as part of the evidence for submission in the HST process.

Mapi look forward to obtaining greater clarity on the proposed changes presented in the NICE consultation document. Persons or organizations wishing to share their views on the proposed changes have until 13th January 2017 to provide comment to NICE through the formal consultation process. Additional information regarding the formal consultation process and upcoming NICE events to discuss the proposals can be found here.

Mapi consider that these proposed changes highlight the importance of seeking early engagement with HTA and regulatory bodies, such as NICE and the Medicines and Healthcare products Regulatory Agency, to navigate the appraisal processes, manage uncertainty with appropriate evidence, and optimise patient access. To obtain insight into how these proposals will impact the regulatory approval and reimbursement appraisal of your products, contact Mapi’s HTA experts.