NICE's STA appraisal process changes

Recent changes to NICE’s STA appraisal process and the CDF


On 26th June 2018, Remap Consulting had the pleasure of attending the annual National Institute of Health and Care Excellence (NICE) conference. The event provided a great opportunity to network with NICE and industry and to discuss recent updates to NICE processes, including the new Cancer Drugs Fund (CDF) and the new Single Technology Appraisal (STA) process.

Two-years into the new CDF: what are the learnings?

In July 2016, NICE launched the new Cancer Drugs Fund (CDF), which allows one of three recommendations for oncology products; a positive recommendation, a negative recommendation, or a recommendation for use within the CDF. Products which are recommended within the CDF are subject to a managed access agreement (MAA), which consists of three components:

  1. Data collection agreements either in the form of clinical trials run by the manufacturer or through the use of real-world evidence (for instance through the use of Public Health England (PHE) data sources). Agreements for data collection are made between the manufacturer, NHS England, PHE and NICE
  2. CDF commercial agreements to determine the level of reimbursement during the MAA period. These are confidential between NHS England and the manufacturer, and are agreed with input from NICE
  3. Expenditure control mechanisms. The new CDF has an annual fixed budget set at £340m. In order to ensure that the budget is maintained, expenditure control mechanisms have been put in place. This means that in the case of the CDF overcommitting at the end of the financial year, manufacturers that have been funded through the CDF budget will be required to pay a rebate to NHS England

CDF MAAs are generally in place for a maximum of two years, after which a NICE re-submission must be made. At re-submission, drugs may either be recommended for baseline funding or not recommended for use. NICE re-submissions involve the calculation of a new incremental cost-effectiveness ratio (considering the additional data), which will influence the final accepted price.

Since launch of the new process, 18 drugs have been recommended for use within the CDF and formed MAAs with NHS England. The vast majority of these (14/18), are utilising clinical trials to collect further data, whilst the remaining four are utilising real-world data collection strategies, managed by PHE. In terms of budget control, to date the new CDF budget threshold has not been reached and as such, expenditure control mechanisms have not been triggered.

First hand insights into the CDF were shared by Takeda, with their experience for Brentuximab. The drug in question transitioned to the new CDF and was one of the first drugs to be recommended for use within the new fund. In terms of data collection, a retrospective survey sent to treating physicians by PHE was used. Takeda mentioned that PHE achieved an 80% response rate and that data collection such as this would not have been possible for a manufacturer alone.

Overall, Takeda gave a positive impression of their experience with the new CDF process. However, they did note that the NICE re-submission has the same requirements as the original submission, resulting in repetition and greater time and resource requirements. In addition, it should be noted that following the recommendation for use within the CDF, it is generally a period of 3 to 4 months before the terms of the MAA are finalised. It was also stressed that manufacturers who believe they may have data gaps and could be eligible for a CDF MAA, should discuss this with NICE as early as possible.

An updated approach to STAs for 2018

NICE has been looking into making changes to the STA process to combat two key challenges facing NICE:

  • The increasing demand for appraisals. NICE could previously be expected to conduct ~20-30 STAs per year. However, the growing number of innovative and expensive technologies launching, in addition to indication expansions, has led to demand for ~70 STAs per year
  • Discrepancies between draft and final recommendations. Currently, 80% of Final Appraisal Determinations (FADs) are positive recommendations. However, 60% of technologies receive negative draft recommendations at the Appraisal Consultation Document (ACD) stage. This discrepancy suggests that manufacturers are not sufficiently addressing NICE requirements at initial submission

NICE has chosen to introduce a new appraisal process with the aim of increasing efficiency and addressing these challenges. The new approach, introduced in April 2018, intends to shorten the appraisal process by allowing only one appraisal consultation meeting rather than two. In addition, the new process has introduced a technical engagement phase, which gives consultees one month to submit comments on the submission, which can then be addressed by the manufacturer.

It is hoped that shortening the appraisal process will enable NICE to more efficiently conduct appraisals and therefore have greater capacity to complete the growing number of STAs. The technical engagement phase aims to provide manufacturers with comments early in the review process, allowing time for these to be addressed and potentially reducing the percentage of negative draft recommendations. Furthermore, the new shortened appraisal process intends to better align with regulatory timelines to enable drugs to be made available sooner after market authorisation.

As yet, all products currently under review are not utilising the new appraisal process. Instead, NICE have identified a list of products who would be expected to undergo consultation meetings in January 2019 and have initiated the new process only for these drugs. The first drug facing this new approach is cabozantinib (TA10287).

Future implications

Overall, in recent years NICE and NHS England have taken a number of steps to address challenges in managing the increasing demand for appraisals and funding, including launching the new CDF and STA process.

The recent changes to the CDF appears to be positive, and has provided several drugs (which may have been previously been rejected due to data-gaps) the opportunity to gain patient access. In addition, the new CDF has managed to provide this funding whilst remaining under-budget. The new fund does present its own challenges for manufacturers, in terms of time taken to agree a MAA and the resource requirement for resubmission. Nevertheless, the opportunity created by the CDF is likely to be attractive to manufacturers and it seems reasonable to assume that it will be a route pursued by a growing number of companies in the future. It will be interesting to see how the CDF budget copes with the increasing number of cancer drugs launching.

The potential impact of the new STA process is less clear. NICE suggest that the approach will be considered a success if there is a reduction in the discrepancies between ACD and FAD decision-making. However, as no drug has completed the new STA process, there is currently a lack of clarity of how valuable the technical engagement stage will be in reducing uncertainty and decreasing negative draft recommendations. Furthermore, NICE aim for the new process to result in a shortened appraisal process and speedier patient access, more closely aligned to market authorisation. However, there are concerns over whether one appraisal meeting will be sufficient for manufacturers. NICE have mentioned that further meetings could potentially be allowed at their discretion. Whilst this would reduce manufacturer uncertainties, it is likely that further meetings would not allow the new shortened timelines to be met. Overall, it is currently unclear whether the steps NICE have taken will be sufficient to address the challenges of a continually increasing demand for appraisals.

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