The National Institute for Health and Care Excellence (NICE) has this week published its updated guide to its health technology appraisal (HTA) process following two rounds of consultations over the last six months. The aim of these changes is to streamline and accelerate the appraisal process. This consultation process has been very low key and has not been publicised. So what do you need to know about the changes to HTAs? And what could it mean for industry, patients groups and others across the health economy?

Summary

  • The timeline for appraisal has changed, with the first appraisal committee likely to be happening earlier in the regulatory process as soon as possible after a positive CMPH opinion, cutting down the time for companies to petition about the benefits of treatment and also potentially limiting opportunity for price negotiations.
  • The new technical committee stage provides an opportunity to deal with the technical issues before a submission reaches the Appraisal Committee, which it is hoped will created efficiencies. It is also hoped this new stage will give companies a chance to engage with the process at an early point.
  • Decisions can now be made after a single Appraisal Committee meeting under the auspices of its Chair if no additional information is submitted after its first meeting. In this case, the Final Appraisal Decision will become NICE’s recommendation.
  • NICE has sought to reassure patient groups, in particular, that they will still be involved in the appraisal process and reiterated that experts will be invited to engage in the process, particularly at the technical committee stage.
  • The time to appeal a negative recommendation has been reduced from 20 to 15 days, meaning submissions will be more straightforward than under the former model.

I want to appraise you like I should: new timelines for the appraisal process

NICE now aims to produce guidance within 90 days of marketing authorisation for all appraised products. However, the timelines between submission and first Appraisal Committee meeting and between invitation to submit and the submission date mean that timelines for NICE have been extended, bringing forward the appraisal itself and placing it earlier in the EMA regulatory process. Indeed, NICE aims to hold ­its first Appraisal Committee meeting as soon as possible after the EMA’s Committee for Medicinal Products for Human Use issues a positive opinion. For those awaiting new, potentially life-saving drugs to be made available, the sooner a successful appraisal can be made the better. But this poses an issue for industry, not least as it cuts down significantly the window available to petition NICE, and policymakers, of the benefits of their new treatment. For example, there may be uncertainty about marketing licences when companies are developing their submissions to NICE and this will also impact on the availability of relevant data to support a submission. The appraisal coming earlier in the process also limits the scope for negotiations surrounding price, with companies being encouraged to put their best price forward right off the bat. Many will welcome a more transparent process, but there are concerns that a hard-nosed approach to negotiation may impact the treatments reaching patients. Companies may need to rethink how they undertake submissions to NICE.

Beware the technocrats?: A new technical committee

Perhaps surprisingly for a re-jig that aims streamline the appraisal process, NICE has introduced a new step to proceedings. A technical ‘consultation’, involving a team of NICE staff and the committee chair, will now take place before the appraisal committee meets to consider the scientific and technical evidence submitted and arrive at preliminary scientific judgements. The resulting technical report will be submitted to the appraisal committee for its consideration.

The Evidence Review Group (ERG) will continue to carry out its existing role. However, under the new system, rather than the ERG’s systematic review being used as evidence during the committee decision meetings, the report (now known as the ‘technical report’) will be opened up to experts, including NHS Commissioners, clinical and patient experts for comment and consultation.

The objective here is to resolve any scientific or technical uncertainties before appraisal stage, which will – it is hoped – increase efficiency. The technical consultation is expected to engage thoroughly with commentators and experts – including patient groups and others – capturing their input much earlier in the overall process. Under the old process, there was little opportunity for committee members and other experts to question or probe the technical aspects of a company’s submission ahead of the committee meeting. This update to the process changes this.

The introduction of a consultative approach to the technical report ought to provide further clarity from the NICE committee when it comes to the evidence and data being presented, allowing for more targeted committee hearings.

Got to be in it to win it: Opportunities for companies to engage with the process

Ensuring external participation in the appraisal process was a key plank of NICE’s ambitions for the updated HTA process, particularly for companies who now have specific times during which they can engage with the process. Feedback from the initial consultation showed strong support for there to be clear, recognisable milestones for companies to engage with, that link to key stages in the regulatory pathway. Representatives who have been involved in developing the company’s evidence submission can now attend part of the Appraisal Committee meeting to discuss their submission. This will be welcome news for companies who now have the opportunity to engage with NICE early on in the appraisal process, giving them more of a chance supplement their written submission and address any concerns the committee may have.

Two become one: more determinations to be made in a single committee meeting

With the updated TA process comes the aim for more ‘final appraisal determinations’ (FADs) to be issued at a single committee meeting rather than two. The Chair has been given greater authority to make a final decision in cases where the company responds to the appraisal consultation with an updated commercial offer only. In such cases, the Chair may decide that a second Formal Appraisal Committee meeting is not required. Any reduction in opportunities for companies to respond during the period between two committee meetings ahead of an FAD being issued will not doubt be a cause for concern for industry.

Turning back time: reducing the appeal process timeline

The updates to the NICE HTA process are all about streamlining and simplifying processes. One update that – on the face of it – appears to be an ‘easy win’ is the reduction in the time frame between the close of appeal and the publication of guidance, down from 20 days to 15. This move allows NICE to further reduce the time taken to produce a decision on a new technology. NICE has come under criticism for its lengthy processes and some therapies getting lost in the malaise of NICE decision-making.

This shortening of the time frame between appeal and publication is one of many areas where NICE is seeking to turn the tide on this particular narrative.

Although this change in process gives companies less time to formulate an appeal to a negative FAD, the argument from NICE will be that other changes further back in the process will provide companies with the abilities to fully formulate and stress-test their position, meaning the detail of an appeal submission will be more straightforward than under the old model. This change again signals the increased importance of companies having a cohesive set of messaging and arguments that can be deployed easily in different scenarios.

Are you on the guest list?: Patient group involvement in HTAs

Instead of a ‘need assessment’ to determine whether patient and clinical representatives should attend committee meetings (which caused much concern among stakeholders), NICE will continue to invite patient, clinical, and commissioning experts to the First Appraisal Committee meeting, but will provide them with an opportunity to opt-out of attendance should they feel that their views have been adequately addressed by the technical team.In a bid to reassure stakeholders that expert views will still very much be an integral part of the process, NICE notes that this change accounts for expert engagement far earlier in the new process, stating “it is important that sufficient expertise feeds into all stages of the technology appraisal”.

Keeping a lid on it: Continued concerns about confidentiality

During the consultation, there was concern around the use of confidential information, particularly at the new technical committee stage. NICE considers it important to provide maximum transparency at this step and has stated it will share all clinical documents and analyses designated confidential with consultees and commentators in their un-redacted form during the technical engagement step. NICE sought to reassure industry, in particular, that commercial discounts and commercial access arrangements will remain confidential to protect commercial confidentiality between companies. Furthermore, organisations who want to be involved in an appraisal must sign a confidentiality agreement first to be considered a participating consultee or commentator. NICE have held firm during the consultation process on this issue, so companies should be expected to be pushed hard on what documents they will keep confidential.

The elephant in the room – but not in the guide: What is happening with MTAs?

There’s only been one multiple technology appraisal (MTA) carried out so far this year, in comparison to nearly 40 in 2017, and the concerns around the appraisal system not being fit for purpose for the more complex, multiple indication innovations coming down the pipeline are of course well established

With the above in mind, and MTAs not covered in the updated guide (we’re instead told by NICE to continue to go on the MTA process guide published back in 2014), a big question is what’s next for MTAs and the way NICE intends to effectively appraise the more complex innovations not suited for the single technology appraisal process?