After NICE publishes its final decision on whether a medicine should be recommended for use in the NHS, the following registered consultees have the chance to launch a written or oral appeal if they disagree with the recommendation:
- The company
- Patient, carer, clinical, and commissioning experts
- Integrated Care Boards, Department of Health and Social Care, Welsh Government and NHS England.
Only the following are considered appropriate grounds for appeal:
- Ground 1: NICE failed to act fairly (specifically related to process unfairness, not the content of the guidance), or exceeded its powers (for example the guidance is contrary to equalities legislation)
- Ground 2: NICE’s recommendation is unreasonable when considering the evidence presented.
When an appeal is received, it first goes through a process known as scrutiny. This is where a non-executive director at NICE reviews the appeal points and decides whether they are valid. This includes a consideration of whether the appeal point falls within one of the criteria noted above, and whether it is ‘arguable’. If the non-executive director determines any of the appeal points are valid, then they are forwarded to the appeal panel, which consists of 5 members, 4 of which are independent of NICE. This is:
- Appeal panel chair (a health service representative, or a patient or career representative)
- NICE non-executive director (but not the person involved in scrutiny)
- Health service representative
- Life sciences representative
- Lay representative.
At the meeting itself, after the standard welcome and introductions, the chair will invite NICE and each appellant to give an opening statement of about 5 minutes duration. At the appeal meetings I have attended, when the appeal is about a negative recommendation, the appellants have often focused on patient need and clinical effectiveness. NICE have responded with genuine empathy but that regretfully they do not consider it to be cost-effective at the given price. Although each medicine and disease area obviously has its own nuances, this is often what the issues on each side boil down to.
The chair then runs through each appeal point, with NICE and the appellants each getting a chance to have their say. The chair/panel can ask probing questions. When all appeal points have been discussed, the chair invites NICE and the appellants to make a closing statement.
After the appeal, the panel meet to discuss their conclusions and aim to provide the outcome within 15 working days of the appeal, with 3 possible outcomes:
- 1 or more appeal points upheld – guidance is returned to committee for reconsideration. The committee must then meet to resolve the appeal issue.
- This is not a direction to the NICE committee to change its final recommendations. It is simply that the committee must further consider the issue that has been raised. This could lead to a change in outcome, but the decision is still within the NICE committee’s remit.
- The Appeal Panel requests changes to guidance – panel may request minor changes to resolve an appeal point, but they deem it does not require reconsideration by committee e.g. clarification wording.
- Appeal dismissed – no changes needed
If appellants still do not agree with the outcome, the next step is judicial review.
When I worked at NICE, I was involved in a couple of appeals, including one where I was in the ‘hot seat’ representing the NICE view. It was probably one of the toughest bits of the job, for several reasons.
- The context of an appeal: NICE team and appellants are ultimately on the same side of wanting to make sure patients get timely access to cost-effective treatments. However, NICE is focussed on all patients, whereas the appellants are (rightly) focussed on advocating for the treatment/disease area in the appeal. Therefore, even though as a NICE representative I knew it was the right thing for the ‘greater good’ that there were checks and balances to protect NHS budgets, it was still heartbreaking to know this specific set of patients might not receive a much-needed treatment.
- The preparation: involves many hours, poring over thousands of pages of documents (emails, meeting notes, scope, decision-problem pro-forma, company submission, clarification, addendums, errata, more addendums and errata if there have been multiple submission analyses, consultation responses, committee slides, and more)
- The meeting itself: recalling the rationale for various decisions, often involving highly complex technical work, with little time to think, and in front of an audience, was intense.
However, despite the difficult nature of appeals, everyone at NICE recognised how important they are as part of a fair process, and I was really proud of it as an example of the openness and transparency of the organisation.
Appeals are open to the public to observe, and they are really interesting. They can help to show the huge impact NICE’s decisions can have on patients, the back-and-forth debates between participants is fascinating, and it’s an insight into NICE’s interpretation of its own decision making, as they justify why and how they made complex and difficult decisions. The kinds of issues I have seen discussed at Appeal include:
- NICE declining requests from the company, for example the company requesting technical engagement, but NICE had deemed it is not needed.
- Section 5.7.2 of the manual states “Technical engagement will only be included if NICE considers that it is appropriate, helpful and proportionate, taking into account whether the technical engagement process is likely to resolve key issues before the committee meeting”. A key point to note here is that the NICE team, when deciding whether to conduct TE, is not determining whether there are any issues that require discussion, but instead whether there are any issues that require discussion that technical engagement can help resolve before the committee meeting.
- Whether NICE gave the company a reasonable amount of time to respond to committee requests
- Debate over whether a comparator is appropriate
- Whether enough time was given for the company to respond to an important discussion point
- Technical issues e.g. whether waning, wastage, extrapolation etc have been applied properly in the model.
My own personal reflections on what to consider during appeal include:
- Appeals often frequently reference the NICE manual. Ensure you are familiar and compliant with it.
- NICE and appellants are all on the same side of getting patients access to cost-effective treatments. But whilst the appellants are (rightly) fighting for what is best for the disease of interest in the appeal, NICE must take the bigger picture view, taking into account opportunity cost. Remembering this context can help both sides take a more empathetic approach, which can maintain good relations during the process.
- To help avoid an appeal, companies should consider:
- Whether you are clear on what analyses are being requested. NICE are usually happy to clarify.
- Although it may not always be possible, try to comply with what NICE requests, even if you disagree.
- A lower price reduces the risk to the NHS, so it can mitigate to some degree committee uncertainties about a medicine.
If you’re interested in observing an appeal, or wish to find out more, you can find out more information here. And if you have any questions on this article, please contact carl@axisconsultinguk.com