Opportunities and exploring the impact of patient involvement in HTA
The aim is to help ALAN to compare opportunities for patient involvement in Health Technology Assessment (HTA) across countries and explore its impact.
This could be useful to enable ALANs member organisations to identify what opportunities there are for them to contribute to HTA in their own country, to lobby for changes (i.e. if there are limited opportunities in their country versus others) and to learn what may be most effective in influencing HTA. This latter point is however uncertain given the broader challenges in disentangling the influence of patient involvement on HTA recommendations (more on that later in this proposal).
There may be also opportunities to leverage the research in the context of Joint Clinical Assessments as part of the new HTA regulation in Europe, in force from January 2022, but due to be implemented in January 2025.
Read the full report –> Opportunities and exploring the impact of patient involvement in HTA
Exploring the Difference that Patient Involvement in HTA Makes in Cancer
This research used data from IQVIA’s Market Access Insights database to identify the number of cancer patient group submissions (n=871) made to HTA agencies in Canada, England, France, Germany and Scotland between January 2020 and March 2023. It also looked at the openness of HTA agencies to patient involvement, informed by previous ALAN-commissioned research and the HTA recommendations made with and without patient group submissions.
The number of cancer patient group submissions has been increasing in Canada (from 26 in 2020 to 27 in 2021 and 37 in 2022), England (26, 35, 50), France (26, 35, 50) and Scotland (21, 24, 28) from 2020 to 2022. That means more work has been done by cancer patient groups and the HTA agencies. Cancer patient group submissions fell in Germany over the same period.
There isn’t a clear picture of whether the cancer patient group submissions made a difference to HTA recommendations, although the approach is descriptive and not able to explore causality.
The most open HTA agencies – CDA-AMC (formerly CADTH) in Canada, NICE in England and SMC in Scotland –have seen the highest proportion of cancer HTAs receive a patient group submission. It may also be capacity constraints are playing a role in whether cancer patient groups can respond to all HTAs in their cancer area.
More research is needed to quantify the cost of patient involvement, the drivers for patient group decision-making on whether to submit or not and what difference submissions make to HTA recommendations. Case study research could add valuable insights to help guide when patient groups are most likely to be successful in shaping HTA recommendations to help optimize their input.
Read the full report –> Exploring the difference that patient involvement in HTA makes in cancer
Nice and Appealing
NICE has received 117 applications for appeal since it started work in 1999, relating to 95 unique pieces of guidance. The appeal rate has been just under one in ten pieces of guidance since 1999, but the appeal rate has been falling over time. In 2023/24, three applications for appeals were made relative to 92 pieces of published guidance (3%).
The majority of applications for appeal have been successful, with eight applications rejected (7% rejection rate). Thirty-nine appeals related to guidance on cancer treatments (36% of appeals), and three were for treatments for leukemia (3% of all appeals, 8% of cancer appeals).
Companies have been involved in 81 of the 96 appeals where information was available. Patient groups were involved in 48, clinicians in 40, commissioners in 7 and providers in three. In five appeals, a patient group was the only stakeholder group to appeal.
Close to half of appeals where information was available were upheld (45 of 96, 47%), and the remainder were dismissed. Patient groups were involved in 30 appeals, which were upheld (66% of the 48 appeals patient groups were involved in). Patient group appeal points were upheld in 21 of these (42% of the 48 appeals patient groups were involved in). Of the five appeals where it was only patient groups who appealed, in only one was their appeal points upheld.
In cancer, 22 (61% of cancer appeals) were upheld, and 14 (39%) were dismissed. Cancer patient groups were involved in 13 of the successful appeals (59%), and at least one or more of their points of appeal were upheld in 11 (50%). A patient group was the only stakeholder group who appealed in one of the upheld appeals.
Appeals have been rarer about NICE exceeding its powers, with appeals more likely to relate to NICE acting fairly or that recommendations have been perverse or unreasonable.
Seventy-six per cent of recommendations stayed the same in 41 appeals where the pre and post-appeal recommendation was available. The recommendation also changed even when appeals were dismissed, with the inference being that other factors changed the recommendations in the final guidance. Previous research has found a greater impact of appeals on final recommendations reached.
The impact of appeals on NICE is likely to be more than the specific appeals themselves because the prospect of appeals may positively influence how NICE works, alongside the potential for further legal challenge.
Appeals come at a cost which has not been quantified, so it is not possible to say if improvements made because of upheld appeals have been worth the cost incurred. There is also likely to be a trade-off with timeliness with appeals adding to the overall timetable for NICE to produce final guidance.
Policymakers who oversee other HTA agencies should consider whether to introduce an appeals process, including allowing patient groups to be appellants.
Further research could also explore lessons by hearing from patient groups who have appealed, which could help patient groups focus their efforts only when an appeal is likely to be successful. Lessons could also come from legal action that has been taken against NICE.
The missing 13 appeals, as well as the small number where pre and post-appeal recommendations were available, have limited this research. There is also the possibility of unconscious bias and human error, given this research was conducted by a single researcher.
Read the full report –> Report – NICE and Appealing
Pace at SMC
Patient and Clinician Engagement (PACE) process at the Scottish Medicines Consortium (SMC) stands out as an additional effort to hear directly from those affected by the disease being looked at.
PACE differs to what can be seen as a more passive approach primarily driven by written submissions used by other HTA agencies because it offers a separate meeting that only includes patient and their representatives, clinicians and SMC staff. It cumulates in a consensus statement on the added value of treatments that is then shared with the decision-making committee at the SMC. The natural question that flows is can we find out what difference does this extra effort make to recommendations?
Read the full report –> PACE at SMC
