Thread by @RitaINdeFaria

Next, live from #ISPOREurope IP8: Integrating Patient Preference into Health Technology Assessment- Can Patient Preferences be Incorporated into the ICER? w/Esther de Bekker-Grob @erasmusuni, Kevin Marsh @evideraglobal Mendwas Dzingina @pfizer & @JacolineBouvy at
@NICEComms

Esther de Bekker-Grob starts with the questions addressed in this panel

The background is that, given the increased focus on pt preferences, #HTA should not fall behind

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Why should #HTA consider pt preferences?
To improve adherence, to increase pt satisfaction, to make HTA decision making more informed & transparent, and it is ethical to listen to the pt voice.

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Ethical because the pt has a right to participate decisions that impact them, pts have experiential knowledge, and provides social legitimacy to decisions

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But there are challenges! From a recent SR in Value in Health, there are conceptual, normative, procedural, methodological and practical issues. Today, the panel will focus on procedural issues

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The panel will start with polling questions, then the panellists will share their thoughts, then the floor is open to the audience's Q&A

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Let's start with the polling questions in this #ISPOREurope panel. 88% thought that pts' preferences should be used to understand pts' treatment choices, 72% in estimating utility gains, and 60% in endpoint selection.

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Kevin Marsh comments by asking what is the appropriate role of pts in HTA?
Endpoint selection is about capturing what matters to pts, so pts pref should play a role.
Also in understanding pts' choices - would pts take the treatment if it is reimbursed?
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In relation to this former points, the questions are how to obtain these pts prefs and when.

The more controversial issues are raised in WTPs (40% votes) and to estimate utility gains (72% votes)

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Kevin considers that, for pts pref to have impact on HTA, this is where it will be achieved. But, in the majority of countries, it is the general population or decision-makers preferences which are thought to be legitimate in informing decisions.

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For Kevin, the utility gain from a specific gain in outcome could be informed by pt pref, while it makes sense to use public or DMs prefs for the WTP for the utility gain.
To use pt pref, there is an issue given that people can get adapted to the condition.

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@JacolineBouvy notes that, from the perspective of NICE, the highest value is in understanding people's choices. Pt pref studies are useful in helping endpoint selection (e.g. product that improves symptoms)

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Other applications: relative impt of non-health benefits (e.g. pill once a week v pill daily v IV delivery).

Also to inform regulatory approval in the benefit-risk profile assessment

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Mendwas Dzingina notes that 5% people voted that pt pref data doesn't have a role in #HTA - why is that?

Interesting that "Don't know" had 0 votes

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Mendwas notes that pt pref is impt to inform endpoint selection. Regulators have shown a lot of interest on this. FDA has recently issued guidance on this.

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Endpoint selection for situations when there are impt non-health benefits - how to determine which non-health benefits are impt? Opportunity to use pt pref to inform this, for example, with ranking or DCE

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Q: what are the barriers to implement pt pref in HTA?
Mendwas refers to the review paper that was shown earlier. The most frequently cited challenge was on the choice of methods given the lack of consistent methodology.

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Other issues: how to assess the impact of pt prefs on the outcome of the HTA process; lack of consensus about scientific rigour.

@JacolineBouvy comments that DCE provides insights but, if the technology is v CE/not CE, the DCE is unlikely to change recommendation

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Next poll question: Given the current state of practice, is it feasible to incorporate pts' pref into endpoint selection?

Mendwas comments that some endpoints are more or less amenable to pt prefs. E.g. how surrogate outcomes translate to symptoms.

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May be a challenge in therapy areas where there are no validated outcome measures - here pt pref are impt to understand what matters to pts and what they wish to be changed.

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HTA agencies may ask the rationale for endpoint selection. Choosing based on pt prefs is a valid rationale for this choice.

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Examples of studies: one study to inform endpoint selection, the other study on the benefit-risk profile, and a third study to inform treatment choice. The question is whether it is possible to do ONE study to answer these 3 questions?

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@JacolineBouvy comments that it depends on the indication if it is feasible to do one single study. E.g. harder in a rare disease.

Also depends on the technology.

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HTA bodies tend to have preference for final outcomes & HRQOL. A pt pref study should not be used to justify a novel endpoint if there are validated endpoints that can be used.

Nonetheless, endpoints should capture the impact of the condition on pts life

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Next question: how feasible is to use pts pref to understand pts' treatment choices?

@JacolineBouvy comments that it's feasible. Interesting that majority that people agree that it is feasible.

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Mendwas comments that there are few instances of when it wouldn't feasible, such as when pts have severe cognitive impairment, very ill people at the end of life (although it may be feasible here), young children (surrogacy via primary caregivers)

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Next question: How feasible is it to incorporate pts' prefs to estimate utility gains to inform a CUA?

Kevin comments that it depends on how we perceive this question. One interpretation is using pts pref to inform the weights of domains of QoL to inform a QALY.

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E.g. How much improvement in survival do pts trade-off for the change in treatment vehicle. There is precedent in the general population, but not really in pts. However, this does pose normative questions on whose prefs count.

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Question about consistency in the way pts trade-off attributes for survival. Chances are that pts may place different values on survival. We don't understand that enough, and more research is needed. If they are different, how acceptable are those differences?

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@JacolineBouvy comments that, from the perspective from NICE, utility weights need to be derived from general population pref. But there are HTA bodies that require pt valuations.

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Question in that, in models, there are utility decrements for AEs, for which evidence base is v limited, is there a role for a DCE - is anyone aware of research in this area?

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Mendwas notes that in most HTA agencies, the role of pt prefs is not clear. It might be useful to think about particular instances where there might be a much larger benefit in using pt pref, e.g. when benefits are not well captured by QALY (e.g. admin mode)

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Regarding the previous question about utility decrements for AEs, see poster: europe2020-ispor.ipostersessions.com/Default.aspx?s…

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Kevin comments that there is a lot of interest in working through the issues in using pts pref data to support HTA, but there are also a lot of questions to resolve

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My take: interesting panel about pts preferences for #HTA. Learnt how pt prefs can be used and the challenges. Really enjoyed the format of poll questions then panel comments!

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