HTAi Symposium “Embracing patient perspectives in health technology assessments: Integrating Quality of Life, Patient Experience and Preferences”

On June 17 I had the privilege of moderating the HTAi Symposium “Embracing patient perspectives in health technology assessments: Integrating Quality of Life, Patient Experience and Preferences”. Panelists included Renate Haidinger Meindert Boysen PharmD MSc Neil Bertelsen and Mihai Iulian Rotaru. Overall, there was agreement that we can improve how to include patient-relevant outcomes into HTA, and to do so, we distinguished between the process for patient involvement, and the evidence patients can bring into the process.

Process: The process for patient engagement is fragmented and patient feedback requests are carried out in many cases in an uncoordinated nature across multiple milestones in a medicine’s life cycle. The need for better alignment between regulatory and HTA decisions was highlighted, as shown during COVID-19. While different processes have their remits, prioritizing areas where patient input is most impactful within regulatory, payer, and HTA realms is essential. We advocated for a systematic, but focused approach to incorporating patient experience data and methods to address specific questions.

Evidence: Evidence is critical to make better, informed decisions. Patients need to play a role in evidence collection post authorisation and (initial) HTA as part of ‘risk management’ strategies, allowing further/future value assessments that provide for price/cost modulation where appropriate. Endpoints used to assess the ‘value’ of the new treatments, both for regulatory and HTA decision making, play a critical role. Traditionally, they focus on medicines’ effects (e.g. overall survival or surrogate measures), but we need to incorporate more ‘patient-relevant’ endpoints, including metrics measuring ‘time to event’ or ‘response rates’. In addition, Patient-Reported Outcomes (PROs), including Quality of Life variables, matter to patients, providing valuable insights into the physical, functional, social and psychological impact of treatments from the patient’s perspective – in oncology, for instance, this might be particularly important in the metastatic setting relative to earlier stages. To do so, we need to improve the way such data is collected and generated, by, for instance, developing better PRO questionnaires based on patient input, contextualising the evidence for decision making, and record and publish transparently how evidence is being used and the impact it has had. We also need to be mindful of the emerging field of Patient Experience Data as an input into both regulatory and HTA decisions. Care must be taken to ensure that patients are engaged with as this evidence is developed and contextualised for decision makers.

As a key final message: patient involvement is essential for meaningful decision-making, so we need to move away from the patient-centricity slogan ‘everything FOR patients’ to ‘everything WITH patients’. The next few years promise significant improvements in patient involvement in HTA, requiring collaboration in a trusted environment. I thank my panellists for their commitment and positive spirit during the preparation and Symposium. If you attended, hopefully you found it useful, and inspiring!

My final thanks go to the Menarini Group, for creating this opportunity to contribute to the important discussion on patient involvement in HTA. Matthijs Van Meerveld Judit Banhazi Santiago Moreno, PhD Lovro Bojanic Vivian Mendonca MD Emanuele Degortes