This year the theme for the ISPOR European conference to be held in Vienna, 6-9 November is Collaborating Across Boarders: Building and Using Evidence to Enable Access. The conference is being held in person and virtually, and registration includes access to on-demand sessions until 9 December 2022. This provides a great opportunity to catch up on topics you missed at the conference, or if you are unable to attend in person.
A couple of topics caught our eye this year – here’s the first:
Collaborations between Health Technology Assessment (HTA) bodies, and between HTA bodies and regulatory authorities.
Monday’s opening plenary (The convergence of HTA and Regulation: A New HTA Reality and Collaboration with Regulatory Agencies) is a must attend session. It includes speakers from the EMA (European Medicines Agency), the Spanish Ministry of Health and EUnetHTA (European Network of HTA), and should be an interesting discussion that looks at the effects of closer collaborations during all phases of the drug lifecycle, and what activities and remits could be optimized if regulatory and HTA assessment converged. No doubt the effects of keeping the (somewhat trying) existing differences in the remits will also be examined.
In recent years both the regulatory bodies, such as the EMA and United States (US) Food and Drug Administration (FDA); and HTA bodies, such as the United Kingdom’s National Institute for Health and Care Excellence (NICE), have become open to using RWE (Real World Evidence) to support decision making. Following the first plenary session, the theme of collaboration between HTA and regulatory bodies and their use of RWE recurs throughout the conference. Here are some of Monday’s sessions that examine this topic from a number of different angles.
In a breakout session, European Payer: Regulatory, and HTA Perspectives on RWE: Closely Aligned or Far Apart?, presentations will be given from the payer, regulatory, and HTA perspectives, followed by a discussion which will aim to identify areas of alignment and discrepancies in the virtues of RWE. EMA, NICE and the Netherlands National Healthcare Institute (ZIN) representatives are on the panel.
A spotlight session, Recommendations for a Harmonized Health Technology Assessment for Digital Medical Devices (DMDs) Across Europe: A Multistakeholder Discussion includes French, Belgium and Luxembourg opinion leaders, looking at how a fragmented approach to the reimbursement of digital medical devices impairs patient access. We believe it will be very worthwhile hearing how solutions such as RWE and innovative study designs might benefit patient access.
In podium sessions:
- RWE Applications in Health Technology from Optum Life Sciences will present case studies on how RWE has been used in HTA assessment.
- IQVIA’s presentation Can You Design and Conduct a Real-World Evidence Study That is Transferable Between HTA Bodies? will cover RWE guidelines from NICE and the French Public Health Department, Haute Autorité de Santé (HAS), to assess the feasibility of conducting one RWE study that is acceptable to both HTA bodies.
With the aim of harmonizing and streamlining the clinical assessment process for HTA bodies across the EU, in December 2021 Regulation (EU) 2021/2282 was published and enacted in January 2022 to implement a European Union (EU) wide Joint Clinical Assessment. An overview of the new EU regulation and the challenges it proposes will be discussed at the HEOR theatre, in the session: EU Joint Clinical Assessments: What Happens When Gold-Standard Evidence is Out of Reach. We think this session is definitely one to attend if you want to better understand the new HTA regulation and the issues likely needing to be addressed.
One of Tuesday’s podium sessions, The Role of Real-World Data in HTA and Regulatory Decision Making, moderated from the pharma industry perspective, will explore how RWE has been used in HTA and regulatory decision making and the lessons that can be learned.
In Tuesday’s Education Symposia, New Pan-EU HTA: How Can Biopharma Navigate the Joint Clinical Assessment?, discussion focuses on the content of the EU joint HTA regulation, and the likely impact for joint and individual EU members of HTA bodies, and those preparing HTA submissions. This will be a great opportunity to get up to date with the EU joint regulation and thoughts from HTA gurus about implementation.
The second topic which caught our attention is Patient-centered data – the topic of Tuesday’s plenary session (Real-World Patient Centered Research: Is it Possible Across Countries?), and of many subsequent sessions. The rationale for patient-centered data is based on the premise that the outcomes of health care will be improved if the voice of patient -the ultimate customer and beneficiary – is heard when making healthcare decisions. During the second plenary session speakers from Australia, the Netherlands, USA and UK will present several initiatives showing how patient-centered research could be conducted across countries in the future.
The Joint ISPOR-ISPE (International Society for Pharmacoepidemiology) Special Task Force on RWE has developed 13 consensus recommendations on how patient experiences and insights can be incorporated into real-world research. These will be presented at a concurrent breakout session on Monday: A Workshop on Developing Patient-Centered Real-World Evidence: Consensus recommendations and Case Examples. Discussants come from the US and UK and represent ISPOR, ISPE; Merck as the industry representative and Applied Patient Experience as the data collection agency. We expect this to be highly informative as the recommendations should provide a guide to best practice and how to overcome the challenges.
A concurrent breakout session How Can the Voice of the Patient be Better Heard in HTA Decision Making in Rare Diseases? will present a case study on patients with glycogen storage disorder Ia. In addition, the challenges for capturing the patient perspective and potential solutions will be discussed with a focus on qualitative data. The Patient-Centered and Rare Disease Discussion Group moderated by Andrew Lloyd (London, United Kingdom) picks up the discussion later that day.
Qualitative patient-centered research is also a popular poster topic. The poster to be presented by IQVIA Spain: A literature review of methodology used in in-trial interview studies in clinical development, addresses how in-trial interviews can help understand patient experience with diseases and treatment, the clinical trial and their preferences. The importance of in-trial interviews for regulatory decision making is on the increase, but still not utilized as much as it could be.
Monday afternoon revisits rare diseases and looks at an evidence-based patient advocacy framework for promoting research in rare diseases (Development of an Evidence-Based Patient Advocacy Framework, and Further Activities Promoting the Research in Rare Diseases). It will be interesting to see if these applications are relevant beyond rare disease.
Patient preference studies
Patient preference studies have been around for at least two decades and, despite the growing interest and increasing use of patient preference studies in health care decision making, their methods and value continue to be debated. The following selected sessions provide a great opportunity for you to keep on top of the debate.
- Monday morning’s podium session, Frameworks and Methods for the Design of Stated Preferences, is moderated by Jacoline Bouvy (NICE—London, United Kingdom) who we expect to be adding comments and questions regarding the NICE perspective. The session features this topic: EMA/EUnetHTA Qualification of the IMI PREFER Patient Preference Framework and Points to Consider for Methods Selection: Key Experiences, Outcomes, Values and Implications, presented by an academic from the Department of Pharmaceutical and Pharmacological Sciences in Leuven, Belgium.
- At Monday morning’s ISPOR Forums in session, A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision-Making in Health: The Final Recommendations from the ISPOR Using Patient Preferences to Inform Decision Making Good Practices Task Force, will highlight the five domains developed by this particular ISPOR Task Force to guide researchers and other stakeholders. These domains should help make patient preference studies more useful to decision makers. Speakers will present the ISPOR Patient Preference Roadmap, the patient perspective and the HTA perspective.
- The Open Meeting- Health Preference Research Special Interest Group taking place on Tuesday will discuss its forthcoming project on individual value clarification and will provide the opportunity to network with others interested in health preference research.
- Concurrent Break Out Session 7 delves into the interesting topic: How to Assess and Implement Patient Preferences in Decision-Making Along the Medical Product Life Cycle?
Social media listening
A number of posters present social media listening as a means to capture patient-centered data. This relatively new approach has yet to be used to its full potential by pharma industry and regulatory authorities. The sessions we highlight here will help you understand the opportunities social media provides. It is a rich resource for describing patients’ and caregivers’ experiences with respect to the impact on health-related quality of life, healthcare and treatment experiences to help characterize unmet need. For example:
- Pathen et al. Identifying patient experiences and unmet needs in Cushing’s syndrome through the voice of the patient perspective – A social media listening (SML) analyses using real world data form 2019- 2022
- Crawford et al. Patient and caregiver insight into the impact of non-muscle invasive bladder cancer and its treatment: A social media review
- Patel et al. Patient and caregiver perceptions around the treatment and quality of life impact of colorectal cancer: A social media listening study
- Speis et al. Retrospective social listening study of patients living with systemic lupus erythematosus (SLE): Understanding the patient experience.
Furthermore, data captured by social media listening can help develop a patient-centered drug development strategy informing the characterization of unmet need, target product profiles, value demonstration and evidence generation strategies, patient-centered clinical endpoints and the patient reported outcomes to implemented in clinical trials. See the poster: Towards an artificial intelligence-enables social media listening solution to inform early patient-focused drug development strategies, presented by Guitian Oliveira (Merck KGaA—Darmstadt, Germany).
Have a great ISPOR!