ISPOR Science and Health Policy Initiatives - August 2022

Published Aug 15, 2022

New ISPOR Reports

  1. The ISPOR Health Preference Research Special Interest Group published a report titled, “Accounting for Preference Heterogeneity in Discrete-Choice Experiments: A Review of the State of Practice—Report of the ISPOR Health Preference Research Special Interest Group” in the May 2022 issue of Value in Health. See the press release here.


  2. The ISPOR Nutrition Economics Special Interest Group published a new report, titled "Are We Ready for a New Approach to Comparing Coverage and Reimbursement Policies for Medical Nutrition in Key Markets: An ISPOR Special Interest Group Report" in the May 2022 issue of Value in Health. See the press release here.


  3. The Joint HTAi / ISPOR Deliberative Processes Good Practices Task Force published a report, titled “Designing and Implementing Deliberative Processes for Health Technology Assessment: A Good Practices Report of a Joint HTAi/ISPOR Task Force” in the June 2022 issue of Value in Health. See the report here. For the press release, see here. A webinar will be held for this Good Practices Report on 30 September 2022 at 9am EDT. You can register for the event here.


  4. The ISPOR Machine Learning Methods in HEOR Task Force published a new ISPOR report, titled “Machine Learning Methods in Health Economics and Outcomes Research—the PALISADE Checklist: an ISPOR Task Force Good Practices Report” in the July 2022 issue of Value in Health. See the press release here.

Recent Events

  1. The ISPOR Health Technology Assessment (HTA) Roundtable – Latin America was held on July 28, 2022.  It included more than 25 HTA bodies, public and private payers, and other governmental decision makers from various countries. They met to discuss how to include economic evaluations into HTA, the use of thresholds and budget impact, and the challenges of conducting HTA in pluralistic healthcare systems. For more information about the ISPOR HTA Roundtables, please see here.

    Speakers offered representation from Institute for Clinical Effectiveness and Health Policy (IECS); National Scientific and Technical Research Council (CONICET), Instituto de Evaluación Tecnologica en Salud (IETS), and the Colombian Ministerio de Comercio, Industria y Turismo (MINCIT).  This Roundtable was Co-Chaired by Sebastian Garcia Marti, MD.

  2. The ISPOR Patient Representatives Roundtable – Latin America was hosted on July 26 and convened over 30 attendees from patient organizations, researchers, government, and HTA bodies and payers. They met to discuss effective social participation in health technology assessment (HTA) and mapped the patient journey in the process.

Speakers offered representation from Unidos Pela Vida | Instituto Brasileiro de Atenção à Fibrose Cística and Institute of Health Technology Assessment – IETS, the Colombian agency of HTA. This Roundtable was Chaired by Gustavo San Martin Elexpe Cardoso, Chief Executive Officer of Amigos Múltiplos pela Esclerose (AME). For more information about the ISPOR Patient Representatives Roundtables, please see here.


Recent Webinars Available for On-Demand Viewing

  1. The Clinical Outcome Assessment Special Interest Group hosted a webinar, titled Leveraging the Value of Clinical Outcome Assessment (COA) Data Beyond Labelling on April 12, 2022. To view the webinar, click here.


  2. The Health Equity Research Special Interest Group hosted its first webinar, titled “Distributional Cost-Effectiveness Analysis to Inform Healthcare Decisions” on June 23rd. If you missed the webinar, click here.  
  3. The Digital Health Special Interest Group hosted a webinar on July 27, titled “Data Privacy and Digital Health: The Manufacturers/Payers Perspective”. To view the recording, click here.

New Opportunities

  1. The Medical Devices and Diagnostics Special Interest Group has launched their community, a place where members can engage, collaborate, share ideas, discuss challenges, get insights, support career development, and post other questions related to medical devices and diagnostics. To get involved with this special interest group, please visit their webpage.


  2. click here. To apply, please contact[EMD1] 
  3. ISPOR member groups have conducted live journal clubs—focused discussions on specific, published, scientific research papers—on topics related to medical devices and diagnostics, open-source models, precision medicine and advanced therapies, in addition to other timely topics. To learn more and view journal clubs on-demand, please click here. Recently added Journal Club recordings include:

ISPOR Open Source Models Special Interest Group

-        Simulating Progression-Free and Overall Survival for First-Line Doublet Chemotherapy with or without Bevacizumab in Metastatic Colorectal Cancer Patients Based on Real-World Registry Data

ISPOR Precision Medicine and Advanced Therapies and Medical Devices and Diagnostics Special Interest Groups

-        Health and Budget Impact of Liquid-Biopsy-Based Comprehensive Genomic Profile (CGP) Testing in Tissue-Limited Advanced Non-Small Cell Lung Cancer (aNSCLC) Patients


Dialogue with Decision Makers

ISPOR 2022 Health Technology Assessment – Pharmacy & Therapeutics (HTA-P&T) Roundtable – North America

On May 27, 2022, ISPOR virtually held the 2022 ISPOR Health Technology Assessment – Pharmacy & Therapeutics (HTA-P&T) Roundtable. 32 participants from Australia, Canada, Mexico, and the United States convened to discuss the challenges of grading and communicating uncertainty of evidence and to discuss the topics of past HTA-P&T Roundtables.

ISPOR has been convening annual Health Technology Assessment (HTA) Roundtables since 2007 with Roundtables now held in Asia Pacific, Europe, Latin America, Middle East and Africa, and North America. These Roundtables are closed-door, invitation only meetings between HTA bodies, public and private payers, other governmental decision makers, and academics when no HTA body exists in a country. They provide an unbiased platform to advance scientific methods; facilitate information-sharing about the current state of HTA, its development, and its role in optimizing healthcare decisions; and bridge the gap between technology assessors, private and public payers, regulators, and patients to improve health globally.

The HTA-P&T Roundtable in North America is comprised of HTA bodies, pharmacy benefit managers, medical insurance companies, and other governmental organizations involved in pricing and reimbursement of healthcare technologies in North America.

At the most recent meeting on May 27, Rebecca Trowman, MSc, Scientific Secretary of the HTAi Global Policy Forum presented an overview of uncertainty of evidence and the findings from the 2021 HTAi Global Policy Forum. The presentation described that there is input, throughput and output uncertainty in HTA. At the input stage, there are three types of uncertainty, namely clinical, economic and affordability uncertainty and, at this stage, uncertainty needs to be characterized and summarized for people to make decisions on. At the throughput stage, the committee considers the uncertainty and determines how the uncertainty affects the evidence. This leads to an HTA decision, and the type and magnitude of uncertainty may impact how a technology is reimbursed or recommended. The output stage is the final stage where the presence and impact of the uncertainty needs to be communicated to all stakeholders. It was acknowledged that issues of uncertainty will not go away, and uncertainty is in fact increasing, and so we need collaborative methods to characterize and communicate uncertainty in HTA.

Ms. Trowman set the stage for a fruitful discussion amongst roundtable attendees by asking them how their organizations handle uncertainty of clinical or economic evidence. Many participants felt that handling uncertainty was becoming more important as cell and gene therapies and new oncology products with surrogate outcomes are being approved by the US FDA. However, there were concerns about transparency and replicability of managing uncertainty. Comments were also made about the use of managed entry agreements and how they cannot be used for every product and must be prioritized.

The discussion then shifted to a brief discussion on coverage and reimbursement of products that do not show clinical benefit and may cause harm to a patient. One payer said, “there are some products that a payer will not want to cover, even if the price is zero because of benefit/risk profile.” Many payers in the US found that for the first time, physicians were not prescribing a product due to the uncertainty of risks and benefits for the patients. The difficulties of communicating value and uncertainty of evidence to employers was also mentioned, with a suggestion to include large employers in future HTA Roundtables.

The second half of the roundtable focused on discussion topics from the last 15 years of HTA-P&T Roundtables and the value that the Roundtable has brought to organizations that attended. Many people who have attended for 5+ years agreed that the HTA-P&T Roundtable – North America added value to their jobs because they were able to take the discussions held at the roundtable and bring it back to their organization to help make decisions. Previous discussions about value-based formularies had multiple payers consider how they would implement such a thing. In addition, the value of having patient representatives at the HTA-P&T Roundtable was emphasized heavily, so much so, that one payer changed their internal policies to include more interactions with patients due to what they learned at the HTA-P&T Roundtable.

There was a robust discussion about the value of having the United States payers interact with the Canadian HTA bodies and payers and vice versa. Both countries agreed that they are able to learn from one another and that ultimately the healthcare systems are not as different as everyone may think. There was a heavy emphasis on increasing the interactions between different countries and regions to learn from one another.

The roundtable concluded with HTA Council Chair and ISPOR President-Elect 2022-2023, Brian O’Rourke thanking the outgoing co-chairs, Jessica Daw of Sentara Health Plans and Mitch Moneo of the British Columbia Ministry of Health, for their terms as co-chairs and leading the roundtables during the pandemic. The incoming co-chairs, C. Bernie Good of UPMC Health Plan and Suzanne McGurn of CADTH, were then introduced and welcomed.


On 8 June 2022, the ISPOR 2022 Patient Representatives Roundtable – North America convened 37 stakeholders from North America representing government agencies, payors, patient-centered organizations, health economic organizations, and research institutions. Discussion centered on key considerations for creating useful fit-for-purpose patient registries, including when and how to best engage patients and other stakeholders, key partnerships, and the use of patient registries to further disease awareness and clinical research.

Survey on stakeholder patient registry experience

A pre-meeting survey was sent to participants to gather perspectives on and experience with patient registries. Although most participants (~75%) thought that registries were very or extremely useful, many participants indicated that their affiliated organizations did not have a registry. Reasons selected for an organization not having a registry included not knowing who to partner with, lack of expertise/resources, unsure of where to begin, and unclear purpose for the organization. Of those associated with an organization that did have a patient registry, most responders (~85%) were satisfied/very satisfied with the registry and indicated that the patient registry fulfilled its purpose better than expected. All responders indicated that their registries had evolved over time.

Common challenges of a patient registry occurred throughout the life cycle and included fundraising/start-up/early planning, data analysis, growth, and continued financial support. Responders indicated that patient registries had a variety of direct contributors and results were shared with a broad audience, where patient communities, biopharma, and regulators were identified as those most likely to change their behavior based on the results. Patient registry results were least commonly shared with legislators and HTA bodies (< 40%), with payors and legislators listed as those least likely to change behaviors based on the results.

Presentations on patient registry execution and key considerations

Three representatives from patient-centered organizations and research institutions presented their experience and their suggested best practices in the design, implementation, and use of patient registries. 

The first presentation reviewed the importance of patient engagement at multiple steps along the clinical research process not only to ensure research is patient centered, but also to improve study quality/value, study governance, feasibility/relevance of the studied intervention, and successful uptake of the research results. Patient engagement should be dynamic, taking different forms throughout the research process. The speaker recommended that patient registries be viewed as a bidirectional proposition, offering contributors a returning value or benefit for participation. Although it was noted that continued patient engagement was feasible, several challenges were identified, including those related to resources, organizational policies, and the balancing of views and priorities.

The second presentation focused on the design and use of patient-driven registries. The speaker noted that having patients drive the registry creates value for their communities, and that patient-driven registries have led to increased disease visibility, scientific publications, additional studies/sub-studies, and research partnerships to further advance disease research and patient treatment. The speaker highlighted their organization’s experience in the practical aspects of patient registry platforms, specifically the consideration of both patient and regulators’ needs in registry platform design and that the platform should support ease of use, customization to the specific disease, and flexibility with data control and input. The inclusion of multiple languages in registry platforms can also support international studies and the development of a diverse and robust registry. Although patient-driven registries have the advantage of patient engagement in creation and oversight to create a meaningful registry, there are multiple support needs for the successful development and implementation, and these registries also need to maintain scientific rigor. A registry advisory board can provide guidance and help make the best decisions for the community.

The third presentation focused on the value of the partnership between patient registries and researchers to ensure that a registry is relevant to the patient while also supporting quality clinical research that will be accepted by the scientific community. The speaker noted that it is important to recognize what each partner brings to drive the research, and effective partnerships should include elements such as transparency, trust, shared interest, communication, and reciprocity. It is also important to have agreement on data collection, storage, ownership, and use, and how the findings are communicated. Both sides of the partnership want to make a real contribution and clarification of that contribution is important to accomplish the goals of the patient registry.

Additional key considerations for patient registries identified during the roundtable discussion

Data privacy requirements: Although patients may be driving the registry and may have control over the privacy of their own data, there are often national/regional laws around data privacy that may need to be taken into consideration.

Sustainability: Registries may be initiated by stakeholders whose interests change with time and/or may not have the resources for registry maintenance despite the broader benefit. The collective stakeholder group may need to work together to identify how to support the long-term function of a registry. 

Strong patient voice: Ensuring continued patient engagement may support maintenance of the registry focus and registry longevity despite a potential change in key stakeholders.

Funding: Funding is important for registry sustainability and may come from several sources such as partnerships, research organizations, donors, and grants. Early partnerships between patient organizations and researchers may help define key research questions that can merit funding through patient organization awards or other funding opportunities.

Diversity: Despite the desire to have a robust patient registry, there are several challenges to the inclusion of a diverse patient population, which may be partially addressed through use of multiple languages or engagement of different patient communities. Registry leadership should also be diverse and represent multiple voices.  

Payor engagement: Although the survey results suggested that payors may be less likely to exhibit a behavior change based on registry data, roundtable participants indicated that some payors are looking for formal patient input to address uncertainties with marketing approval. In addition, payor inclusion in registry design may be beneficial.

Publication of registry data: Publication of patient registry data in reliable peer-reviewed journals may better support scientific acceptance and use of data to advance clinical research and disease awareness. Patient registry data may also help inform the review of clinical trial publications to assess whether trial design is relevant to the needs of the patient.


Patient registries are useful and can add significant value, especially patient-driven registries; however, there are several barriers to creation and implementation. In addition to the effort, resources, and financial support required to create and maintain registries, patient engagement, establishment of key partnerships, and multiple other factors should be considered to ensure the registry is fit-for-purpose and effective at furthering clinical research. Finally, putting a process in place that allows registry evolution may support registry growth, long-term success, and continued benefit and relevance over time.


This summary was developed by Brittany Carson, BSc, MHEcon, PhD. We would like to thank the co-chairs of the ISPOR Patient Representatives Roundtable – North America for their direction and leadership - Alan J. Balch, PhD, Chief Executive Officer of Patient Advocate Foundation and National Patient Advocate Foundation and Lisa J. Pieretti, MBA, Executive Director of International Hyperhidrosis Society. We greatly appreciate all speakers and attendees for their participation and valuable contributions.


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