Each year global health economics and outcomes research (HEOR) leaders, policy makers, regulators, researchers, academics, payers, patients, and patient groups come together in the United States for the annual ISPOR conference. A staple in the HEOR calendar, the event is a great opportunity to network with peers, connect with clients, and debate the latest hot topics in the industry with like-minded professionals.

Back with a bang this year after ISPOR dropped the hybrid format it had adopted immediately following the COVID-19 pandemic, the conference was well attended and had a feeling of real buzz about it as consultants, pharmaceutical professionals, academics, and students descended on this year’s host city, Boston.

In this short article, we summarize some of the hot topics we identified from an insightful few days at the conference. Being only a short summary, where possible we have linked to further resources on these themes, however you can subscribe to receive all our illuminating insights on these topics and more by registering here.

If you have any questions, do not hesitate to contact us.

1. Inflation Reduction Act – a source of angst and scepticism

Very few familiar with the US model for pricing and reimbursement will be shocked to see this at the top of our list because the Inflation Reduction Act (IRA) has so far created more questions than it has answered. In fact, the lack of available detail, in particular for how real-world data (RWD)/real-world evidence (RWE) will be used, and whether the Center for Medicare and Medicaid Services (CMS) will use comparative effectiveness measures at all in setting the maximum fair price for new drugs, were key talking points at the conference.

Questions over whether the IRA will stifle innovation also continue to be raised, as do concerns about the pace of the roll out, lack of transparency, lack of a value framework and limited opportunities for manufacturers to engage with related government departments under the new regime. Concerns were also raised about the IRA’s possible impact beyond the US, future expansion of the IRA and its likely effect on small companies in terms of fund raising. Amongst the questions and evident cynicism are also anxious voices from some of the manufacturers with therapies in the first and second wave assessments under the IRA. There are questions about how the CMS will assess information and evidence in a useful way to support negotiations. Such issues may become clearer when the CMS publishes its revised guidance in July.

What remains certain is that the IRA will continue to be a topic that will divide opinion as it continues to be rolled out. Want to find out more about the IRA? Watch our recent webinar on demand.

2. Using Real-World Evidence in Health Technology Assessments

Immediately ahead of the main conference, ISPOR—The Professional Society for Health Economics and Outcomes Research, the International Society for Pharmacoepidemiology (ISPE), and the Duke-Margolis Center for Health Policy, hosted an RWE summit to discuss key issues surrounding the use of RWD for treatment effectiveness in regulatory decisions and health technology assessment (HTA).

With general acknowledgement that sufficient analytic methods are now available for data analysis, four panels of thought-leaders provided keen perspective on the new initiatives and best practices to ensure data quality, appropriateness or “fit-for-purpose” of data for a given research question, and transparency beginning with data selection to protocol design, and from research execution to reporting study results. Pre-registration of protocols for “hypothesis-evaluating treatment effectiveness” RWD studies now begets accelerated review for subsequent manuscripts submitted to ISPOR’s journal, Value in Health. So, while appropriate scepticism remains about the ubiquitous use of RWD for treatment effectiveness in regulatory submissions, the ongoing progress for new standards and processes from across stakeholders provide further steps toward a possible commonly accepted pathway in future.

Moving away from regulatory use of RWD to the use of RWE in HTAs, the lack of a structured approach and best practice that is acceptable to the major HTA bodies globally was also a key theme on the first day of the ISPOR conference. Lumanity’s HEOR Executive Chair, Ron Akehurst, and Senior Director, Linda Murphy, hosted and participated in a panel discussion which aimed to present a partial solution to HTA agencies’ reluctance to use RWE to reach reimbursement decisions.

The content of the session was based on a series of articles Ron and Linda recently published in Value in Health outlining the key obstacles and proposing practical solutions to increasing the optimal use of RWE in HTA decisions. The session was well attended and a poll at the end of the session highlighted that most of the audience were ‘very enthusiastic’ about the solutions discussed.

You can find out more about this series of published articles below.

3. Incorporating the patient and carer voices

Our final key theme from ISPOR is around including the patient and/or carer voice in the drug development process. There has long been discussion around including patient reported outcomes in HTA and a growing acknowledgement among HTA bodies that such data can be hugely valuable in the HTA process. An interesting extension of this was the proposition that “caregiver reported outcomes” should also be considered for use in HTA – while some seemed hesitant at this idea, others acknowledged that even anecdotal/highly qualitative data could be useful in contextualizing the impact of treatment on caregivers.

Patient preference was another topic discussed in detail at ISPOR. The development of “core outcome sets” for use in regulatory decision making, HTA, and patient-clinician decision making gives the patient the opportunity to define criteria important to them, in effect giving them an effective veto on selections made solely by clinicians or specialists, thus ensuring what matters to patients is what is included in clinical trials.

In addition, patients were identified as key decision-makers whose opinion is usually not considered when developing trials. The impact of including patients in the design of trials was considered mixed: some felt the burden of trial participation could increase if a lot of patient-identified measures were included in trials, while others felt the number of times data was collected could fall based on patient feedback thereby decreasing burden.

Overall, conference presentations noted the importance of including the patient’s voice when evaluating treatments and documenting the burden of a disease.

We produced a deep dive article, published in PharmaPhorum exploring how Burden of Illness Studies could add value to HTA submissions. Click below to access the full article.


We hope this quick digest of some of our key conference takeaways is helpful. If you missed us at the conference, or if we can be of any assistance, please do not hesitate to reach out to us.

In the meantime, we look forward to attending the ISPOR Europe conference in Copenhagen in November.

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If you missed us at ISPOR, or if you would like to find out more about any of the topics covered in this article, please contact us.