HEOR and RWE in the COVID-19 pandemic: highlights from the first virtual ISPOR meeting
In May 2020, members of the Oxford PharmaGenesis Value Demonstration Practice joined more than 1300 health economic and outcomes research (HEOR) and market access professionals from 54 countries for the first ever fully virtual ISPOR meeting, participating in a broad range of presentations, workshops and symposia across the three-day conference. Although real-world evidence (RWE) and HEOR methods were naturally the focus, the COVID-19 global pandemic took centre stage in many presentations and discussions. Nevertheless, there was broad agreement that the role of RWE and HEOR in healthcare decision-making will continue to grow in the coming years.
The pandemic is shifting health policy conversations back to basics
The opening day plenary session promised a discussion on the future of health policy and the role of HEOR and RWE in shaping healthcare. Margaret Anderson (Deloitte) explained that the session had been planned before the COVID-19 outbreak to focus on the rise of value-based care, R&D productivity and customer engagement. However, the conversation turned quickly to the current global pandemic, and the panel experts offered several important insights into more pragmatic concerns around budgets and patient access to care in these unprecedented times. Andrew Dillon (St George’s Hospital, London) offered that public health and healthcare are cornerstones of a functional society and that better coordination is needed between governments, industry and donors to improve the scope and resilience of the global health system. However, Tomas Philipson (United States Council of Economic Advisers) cautioned that it should not be assumed that government alone can drive these improvements. Professor Philipson cited examples from the US COVID-19 response that show that in many cases, the private sector and citizens have been responding to the pandemic more nimbly and efficiently than federal, state or local governments.
Will COVID-19 affect the importance of RWE in healthcare decision-making?
Expert panellists agreed that there was an upside and a downside to the application of real-world data (RWD) use in the fight against COVID-19. The upside is that RWD will be essential to monitor the long-term effectiveness and safety of new vaccines for COVID-19, which will be the subject of close scrutiny in the coming months and years. The downside is the plethora of small RWE studies reporting short-term beneficial effects in COVID-19, often published in preprint form. Hans-Georg Eichler (European Medicines Agency, EMA) noted that the findings of these studies should be interpreted with great caution, and that the EMA has released guidance warning researchers against overstating the results. Effect sizes in COVID-19 are likely to be small and so unlikely to be detected robustly by non-randomized studies using RWD; randomized controlled trials remain the gold standard for demonstrating treatment efficacy in such conditions.
There is a growing need for RWE in both regulatory and payer decision-making
The COVID-19 pandemic has led many regulatory and payer decision-making bodies to focus their efforts in the short term on the management of COVID-19, and on accelerated review of potential treatments. For example, in the UK, NICE has prioritized the production of rapid guidelines and therapeutically critical guidance on COVID-19 infection, and ongoing health technology assessment (HTA) reviews of cancer therapies, while putting planned reviews for other therapeutic areas on temporary hold.
Of relevance to this area were discussions at the ISPOR meeting that although HEOR and RWE have traditionally fallen within the domain of HTA and reimbursement negotiations, these types of data are appearing more frequently in regulatory submissions. Through a series of use cases, experts presented compelling data supporting this trend, particularly in oncology, haematology and rare diseases. Encouraged by the 21st Century Cures Act, in 2018 the FDA provided guidance on RWE for use in regulatory submissions. Although the use of RWE in regulatory submissions dates to 1979, US-focused research showed a spike in 2019 (11 of 54 total submissions, with no more than 6 in any single year prior), suggesting that the FDA’s guidance has prompted an uptick in its use. Of note, medical and multidisciplinary reviews of RWE included in regulatory submissions highlighted limitations of lack of pragmatic design, but rewarded the use of real-world baseline characteristics for patient populations; these findings should be considered by any manufacturer planning to incorporate RWE in future submissions.
Ex-USA data also support a trend towards increasing use of RWE in HTA submissions, although with some differences between HTA agencies. A study of rare disease HTA submissions in Canada (CADTH) and the UK (NICE) showed that RWE formed part of almost 70% of rare disease submissions to CADTH, and 40% of such submissions to NICE. For the most part, the RWE came from patient preference and health-related quality of life studies; however, this is expected to expand to other study types as the role of RWE evolves.
While RWE is being used more often in regulatory appraisals, payers are increasingly being invited to provide input on pivotal clinical trial designs: an area that was traditionally the sole domain of regulators. Research on core outcomes recommendations for oncology trials and other important initiatives provides valuable insights for manufacturers and regulators about the types of outcomes data that payers and HTA committees will want to see in the future.
HEOR researchers continue to strive to meet the needs of healthcare decision-makers
The rapidly expanding applications for RWE in healthcare decision-making have triggered greater scrutiny on the quality of core methods and data sources. The HEOR research community is preparing to rise to new challenges with innovative and creative analytic approaches. As such, several workshops focused on new methods and sources of data; notable topics included patient-generated health data, new comparative effectiveness approaches using master protocols, and applications for artificial intelligence and machine learning. Overall, the first virtual ISPOR meeting was a triumph for the organizers and the HEOR community amidst the adversity of the COVID-19 pandemic, and provided an excellent opportunity for researchers and attendees to connect and discuss new challenges in healthcare, and how HEOR and RWE can help to meet those challenges now and in the future.
