ISPOR EU 2020 delivered a wide spectrum of topics related to market access. Avoiding the topic of COVID-19 and the challenges this pandemic has brought to our discipline was impossible, it seems, but the event provided lots of food for thought. COVID-19 aside, one of the most striking themes in the sessions we attended was that of challenging the status quo and disrupting traditional market access approaches and health technology assessment (HTA) methodologies. How? By creating new elements of value, increasing patient-reported data, and simply delivering efficiencies that fast-track market access process.
The focus on disrupting the status quo has partly been driven by an increase in small and medium-sized biotech entities, in which smaller, inexperienced access teams are bringing products to market with limited data sets. This discussion was part of a larger theme, discernible across the conference (and undoubtedly influenced by the pandemic), of expediting access to get needed drugs to patients quicker.
The HTA Approach to eHealth
An interesting discussion questioned whether the adoption of digital health (DH) technologies should be accelerated during global disease outbreaks. Currently, randomized controlled trials (RCTs) are used to provide evidence for DH when there is a large and expected effect on clinical outcomes. Apps and software that aim to address other aspects of care do not need to go through an RCT. One relevant paper presented was a scientific literature review on HTAs and eHealth (PubMed article), which recommends standardization of HTAs for eHealth to improve the quality and comparability of the assessments.
Meaningful Patient-Reported Outcomes
Another significant topic was the role of meaningful patient-reported outcomes data in complementing reimbursement submissions. Recognition of disease-specific quality-of-life metrics is a major step forward in the current “Access Era,” both for consistency of outcomes and importance to patients.
A challenge to the status quo was embedded in a discussion of new possibilities for substantiating value. Those possibilities include equality, population-level productivity, the value of hope, and reductions in uncertainty and are particularly relevant when considering the provision of value frameworks for next-generation testing and diagnostics: How do public/private providers alike manage decision-making about multiple-marker testing and interdependent technologies?
Traditional access and commercialization routes are now challenged by new products that produce not mere incremental advantages to the current standard of care but rather abrupt changes that deliver better outcomes—for example, new products designed to be curative and life-long. It is clear we need new ways of measuring innovation.
COVID-19: Real-World Data Challenges
The pandemic may go away, but COVID-19 will not. Attending this session about real-world data challenges heightened our appreciation of the importance of real-world evidence generation for COVID-19 to provide rapid insights around treatment decision guides. Doing so guides effective prevention and treatment strategies, informs us of variation in outcomes within high-risk populations and subgroups, and demonstrates the effectiveness of several breakthrough vaccines. With such a vast proportion of the population affected by COVID-19 over such a short period of time, the amount of real-world data available globally is vast. The real challenge, however, is capturing data in such a way as to ensure that its validity and statistical integrity are maintained to produce robust and relevant outcomes in such a fast-moving pandemic.
In 2021, we expect to see a drive toward the creation of universally acknowledged and validated COVID-19 measures. Thus far, due to the lack of validated measures and unambiguous, unbiased defined COVID-19–specific endpoints, it has proved challenging to compare analyses generated by various COVID-19 studies. These recommendations are based on lessons learned from oncology, where the creation of specific endpoints such as progression-free survival and overall survival have informed drug-approval decisions and ensured that treatment options can be evaluated appropriately across the therapeutic area.
Future Access Pathways and Assessment Routes
There was also a sense check across the conference—perhaps driven by the COVID-19 pandemic—that the current course of healthcare provision is unsustainable for future generations. Worldwide, an estimated 20%–40% of healthcare resources are wasted (WHO/OECD), primarily through misdiagnosis, low treatment adherence, and inappropriate hospitalization. Those who forge future access pathways and assessment routes will need to keep healthcare sustainability top of mind.
What Does This Mean for Market Access in 2021 and Beyond?
- Gone are the days of “off the shelf” access programs and ready-made solutions. Global market access is unique, and each product journey will be different.
- It is important not be afraid of challenging the status quo; reimbursement authorities are prepared to listen and learn, as new elements of value are continually emerging.
- The patient voice and meaningful patient-reported data are continually helping to reinforce and even shape the value narrative.
- Health system sustainability, service efficiencies, and cost impacts of new technologies will continue to gain importance. But be prepared: Expectations on financial clarity will only increase.
- Expect more organizational co-operation and partnering to surmount various regulatory, reimbursement, and commercialization challenges. This may be particularly pertinent in rare diseases.
- Expect to see tradition challenged. Nimble, fast-acting companies that are prepared to take risks will benefit in this evolving access landscape.