Reflections on the 2024 World Evidence, Pricing and Access Congress

Costello Medical were delighted to attend the World Evidence, Pricing and Access (EPA) Congress in Amsterdam last month, with members from our Market Access and Rare Diseases teams in attendance.

The conference felt like a natural progression from the discussions held at ISPOR Europe 2023, with continued conversations around the upcoming launch of European Union (EU) Joint Clinical Assessment (JCA) and the potential uses of artificial intelligence (AI) in Health Economics and Outcomes Research (HEOR) and market access, but with an additional and notable focus on the importance of multi-stakeholder collaboration.

EU JCA – Are we ready for it?

Our key takeaway from JCA discussions at ISPOR EU (summarised here) was that a lot of uncertainty remains about the practicalities of the JCA process and outputs. Although we are now five months closer to the introduction of JCA, the consensus at EPA was the same – there are still more questions than answers:

• JCA was supposed to strengthen the industry’s competitiveness in Europe, improve patient access to novel therapies, and reduce inefficiencies associated with Health Technology Assessment (HTA) processes – yet, industry is already experiencing increased bureaucracy associated with JCA consultations and preparations, so will the JCA process actually deliver on its promises?
• JCA represents a conscious decoupling of clinical and economic evidence, where the clinical value of a product is assessed through the JCA process, and economic assessment remains at the country level. This should result in efficiencies at the national level and some countries (e.g. Italy) are already adapting their HTA approach, assuming clinical assessment will not be needed at a national level. Will all be keen to adapt, or will workload for industry expand if other countries do not follow Italy’s lead?
• Not only will HTA processes need to change, companies may need to change, too. Large pharmaceutical companies may be able to easily adapt by creating cross-functional HTA task-forces to ensure alignment on the new JCA processes and responsibilities. Small and medium enterprises (SMEs) may have the flexibility in their internal resourcing to put together similar cross-functional teams, but will they struggle to adapt to the increased workload associated with the new JCA process given that they often do not have affiliates in every relevant country?
• Timelines are tight and potentially unrealistic! Companies will have just 90 days after receipt of the final scope to submit the JCA dossier, with little flexibility. These tight timelines raise the question – what will data requirements for JCA dossiers, as well as best practices for efficiently preparing these documents, look like?

So, are we ready for it? The consensus at EPA was, simply put – no, we’re not. There are still so many open questions, with one speaker noting it feels we are “crossing a bridge we are still constructing”. Training and education for patients, industry and HTA assessors will be key for ensuring stakeholders are ready to adapt to the new regulation. Now, more than ever, it will be important to foster a collaborative mindset within industry – internally, between local and global teams, to align on EU- and country-level opportunities and challenges, as well as externally, between industry and payers, working together to ensure the success of JCA.

Unlocking market access with Artificial Intelligence (AI) and innovation

The potential of AI technologies to make impactful advances in market access and Health Economics and Outcomes Research (HEOR) emerged as a central topic of discussion across a variety of sessions. Common themes and potential applications of AI highlighted in the conference included:

• Leveraging AI in real-world evidence (RWE), such as synthetic data development for the accelerated monitoring of drug performance post-launch, as well as utilising AI to identify target patient populations for products and/or study enrolment.
• Support with the identification of data gaps and/or data limitations within an evidence base.
• Automation of literature review stages and (network) meta-analysis, such as the process of data extraction.
• Automated regulatory and/or reimbursement dossier development utilising generative AI, for example supporting with EU JCA dossier development to ease the burden of the tight dossier development timelines.

Next steps for AI in market access should include agency-initiated pilot projects centred on AI usage, accuracy and validity, enabling agencies to support and advise the pharmaceutical industry on appropriate AI use within HEOR. Such steps are crucial to fully gauge AI's capabilities and determine whether it has the potential to enact transformative change within?the market access field. Extending our view beyond the pharmaceutical industry to gain valuable insights from successful AI adoption in other sectors will also be needed to fully capitalise on opportunities for employing AI within pharmaceutical market access.

A case for authentic collaboration and joint efforts

Whilst AI certainly has a role to play in increasing efficiency in day-to-day work, it’s clear that relationship building and collaboration will still be essential to successful market access:

• Kirsty Hoyle (Metabolic Support UK) delivered an impactful presentation outlining some key areas where industry could improve on their collaborations with patient organisations, focusing on the importance of authenticity (transparency as to why patient groups are being involved in a particular activity), and genuine collaboration (viewing partnerships with patient advocacy groups [PAGs] as defined projects with set timelines, and providing funding for PAG time and expertise). Although industry are now more regularly engaging patients in evidence generation and market access activities, the quality and openness of these relationships is key to delivering a meaningful outcome.
• The need for increased collaboration between internal stakeholders was a common theme across presentations, and particularly the need for internal market access teams to collaborate across countries to define new processes and responsibilities in preparation for the challenges of JCA in Europe.
• The value of early collaboration on integrated evidence generation planning (IEGP) was also emphasised: Elena Panitti (Global Director, Evidence Valuation and Enablement at Novartis) explained that whilst front-loading evidence generation through early IEGP represents a financial risk, early and collaborative planning benefits all internal stakeholders and functions, through alignment on the end goals of evidence generation activities. She also emphasised the need for behavioural and process changes in order to establish IEGP as a more routine and streamlined internal process.

How should health be valued?

A key theme across the conference was the importance of expanding the current definition of value in health. HTA bodies currently measure the value of a new product under three buckets, all integral to decision making:

• Clinical value in the form of efficacy outcomes
• Humanistic value in the form of health-related quality of life (HRQoL), depending on HTA framework/country archetype
• Economic value in the form of budget impact and/or cost-effectiveness, depending on HTA framework/country archetype

Payers tend to give minimal consideration to other elements like patient preference and health equity, aspects that could arguably play a significant role in augmenting the overall value of global health.?A significant unresolved challenge in transforming the perception of value in health lies in the objective quantification of these other elements. An initial suggestion made during the conference was to consider modifying the 'quality of life adjusted life year (QALY)' metric into 'individual preference adjusted life years', thus capturing broader aspects of value in the metric than just quality of life. This is a key area requiring further discussion and research, in order to fuel change, and to acknowledge the wider benefits of treatments.

Sonia Shaw, Eve McArthur and Isabelle Newell are employees at Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners. If you would like any further information on the themes presented above, please do not hesitate to contact Isabelle Newell.

Costello Medical provides scientific support to the healthcare industry in the analysis, interpretation and communication of clinical and health economic data. Due to growing demand across an increasing range of service offerings and geographies, Costello Medical has grown organically since its foundation in 2008 to a team of over 350 based in Cambridge, London, Manchester, Bristol, Boston, Singapore and Shanghai. Alongside our evolving technical and creative capabilities, we remain committed to our core values of high-quality scientific work coupled with exceptional customer service at competitive and transparent prices. For more information on our services, please visit our website.