This summary of our daily insights from ISPOR 2024 tackles some of these questions: specifically, our expert perspectives on building a patient-centric value story, navigating competing evidence requirements across markets, amplifying and scaling data value, and early pipeline planning for the new realities of the market.

 

 

Patient centricity: a crucial approach for access, value, and evidence

The landscape of commercial development and market access is constantly evolving, with patient engagement playing a crucial role in shaping these trends. Here are the key take-aways discussed at ISPOR 2024 with the Avalere Health team:

• Growing recognition of patient-centric approaches: Biopharmaceutical manufacturers are increasingly acknowledging the importance of patients as consumers and recognizing the need to understand patients’ own definitions of value and valuable treatments. This recognition strengthens the entire product development cycle, with patients contributing to study design.
• Influence on regulatory and HTA activities: Regulators and HTA agencies are increasingly incorporating patient insights into their assessments. Patient preference data from rigorously conducted studies provides decision-makers with a wider pool of views on outcomes that patients value. The US FDA’s new guidance on gathering patient input underscores the growing significance of patient involvement in regulatory processes, as does the EUnetHTA 2021 guidance.
• Transformation of patient groups into expert influencers: Patients are leveraging their exposure to regulatory and HTA activities to drive policy changes, challenge industry practices, and find solutions for accessing high-cost treatments. Initiatives such as the European Patients’ Academy on Therapeutic Innovation (EUPATI) are educating and training patient advocates to engage in drug development and market access.

Given these trends, it’s essential to consider the following:

• Early engagement with patients: Engage with patient advocacy groups early in product development to ensure their perspectives are integrated throughout the process.
• Long-term partnerships: Establish long-term partnerships between manufacturers and patient/advocacy groups to successfully incorporate the patient voice into product development.
• Understanding patient preferences: Invest in understanding patient preferences through rigorous studies to support regulatory submissions effectively.

Embracing a patient-centric approach, collaborating closely with patients and advocacy groups, and recognizing the transformative influence of patient insights will be critical for success in navigating the evolving global landscape and making science accessible to every patient.

 

 

 

Early pipeline: new realities of the market

Value-based decision-making early in the clinical development cycle is crucial given the substantial investment required to conduct clinical trials on the path to commercialization. Scientific breakthroughs are propelling an increase in regulatory approvals, driving the need for evidence that determines the value of a drug to ensure successful patient access. Approaches that evaluate the market access potential can also inform the strategy for pipeline assets.

Here are the key take-aways discussed at ISPOR 2024 with the Avalere Health team:

• Real-world evidence is playing an increasing role in validating trial outcomes, providing context for economic modeling assumptions, including natural history of disease, and contextualizing reimbursement decisions.
• Budget scrutiny over newly launched products may be leading to complex managed entry agreements and a growing focus on achieving better value for money.
• Demand is growing for qualitative but also increasingly quantitative economic insights from rapid early economic modeling and evaluation to contribute critical insights.

Recommendations include:

• Right-size your approach to market access to ensure informed insights during early clinical development.
• Prioritize resources for meaningful input into clinical development and strategic decision-making.
• Incorporate critical HEOR insights early and often for more informed decisions on pipeline asset with tools like hēRo3

 

 

 

 

Evidence: navigating competing requirements across markets

The evidence and value landscape has expanded in complexity, leading to increased and diverging requirements for evidence strategies. Avalere Health experts continued the conversation started during our Monday theater presentation, “No longer business as usual: establishing value in an uncertain and dynamic landscape,” today at #ISPORAnnual. These recent value developments don’t occur in isolation, furthering the complexities facing life sciences companies.

 

• IRA implementation: The IRA substantially changed the HEOR landscape, requiring shifting strategies to ensure preparation for new evidence needs. As the first negotiation cycle wraps up, there are opportunities to leverage learnings for the future. Separately, what does the redesign of the Part D benefit mean for value? How does this differ by plan and therapeutic area?
• PDABs and other state drug price control initiatives: PDABs may evaluate the affordability of drugs earlier in the product lifecycle than CMS during negotiation, leading to questions about the impact of these earlier assessments on evidence planning. Further, PDAB methodologies vary from state to state, where certain approaches could present greater portfolio risks.
• Joint clinical assessments: With JCA implementation starting in 2025, manufacturers need to prepare for a rapidly evolving pricing and reimbursement landscape in the EU. Early pipeline planning, evidence generation strategies, incorporation of tighter timelines, launch sequencing, and market prioritization are some of the most top-of-mind concerns stakeholders must consider.
• Upcoming US election and potential changes to drug pricing legislation: Potential changes tied to 2024 US election outcomes will have very different implications for the drug pricing political landscape, leading to questions about the future of the IRA and other drug pricing legislation. How will outcomes differ by election outcome scenario? What are the implications for ex-US markets?
• Value framework application and implications: Value assessors continue to leverage different metrics and explore the application of new methods (eg, GRACE, lifecycle value assessment), leading to questions around the implications for life sciences companies and how this should shape strategies.

 

 

 

Data assets: amplifying and scaling data value

 

As the breadth, depth, and content of data in healthcare are continuously growing, the industry needs to make data actionable and leverage it to make intelligent business decisions. As ISPOR 2024 concludes, Avalere Health experts are wrapping up the conversations with one last, but not least, focus area: amplifying and scaling data value.

 

Here are the key take-aways discussed today at #ISPORAnnual with the Avalere Health team:

 

• Health equity and social determinants of health: Analysis of data related to social and behavioral factors, as well as through a health equity lens, is a key strategic approach that can help organizations minimize incomplete decision-making based on partial evidence. By integrating data on health equity and social determinants of health with clinical and claims data, stakeholders gain a more comprehensive understanding of patient populations and the patient journey while developing targeted interventions and allocating resources more effectively.
• Economic burden of disease: Calculating the total burden of illness or determining the economic impact of a disease at various levels—whether patient, caregiver, healthcare system, and/or societal—may be challenging. However, a robust dataset and well-designed research strategy can offer useful insights and context for patients, caregivers, payers, health technology assessment agencies, healthcare providers, policymakers, manufacturers, researchers, and society.
• Data ecosystems: An optimal data ecosystem can improve decision-making processes within an organization by centralizing and standardizing data from different sources. The application of artificial intelligence and machine learning to healthcare may help align the healthcare systems to quality care and drive positive patient and societal outcomes.
• Novel approaches to data generation: As real-world evidence continues to drive the research discussions and decisions in healthcare, stakeholders need to keep up with the pace of innovation to increase clinical research efficiency and develop targeted strategies to enhance value stories and provide compelling evidence for stakeholder engagement. Full understanding of novel real-world data sources and innovative technologies to gather, process, and analyze them creates a need for a more robust approach to creating real-world evidence.

 

Exploring ways to incorporate data into daily functions, investing in new ways to provide healthcare to patients, and incorporating real-world evidence into business decisions can help the industry deliver better healthcare to those who need it the most.