With ongoing changes in the external healthcare environment, holistic, patient-focused, end-to-end leadership is required to unlock the potential of the integrated evidence generation planning process. Medical Affairs professionals are in a suitable position to lead these efforts; however, close and strategic collaboration with cross-functional and regional colleagues is essential for navigating a constantly evolving landscape. Overall, there is a need to ensure that the patient voice is captured early in product development—ideally as early as development of the target product profile (which serves as a foundation for the clinical development plan and IEGP). This ultimately increases the likelihood of acceptance by, for example, HTA bodies, and creates greater understanding of real-world situations, given the direct applicability in clinical practice.

The EU joint HTA regulation, which began implementation in a staged fashion by therapy area in 2025, streamlines and centralises the HTA process by consolidating the clinical assessment stage into a regional JCA process. To enable and support this process, pharmaceutical companies can engage in Joint Scientific Consultations (JSCs) to obtain optional, non-binding scientific advice, for example to inform clinical trial design or the scope of assessment [13]. The JCA scope uses an initial PICO framework that is subsequently updated and refined in line with the feedback obtained from the optional JSC and EU Member State Coordination Group/HTA bodies [14]. Fulfilling the PICOs that form part of the assessment should be a key focus of the IEGP; this requires earlier and closer internal alignment of stakeholders in a pharmaceutical company and increased external engagement to reflect and validate the needs of all EU countries.

Medical Affairs professionals can collaborate closely with the Market Access team that may be involved in developing PICO framework content, and the interpretation of goals and results. Medical Affairs can equally play a key role in identifying potential evidence gaps by comparing PICO scoping results with the evidence generation activities mapped in the IEGP. The JCA dossier must be submitted before the Committee for Medicinal Products for Human Use (CHMP) opinion stage, which is another reason why implementing an earlier, more efficient IEGP process is beneficial [14]. As pharmaceutical companies update existing IEGP processes (or implement new processes) to respond to these changes, Medical Affairs will need to strengthen collaborations with Market Access and Regulatory Affairs colleagues.

Pricing negotiations are becoming increasingly reliant on optimal evidence generation, and Medical Affairs can lead the response to this evolution in collaboration with Market Access and R&D colleagues. The USA introduced the Inflation Reduction Act (IRA) in 2022, which mandates that drug manufacturers pay a rebate to the government if the prices of certain drugs (e.g., those covered by Medicare Part D) increase faster than the rate of inflation [15, 16]. For the drugs that cost the most under the Medicare programme, the IRA also authorises Medicare to negotiate drug prices directly with pharmaceutical companies [15, 16]. Medical Affairs can play an important role in navigating this change by understanding the short- and long-term impacts of the IRA and leading the response from an evidence generation standpoint. Evidence generation strategies should consider the potential future implications of the IRA on any pipeline products, and a Medical Affairs-led IEGP process is well placed to build on these considerations and ensure that evidence answers appropriate research questions.

A strong IEGP generating tailored, robust RWE will support manufacturers during pricing negotiations across geographies. This in turn will support reimbursement outcomes in countries that use international reference pricing approaches. Medical Affairs has the expertise to not only ensure that the appropriate RWE is generated at the right time to support the justification of prices, but to also lead the design and execution of such studies in a patient-centred manner, using novel and innovative approaches.