Interviews with patients
Interviews with patients provide the most in-depth understanding of their experiences and perspectives. Through direct conversations, researchers can gain qualitative insights into the journey, including the challenges they face, their treatment decision-making processes, and their preferences for specific outcomes or treatment options. Interviews allow a deep exploration of patient perspectives and can uncover valuable insights that surveys alone may not capture. Prescription data or a survey can indicate that patients are not using a newly approved product, for instance, but qualitative feedback from individual patients is vital for understanding why, and therefore how, any uptake issues can best be addressed.
As markets become more competitive, value arguments need to become more specific. Conversations in early development will become increasingly valuable for informing a therapy’s target population and competitive positioning and setting the new product apart. For instance, one sponsor assumed that a novel oral formulation of an orphan drug would command the vast majority of market share when approved. However, through conversations with patients, we learned that improvements in efficacy and safety would be needed to attain the forecast sales. As a result, the development program was refined to build a data package that could demonstrate meaningful clinical improvement for patients, in addition to the improved route of administration.
Surveys
Surveys can be conducted with many patients or clinicians to obtain quantitative information on patient demographics, treatment patterns, and outcomes, with different goals for each group. Patients have a greater sense of the day-to-day disease burden and factors impacting their quality of life, while clinicians are generally more oriented toward data-driven endpoints.
Once developers have a strong qualitative understanding of patients’ needs and expectations, surveys can strengthen patient population categorization, assumptions about patient preferences, and commercial forecasts. We often conduct patient surveys to refine revenue forecasts for different geographies and sites of care, particularly when patients of academic centers and community practices have different treatment goals.
Interviews with key opinion leader clinicians
Interviews with key opinion leader clinicians are valuable for understanding how the patient population may evolve with pipeline therapies and novel diagnostics. These clinicians are keenly interested in the possible evolution of treatments with future drug approvals. So, conversations with them are useful at every stage of product development for understanding where a future product may fit in a rapidly evolving landscape, how to recruit patients for trials, and the commercial implications of recent developments.
Patient advocacy groups
Patient advocacy groups play a crucial role in representing the interests and needs of patients to a range of stakeholders, including manufacturers and regulators. Engaging with these groups can provide valuable insights into the patient experience and help shape research and development efforts. Patient advocacy groups (PAGs) have a wealth of knowledge about specific conditions and can provide a collective voice for patients.
Of course, some PAGs, particularly in rare diseases, have limited bandwidth and many interested biopharma partners. In those situations, it is important to enter into conversations with a compelling value argument that can be iteratively refined with the PAG. Because competition for trial patients can be fierce, building early excitement in the patient community will help uptake and expedite early and later-stage clinical trials with more effective patient recruitment. Many rare disease PAGs are best positioned to improve access to trial-eligible patient populations and build support for regulatory access and commercial use.
PAGs also play a strong role in enabling market access, such as for HIV/AIDS, where state government funding is critical to ensuring patient access. For a developer of HIV therapies, we interviewed heads of national PAGs to understand the needs and perspectives of patients. This feedback was crucial in shaping our understanding of the minimal product attributes patients wanted in their treatment and prophylaxis regimens. In particular, as HIV therapies became more effective and patient management became chronic, the longer-term toxicity and side effect profile were more important than we had realized. This information had significant implications for the optimal combinations of products in our recommended regimens. The interviews were also critically helpful in ensuring that novel combination products were aligned with local funding, helping ensure access throughout the patient community.