NOVEMBER 20258EUROPEIN MY OPINIONtBy Marcelo Vaz, Vice President, Medical Services, TMC Pharma Services Ltd.TAKING AN INQUIRER-CENTRIC APPROACH TO RARE DISEASE MEDICAL INFORMATIONhe rare disease medical information landscape is markedly different from that of prevalent conditions. Small patient populations, limited clinical trial data and minimal details about the disease's natural history, prognosis and related treatments all contribute to significant gaps in evidence. Despite their positive impact, novel therapies that alter the natural history of a disease, such as extending survival in conditions once deemed fatal in early childhood, may further exacerbate the knowledge gaps as both clinicians and patients navigate uncharted territory. Sparse medical literature, clinical expertise concentrated in just a handful of reference centres globally and insufficiently documented patient experiences also compound the challenge.This scarcity of information makes it difficult for newly diagnosed patients to find accessible information, while physicians and even specialised treatment centres may struggle to adjust disease management. In this context, the medical information function evolves from a mere repository of product details into a critical source of support across the disease spectrum. Biotech and pharmaceutical companies must, therefore, address inquiries Marcelo Vaz is a physician with a doctorate in Pulmonology from São Paulo University. His experience spans academia, clinical practice, and the pharmaceutical industry. Marcelo has held leadership roles in medical affairs and clinical research functions and supported regulatory teams across Brazil, Latin America, the US, the UK, and Europe. He is currently Vice President of Medical Services at TMC, supporting drug development, regulatory submissions, and post-marketing activities.ranging from basic product questions to complex concerns about treatment outcomes and disease progression. The challenge is how to deliver this information in a way that ensures strict compliance with regulatory standards while also providing a humanised, accessible explanation--bridging the gap between clinical language and everyday understanding.Understanding Diverse Information Needs An `inquirer-centric' approach recognises the diversity of information seekers and understands that every inquiry is unique. As such, responses must be tailored to the individual asking the question--whether the inquiry comes from a caregiver, a healthcare provider or a patient. Key to this approach is the differentiation among stakeholder groups. For example, a key opinion leader (KOL) working at a reference centre requires emerging data, research methodologies and nuanced clinical interpretations. In contrast, a general practitioner might benefit more from practical advice on symptom recognition, patient referral pathways and product administration support. Meanwhile, patients and caregivers need clear, accessible language that emphasises day-to-day management and supports their emotional needs. Even though the core scientific information remains consistent, how it is framed and delivered must be adapted accordingly. While the benefits of an inquirer-centric approach are clear, its implementation is not without challenges. Marcelo Vaz
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