PHARMACEUTICAL · NEUROSCIENCE Case study · Recruitment & qualitative research Recruiting the experts behind clinically meaningful change in Alzheimer’s agitation MedPanel sourced and screened a specialist panel of physicians and professional caregivers for a peer-reviewed qualitative study on the Cohen-Mansfield Agitation Inventory (CMAI). Agitation is one of the most burdensome
Why Patient Advocacy Partnerships Matter in Rare Disease Research For pharmaceutical and biotechnology companies, the success of any rare disease program—from clinical development to commercial launch—is inextricably linked to the patient community. Indeed, Patient Advocacy Groups (PAGs) are not merely stakeholders; they are the gatekeepers of access, the custodians of
The Role of CROs in Rare Disease Clinical Research In rare disease research, Contract Research Organizations (CROs) play a pivotal role in the clinical development landscape. Moreover, they provide the infrastructure, expertise, and operational support necessary to conduct complex clinical trials. As a result, sponsors can reach small, geographically dispersed
Uncategorized — May 6, 2026 What Is Rare Disease Market Research? Rare disease market research systematically gathers clinical, economic, and patient-centered evidence from verified rare disease specialists, patients, and caregivers. Teams use these insights to shape drug development, regulatory strategy, market access, and commercial planning. For a program overview, see
Introduction Physician recruitment for market research is one of the most critical and challenging aspects of healthcare insights. Physicians are the most analyzed group in healthcare market research and, at the same time, the most difficult to recruit. They have the highest incentive demands, the lowest response rates, and the
Introduction You scoped the study carefully. Your incidence estimate came from a published research paper. In addition, your medical affairs team reviewed the screener. As a result, the fieldwork kicked off on schedule. However, three weeks later you have completed only 4 of 30 target interviews — and you suspect
Introduction Recruiting rare disease patients for market research sounds straightforward until you try it. A condition affecting fewer than 200,000 people nationally may have no organized patient community, unreliable diagnosis rates, and a population scattered across dozens of specialist centers — none of which makes for an easy screener call.
HEOR models are only as credible as the inputs behind them. In rare diseases, those inputs are frequently incomplete: natural history is poorly characterized, endpoints are inconsistent, and small, fragmented patient populations amplify uncertainty in both clinical and economic parameters (Grand et
al., 2024; Pearson et al., 2018).
Real-world evidence (RWE) underpins modern health economics and outcomes research (HEOR), especially when traditional clinical trials are infeasible in rare diseases. Small patient populations, varying symptoms, and uncertain etiology can limit trial insight and complicate endpoint selection.
100 rare disease treaters in the US and the UK shared their biggest challenges in treating and managing rare disease patients.