A Survey of Medical Oncologists and Interviews with Payers in the EU5 Hepatocellular carcinoma (HCC), thyroid cancer, gastrointestinal stromal tumor (GIST), and neuroendocrine tumors (NETs) fall into the category of rare cancers as defined by the EMA—diseases with a prevalence of fewer than 5 cases out of a population of 10,000. Efficacious treatments are lacking for many rare or niche cancers, signifying a high level of unmet need and an opportunity for drug developers. Manufacturers can apply for orphan designation for emerging drugs for rare cancers, which if successful, may result in several financial incentives such as ten-year market exclusivity and reduced fees.
Several premium-priced targeted agents have been approved in Europe for HCC, thyroid cancer, GIST, and NETs, and although many have the benefit of orphan drug designation, all are subject to prescribing and/or distribution guidelines and controls impacting uptake. Furthermore, regional variations in drug access, notably in Italy, Spain, and the UK, and delays between EMA drug approval and the completion of country-specific pricing and reimbursement negotiations further impact uptake. Developers of emerging therapies for niche and rare cancers should be mindful of the barriers and levers to market access for such therapies in the cost-constrained EU5.