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.

Questions Answered in This Report:

  • The level of reimbursement of approved premium-priced therapies for HCC, thyroid cancer, GIST, and NETs can have a significant impact on patient share. How does reimbursement for these agents vary between countries? How do national, regional, and local healthcare authorities regulate the prescribing of these agents?

  • Developers need to be aware of the current drivers and barriers to prescribing for niche and rare cancers in order to maximize uptake. What are the main cost-related/clinical constraints to the uptake of premium-priced therapies for HCC, thyroid, GIST, and NETs? How do surveyed oncologists across the EU5 anticipate that prescribing of current and emerging therapies for HCC, thyroid, GIST, and NETs will evolve in light of anticipated regulatory approvals, reimbursement restrictions, and budgetary constraints?

  • Recently approved and emerging agents could change the prescribing landscape in these rare oncology indications. What are payers’ reactions to recently approved therapies for HCC, thyroid cancer, GIST, and NETs, and what are their reactions to emerging therapies in development? To what degree will prescribing constraints present a hurdle for future targeted therapies in the EU5? How do surveyed oncologists across the EU5 anticipate that prescribing patterns will change with the launch of new therapies?

  • Meeting acceptable requirements in clinical trials will be critical to securing favorable reimbursement and physician acceptance of novel therapies. What clinical benefit do surveyed physicians expect emerging therapies for HCC, thyroid cancer, GIST, and NETs will need to demonstrate to secure favorable reimbursement? What do they believe to be an appropriate clinical trial comparator and primary end point for emerging therapies?


Decision Resources Group's European Physician & Payer Forum report Navigating Reimbursement and Maximizing Market Access for Niche and Rare Solid Tumors in the EU5: Physician Prescribing Perspectives and Payer Insights explores the evolving reimbursement and market access landscape for therapies for HCC, thyroid cancer, GIST, and NETs; the dynamics affecting prescribing of key drugs for these niche cancers in a cost-constrained climate; and the primary challenges facing emerging therapies aspiring to significantly penetrate niche cancer markets.

Markets covered: France, Germany, Italy, Spain, United Kingdom.

Primary research: Online survey of 196 medical oncologists, interviews with 15 European payers.

Author(s): Gemma McConnell, Ph.D.