Jacob Epstein is an Associate Engagement Manager at Prescient, leading Value, Pricing & Access engagements for global pharmaceutical companies, with a particular focus on rare diseases and oncology. He has extensive experience spanning market access strategy and health policy, helping clients navigate complex access challenges across international markets. His work combines rigorous research and stakeholder engagement to translate payer, policy and patient perspectives into practical strategies that support access to innovative medicines.
Patients with rare diseases often have limited licensed treatment options, leading to the use of off-label therapies that may be considered as comparators in value assessment. However, clinical use alone does not necessarily make an off-label treatment an appropriate comparator, and comparator choice can materially influence the assessment of licensed treatments and subsequent patient access. Drawing on expert perspectives and the example of primary biliary cholangitis, this panel will explore the key considerations that should guide comparator selection in rare diseases, including regulatory status, evidence quality, clinical relevance, legal and ethical safeguards, and longer-term access implications. It will also consider potential ways forward to support fair assessment and patient access.