Focusing on orphan and ultra-orphan drugs, this event will provide a fresh perspective on one of the industry’s hottest areas for market & patient access. Communicating value and cost-effectiveness with regulators, payers, HTA bodies and other key stakeholders, in such niche markets, requires innovation & a clearly defined process to ensure the message is achieved.
This segment is currently challenging normal market and patient access models due to its niche nature and the shift towards highly patient centric models of treatment, data collection, and cost-effectiveness measurement. Payer priorities also must be understood as this remains central to the viability of many orphan products, as some payers seem more willing to encourage and reward innovation, yet others remain hesitant.
From the pharma industry’s perspective, stepping into this high-potential, niche area and achieving commercial goals, requires superb strategic and operational decision-making. Identifying patients with orphan diseases and building a viable R&D and commercial plan in such rare fields is challenging, costly and risky. Unique approaches towards product and portfolio development, clinical studies, value demonstration, communication and marketing strategy are key elements for success in such a niche area, in order to achieve a return on investment.
The event will be focused purely on orphan therapies and will guarantee all participants access to today’s most relevant case studies and views which directly affect their future business plans and can assist them to maximise market and patient acce