Presentation (CTI)

OBJECTIVES: Limited market incentives, combined with the atypical study designs often required in research and development (R&D) for ultra-orphan therapeutics, can threaten the translation of scientific breakthroughs into effective therapies. In the UK, more than 3.5 million people live with a rare disease but only 5% of rare diseases have an effective and approved treatment on the market. This research seeks to develop a pricing framework within which payers can reward successful translation of breakthrough science to treat ultra-orphan conditions through selective adoption of a risk-seeking attitude, and to explore its potential impact on R&D investment decisions.
METHODS: The research will comprise three phases. Study 1: In-depth interviews will be conducted to ascertain the decision domains and product attributes that could drive different risk attitudes by payers. Study 2: A discrete choice experiment study will be carried out to quantify the weights and trade-offs between decision domains and product attributes in order to understand payer risk attitudes (averse, neutral, seeking) under different scenarios. Study 3: A cost-effectiveness model will be developed for an exemplar advanced therapeutic and medicinal product targeting an ultra-orphan condition to illustrate the potential of risk-based pricing on evidentiary requirements and/or price premia.
RESULTS: In conventional health technology assessment (HTA) and pricing processes, payers are often assumed to adopt a risk-neutral attitude toward investment decisions. This research presents a novel framework to incentivise high-risk high-reward investments where payers can adopt a risk-seeking attitude for appropriate technologies in HTA and pricing processes. We aim to provide decision makers with a toolkit that can support R&D investment in high-value technologies, improve patient access to ultra-orphan therapeutics, and help address the gap in effective treatments for rare diseases.
CONCLUSIONS: N/A