Phil McEwan
CEO, HEOR
Rare disease health economics is being transformed with a new approach that manages limited data, assesses broader value and supports fairer funding decisions.
At its heart, health economics is about squeezing the greatest health benefit from constrained healthcare budgets. That’s hard enough for common conditions, but for rare diseases, it’s an entirely different challenge.
Challenges of having limited rare disease data
Rare disease data is limited because the patient cohort is so small. “This creates a tension between maximising benefit for higher patient volumes and delivering equity and fairness for small numbers of patients,” explains Phil McEwan, CEO of HEOR, an independent market access consultancy. “Rare disease symptoms can also mimic more common conditions, leading to long diagnostic delays.” However, despite the absence of data, rare disease treatments are still expected to meet the same evidential bar as common disease treatments.
A payer, such as the National Institute for Health and Care Excellence (NICE), reflects this tension in its thresholds, with standard treatments assessed against a cost-effectiveness threshold of £25,000 to £35,000 per Quality-Adjusted Life Year (QALY). For rare diseases assessed via NICE’s Highly Specialised Technologies route, it rises to £100,000 per QALY.
But the cost of not doing something in healthcare isn’t nothing
New methodology for better decision-making
To make more robust, fundable decisions with limited data, HEOR takes a different approach. Instead of trying to produce one ‘definitive’ number from patchy data, HEOR establishes credible upper and lower bounds. “It’s not necessary to eliminate uncertainty, which is impossible anyway with rare disease data,” says McEwan. The goal is to narrow it enough to support a defensible decision.
The method also groups diseases into archetypes based on shared characteristics. “Rather than saying, ‘Here are all the things we don’t know about the disease,’ we describe the things we do know,” says McEwan.
The consultancy also argues that real value in rare disease treatment development extends beyond direct healthcare costs and QALYs. After all, management (or lack of treatment) of these conditions creates an economic burden for patients, their families and wider society. “We say this a lot,” notes McEwan. “But the cost of not doing something in healthcare isn’t nothing.”
