Ultra-rare, ultra-slow

How NICE’s £100,000 threshold for ultra-Orphan drugs puts patient access at risk

NHS England and NICE have grappled for years with how to evaluate ultra-Orphan drugs, but neither has applied a cost-effectiveness analysis in funding decisions - until now. The proposed £100,000 threshold would not factor into NICE’s recommendations. But it would provide the NHS an out from automatic funding for expensive ultra-Orphan drugs, even if they have a positive recommendation from NICE.

In cases where NHS decided not to automatically fund one of these drugs, the joint NHS/NICE proposal could delay patient access to ultra-Orphan drugs by a year or more and could theoretically remove funding for some of the most expensive drugs.

The delay would result from passing ultra-Orphan drugs on to the Specialised Services commissioning prioritization process after NICE’s Highly Specialised Technologies (HST) process has completed.

Specialised Services cover the treatment of rare diseases or procurement of rarely needed interventions, along with most specialized cancer services, including drugs funded through the Cancer Drugs Fund.

Under this process, drugs are given a priority ranking based on clinical effectiveness and cost per patient. The drugs are then funded in order of their priority rankings - until the budget for new specialized therapies runs out.

The process is normally done once a year, so a decision can take a year or more.

In addition, one rare disease group and three industry representatives told BioCentury the prioritization process isn’t adequate to evaluate ultra-Orphan drugs because it doesn’t factor into its decision-making process the limited data sets and small populations that are typical of ultra-rare diseases.

They are concerned that these limitations could result in low priority rankings for ultra-Orphan drugs, effectively cutting off funding when budgets are tight.

“Imposing that threshold would actually result in few, if any, patients suffering from ultra-rare diseases having access to those products,” Heidi Wagner, Alexion Pharmaceuticals Inc.’s SVP of

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