Regulatory Focus™ > News Articles > NICE to Assume Review of High-cost Orphan Drug Products

NICE to Assume Review of High-cost Orphan Drug Products

Posted 23 July 2012 | By Alexander Gaffney, RAC 

The UK's national health technology assessment (HTA) body, the National Institutes for Health and Clinical Excellence (NICE), is set to start assessing what it refers to as the "very high cost [of] drugs for people who suffer with rare conditions" under the new Health and Social Care Act of 2012.

Under the plan, the agency-which assesses a product's value relative to its cost, societal impact and benefit to patients-would assume the responsibilities of conducting similar assessments on drugs for orphan conditions as it currently conducts on regular drug products. The assessments are currently conducted by the Advisory Group for National Specialized Services (AGNSS).

Previously, ANGSS operated under the assumption that "given the small number of patients or procedures involves and the very high level of clinical expertise required to provide such treatments," such assessments were best conducted by a specialized national commissioning body.

As part of its new responsibilities, NICE said it will be developing "interim methods for the first few drug assessments and will take forward a consultation exercise in 2013/14 to ensure the process put in place is robust, transparent and consistent."

The agency said it does not expect to entirely re-invent the work of AGNSS, which Health Minister Lord Howe called "an important strand of work."

"Our decision to give this work to NICE from April 2013 means that there will be a robust, independent and transparent assessment of these drugs, Howe explained. "In taking this work over from AGNSS, NICE will wish to build on the decision-making framework that AGNSS has developed to ensure that the needs of people with rare and very rare conditions are properly considered."

Orphan Drug Regulations Strain State Payors

In Europe, orphan medical products refer to drugs intended for treating a condition affecting either fewer than one in every 2,000 persons in Europe or impacting a serious disease or condition for which there is no currently marketed treatment. While many manufacturers have been developing products for orphan conditions in recent years-partially the result of flagging pipelines and partially the result of attractive market exclusivity provisions in European law-the resulting products have in some cases been vastly more expensive than drugs marketed for non-orphan indications.

A product highlighted in Pharmalot in 2010, for instance, is similar to a US case involving KV Pharmaceutical's Makena. Like KV Pharmaceutical, UK manufacturer BioMarin got a recently-acquired drug, Firdapse, approved for an orphan indication, As with Makena, the drug had been on the market for years without a license. Once approved by the UK's Medicines and Healthcare products Regulatory Agency (MHRA) under an orphan indication, the drug's price skyrocketed from $1,600 a year to more than $120,000 a year. The condition for which the drug is now indicated, Lambert-Eaton Myasthenic Syndrome (LEMS), affects just 150 people per year in the UK.

While the cost of such drugs in the US would likely be a matter for insurers, state Medicaid agencies, Medicare or manufacturer-administered patient assistance programs, the UK's state-run health service means such high costs are borne exclusively by taxpayers.

NICE's Chairman Michael Rawlins said the agency will be looking to establish a "robust and transparent process for making decision about these highly specialized drugs."

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NICE - NICE to assess high cost drugs for rare conditions

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