Rare Daily Staff

The National Institute for Health and Care Excellence, the United Kingdom’s drug value watchdog, needs to use more flexible means for assessing the value of drugs to treat rare diseases if it wants to ensure patient access to innovative new medicines are not blocked by the use of inappropriate value measures, according to a new industry funded report.

The report from MAP BioPharma, a consultant to biopharmaceutical companies on pricing and reimbursement, centers on NICE’s decision to route most orphan drugs through its more restrictive Single Technology Appraisal (STA) program rather than its Highly Specialized Technology (HST) program. Under the STA program, a therapy must demonstrate an incremental cost-effectiveness ratio of between $26,000 and $39,000 (£20,000 and £30,000) compared to $130,000 to $390,000 (£100,000 and £300,000) under the HTA program.

“This report helps to show the systemic failings in our decision-making systems for access to rare disease treatments,” Jayne Spink CEO of Genetic Alliance UK. “This system will continue to fail people living with rare diseases until the fundamental challenges of assessing rare disease treatments are better addressed.”

The report calls on NICE to adapt its STA processes to enable more flexibility. It found that, while NICE can accommodate for the rarity of a disease, to an extent, through its HST appraisal, many rare disease medicines do not qualify for an HST. Furthermore, the STA criteria fail to take into account the nature of rare disease medicines both in terms of the evidence base, unmet need, and cost, according to the report.

The report found that of 24 completed STA reviews of rare disease medicines between 2013 and 2017, only 13 percent were recommended for the full eligible population. That compared with more than two-thirds of non-orphan medicines in the same period.

Half of rare disease medicines were given a restricted recommendation, compared to 21 percent of other medicines.

Only six non-cancer orphan drugs were reviewed under STA program. Of these, only four appraisals were complete, and none were recommended within their full marketing authorization. That compared to more than two-thirds of non-orphan medicines.

“MAP’s research illustrates that the current processes are not fit for purpose and we urge NICE, NHS England, and the Government to look into how they can improve patient access to rare disease medicines,” said Christian Hill, CEO of MAP BioPharma.

Map BioPharma’s steering group financially supported the report. This includes Amicus Therapeutics, AveXis, Bluebird Bio UK, Chiesi, Gilead Sciences, Kyowa Kirin International and Santhera Pharmaceuticals.

Photo: Jayne Spink, CEO of Genetic Alliance UK