Rare Daily Staff

The Institute for Clinical and Economic Review said its analysis of the comparative clinical effectiveness and value of Vertex Pharmaceuticals cystic fibrosis therapies Symdeko, Orkambi, Kalydeco would need to be discounted as much as 77 percent from their wholesale prices to be cost-effective.

“Our report summarizes the evidence demonstrating that CFTR modulator therapies, paired with best supportive care, provide clinical benefit for patients who have not been treated previously with disease-modifying therapy,” said Dan Ollendorf, ICER’s chief scientific officer. “The current prices for these drugs, however, produce overall costs that are far in excess of those needed to reach commonly cited cost-effectiveness thresholds.”

ICER evaluated the therapies in three distinct populations, based on a number of different types of genetic mutations involved in CF. The indicated therapies were compared to best supportive care in each of the key populations.

ICER’s economic analyses found that, in all populations considered, the cost of the drugs far exceeded commonly accepted thresholds for cost-effectiveness of $100,000-$150,000 per quality-adjusted life year gained. To align the costs of the drugs with the clinical benefit provided to patients, net prices would need to be reduced by 71 percent to 77 percent, ICER said.

The report from the independent non-profit research institute will be the subject of a public meeting May 17 of the organization’s Midwest Comparative Effectiveness Public Advisory Council in St. Louis, but Vertex will likely not be there.

Samantha Ventimiglia, vice president of government affairs and public policy in a letter to ICER President Steven Pearson, called the evaluation a “sham,” and said the organization had reached its conclusion before public hearings at which patients, physicians and the company was scheduled to present their evidence.

“Over the last seven months you have ignored numerous concerns from patients, physicians, the CF Foundation and other advocates concerning your methodology and its inability to evaluate breakthrough orphan drugs for CF,” wrote Ventimiglia, who said further participation in the ICER process by Vertex would be “pointless.” “By intentionally ignoring this evidence, it is clear that your conclusions were agenda-driven and pre-ordained.”

May 7, 2018
Photo: Dan Ollendorf, chief scientific officer of ICER

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