Fund Orkambi based on individuals’ response

Re: “Sky-high prices don’t mean a drug is effective,” comment, Nov. 23, and “Politics, price, process: Why Orkambi is worth it,” comment, Nov. 29. These two commentaries offer contrasting perspectives on the potential worth of Orkambi in patients with cystic fibrosis.

In his commentary, Alan Cassels focuses on pooled data from patients enrolled in controlled studies to challenge both the clinical effectiveness and costeffectiveness of Orkambi. Undoubtedly, pooled data from well-controlled studies represent the gold standard for medical evidence. However, solely focusing on this can miss the possibility that a smaller subset of patients might respond differently than is reflected in the pooled data.

John Wallenburg draws attention to this important consideration by pointing out that one in four cystic fibrosis patients treated with Orkambi meets the generally accepted criterion for a clinically significant improvement in lung function, and one in eight shows a much greater benefit.

Orkambi trials have shown that any beneficial responses occur rapidly — within the first few weeks for lung function, and perhaps slightly longer for other measures of health status.

Considering all of the above, isn’t there an obvious way forward — based on the response of individual patients — during this interim period while awaiting further clinical evidence and the outcome of price negotiation?

Offering a four- to eight-week initial trial of Orkambi to qualifying patients, with clear criteria for assessing their individual responses and therefore for determining whether treatment should continue, would strike an appropriate balance between evidence-based and compassion-based decision-making. Patients currently receiving Orkambi, who have already demonstrated an individual response that meets such criteria, should receive continued treatment. Rennie Heel Saanich