An Open Letter to the CF Community

(The following cover letter opened ICER’s published response to the comments we received on our Draft Evidence Report assessing treatments for cystic fibrosis.)

Dear Stakeholders,

ICER thanks each patient, caregiver, and family member who submitted public comments on our draft report on CF modulator therapies. While we are unable to respond to every letter, we would like to acknowledge and reflect upon several key themes we heard.

Many of you shared personal stories about how CF affects all aspects of your lives. Patients and caregivers described how CF robs them being able to spend time with family and friends, pursue educational/professional opportunities, and to plan for the future. Patients also shared how much Trikafta, Symdeko, Orkambi, and Kalydeco have helped them be more active, spend more time with family and friends, and plan for the future in a way that would have been unthinkable just a few years ago. We deeply appreciate the thought and effort that went into these comments, and we have described these important considerations in Chapter 2 of our report.

In our report Trikafta earned an “A,” ICER’s highest and rarest rating for comparative clinical effectiveness, indicating that we have high certainty that the treatment delivers substantial health benefits. Even though our report’s findings suggest that the price of Trikafta is set too high in relation to its clinical benefits, we certainly do not call for non-coverage as the answer. We also feel confident that no insurer in the United States will even briefly entertain the option of non-covering Trikafta.

The experience with our last report may serve as a reassurance. To our knowledge no insurer, including New York Medicaid, who used our report as part of their identification of a price target for negotiation over the price of Orkambi, even whispered about possible non-coverage. In fact, New York Medicaid made explicit that in no way would its negotiation include any possibility of erecting increased barriers to access in any way. We too, at our public meeting, started by asserting that payers will cover these drugs (see video, 20:00-21:00).

When treatments are priced too high, they contribute to higher insurance premiums, copayments and restrictions on access. Studies have shown that as insurance costs increase patients may delay care, forego care entirely, or even drop their health insurance. This leads to increased suffering and mortality. We heard stories of CF patients in these exact circumstances in the public comments on our draft report.

To be entirely clear, in all cases we support actions to achieve fair prices that maintain the ability of patients to get the treatments they will benefit from. Our broader view is that when we as a nation give a company a monopoly on a treatment and, instead of “wrestling” with them over coverage, tacitly agree that coverage WILL be provided because we want all patients to benefit, we need some mechanism to suggest an upper limit to the price that a company feels it can charge. It is precisely because access to Trikafta is not and should not be viewed as negotiable that we believe it is essential to use evidence of how much it benefits patients as a guide for its price. When the price of any service throughout the health system is way out of proportion to its ability to improve lives, it can actually cause more unseen harm to other unknown patients — some with CF, some with other diseases – who can no longer afford their health care.

In closing, we are grateful to the patients who engaged with us — not just through this recent public comment period, but throughout the past several months. Few patient communities are as committed and as passionate as the CF community. While we may continue to disagree on certain aspects of this review, you have made our work more accurate, and more reflective of patients’ lived experiences. Conversations about value and health care always carry a certain level of tension, but we hope you have seen that we have taken a good-faith effort to be evidence-based, patient-focused, and transparent. And we also hope these efforts will help the US achieve a fairer price for these innovative CF therapies, without putting access at risk.

Sincerely,

Steven D. Pearson, MD, MSC
President, Institute for Clinical and Economic Review

David M. Rind, MD
Chief Medical Officer, Institute for Clinical and Economic Review