February 19, 2020
5 min read

Making tafamidis cost-effective for TTR amyloid cardiomyopathy may require 93% price reduction

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Dhruv S. Kazi

Although tafamidis can provide substantial clinical benefit for patients with transthyretin amyloid cardiomyopathy, the drug exceeds cost-effectiveness thresholds at its current list price of $225,000 per year, according to an analysis published in Circulation.

To make tafamidis cost-effective at $100,000 per quality-adjusted life year, the price would need to be reduced by 92.6% from $225,000 per year to $16,563 per year, according to the researchers.

“The Orphan Drug Act provides pharmaceutical companies substantial incentives to develop novel therapies for rare diseases,” Dhruv S. Kazi, MD, MSc, MS, associate director of the Richard A. and Susan F. Smith Center for Outcomes Research and director of the cardiac critical care unit at Beth Israel Deaconess Medical Center, who led the study, told Healio. “Pfizer has relied on these taxpayer-funded incentives to accelerate the development and approval of tafamidis. It is now leveraging its position as the first approved therapy for ATTR cardiomyopathy to charge an incredibly high price for a drug that has the potential to improve quality of life and survival among patients with this devastating illness. If patients, particularly Medicare beneficiaries, have trouble getting the drug because their copays are very high and we suspect the copays will be in the thousands of dollars, then the Orphan Drug Act will have failed to achieve its stated purpose of improving access to novel, effective therapies. And at some point the market will have to signal that we are not prepared to pay these outlandish prices for drugs even if they’re effective. That’s what it will take to keep prices in check in the long run.”

ATTR-ACT trial data

Researchers developed a Markov model using data from the ATTR-ACT trial of patients with wild-type or variant transthyretin amyloid cardiomyopathy.

As Healio previously reported, the ATTR-ACT trial found that tafamidis (Vyndamax and Vyndaqel; FoldRx/Pfizer) reduced the risk for all-cause mortality and CV-related hospitalizations in patients with hereditary and wild-type transthyretin amyloid cardiomyopathy.

The model reproduced quality of life, survival and CV hospitalization rates at 30 months using data from the ATTR-ACT trial. Researchers used a price of $225,000 per year for the drug based on costs in September 2019. The primary outcome was the incremental cost-effectiveness ratio of the drug compared with usual care regarding cost per life-year and cost per QALY.

Estimated price reduction needed to make tafamidis cost-effective.

Compared with usual care, it was estimated that tafamidis would add 1.29 QALYs (95% uncertainty interval, 0.47-1.75) at an incremental cost of $1,135,000 (95% uncertainty interval, 872,000-1,377,000). This resulted in an incremental cost-effectiveness ratio of $880,000 per QALY gained (95% uncertainty interval, 697,000-1,564,000).

Tafamidis was cost-effective in 0% of 10,000 probabilistic simulations when the threshold of $100,000 per QALY gained and the current drug price were taken into consideration. Results of the analysis were sensitive with assumptions on the long-term effectiveness of tafamidis.


Annual heath care spending would increase by $32.3 billion if all eligible patients with transthyretin amyloid cardiomyopathy (with a conservative estimate of 120,000 eligible patients) were treated in the U.S.

Conversations with patients

“An important message here is that we have to be willing to regularly have the tough conversations with our patients about out-of-pocket costs,” Kazi said in an interview. “Even our fully insured patients may not be able to afford the drug because of prohibitively high out-of-pocket costs. Patients are often uncomfortable bringing up this issue with their physicians, so it’s our job to discuss this with them when we initiate the drug and periodically throughout the year. Anyone’s who’s worked with Medicare Part D knows that a lot of these copayments vary depending on the month of the year, depending on where you are with regard to your catastrophic insurance vs. your deducible, so this isn’t a one-and-done conversation where we talk about costs just at the time of initiation.”

In an email sent to Healio, Pfizer wrote: “We do not believe cost-effectiveness analyses using QALY alone are an appropriate framework for evaluating rare disease medicines. The cost/QALY methodology is flawed for tafamidis and orphan medicines, as it fails to account for non-health and societal benefits, and it is biased against elderly populations suffering from serious diseases who have a shorter life expectancy and less time to benefit from treatment. Lack of cost-effectiveness is a common challenge in rare diseases, especially when compared against best supportive care.” – by Darlene Dobkowski

For more information:

Dhruv S. Kazi, MD, MSc, MS, can be reached at dkazi@bidmc.harvard.edu; Twitter: @kardiologykazi.

Disclosures: Kazi reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.