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Making tafamidis cost-effective for TTR amyloid cardiomyopathy may require 93% price reduction
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.
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.
Perspective
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The findings confirm that even with a highly effective drug like tafamidis, it will fail traditional cost-effectiveness metrics when it is priced as high as $225,000 per year.
The drug was priced as if transthyretin amyloid (ATTR) cardiomyopathy was an exceptionally rare disease, and quite simply, it isn’t. For truly rare diseases, it’s understandable that drugs may need to be priced beyond traditional “cost-effectiveness” numbers or it wouldn’t be financially viable for pharmaceutical companies to invest the money/resources into their development. But the overall impact on the health care system in such cases is small because the number of people with the disease is very small — and for the unfortunate people with the disease, the opportunity to receive effective treatments can be life-changing.
The problem here is that ATTR cardiomyopathy not only isn’t rare, it’s actually downright common if you are looking at older populations with HF with preserved ejection fraction. Plus, we now have an easy/inexpensive screening test available to make the diagnosis, by sending off a couple of simple laboratory tests and ordering an inexpensive/noninvasive nuclear scan, so the sky is the limit for the number of people who will be eligible to take the drug.
On an individual patient level, because most patients eligible for tafamidis are older and insured through Medicare, they have the “donut hole” and the 5% drug copay, so their out-of-pocket costs approach $15,000 a year. While there are assistance programs available for some patients with the lowest incomes, for patients who have a moderate income but who are by no means wealthy, they are caught in a really bad situation.
What needs to be done is pretty straightforward — Pfizer could lower the cost. There’s really no way to justify the current cost when the number of new diagnoses and number of new prescriptions are skyrocketing so fast, and again, the only justification for the $225,000 a year cost was that the disease was truly rare. So as that’s clearly not the case, Pfizer needs to significantly drop the cost. My hope is that with the ongoing pressure from physicians, patients and patient advocacy groups, that they will listen. When many of the authors of the landmark study that led to tafamidis’ approval — including the lead author of this study — are publishing a manuscript stating that it would require a greater than 90% price reduction to make it cost-effective, it’s not a great look.