Initial Experience Prescribing Commercial Tafamidis, the Most Expensive Cardiac Medication in History

Abstract

This economic evaluation study examines the authors’ experience of prescribing tafamidis in terms of their cost and the insurance status of patients.

Tafamidis is a stabilizer of the transthyretin (TTR) protein tetramer, which was recently shown to reduce all-cause mortality and cardiovascular hospitalizations.¹ Tafamidis was approved by the US Food and Drug Administration on May 3, 2019, for the treating TTR amyloid cardiomyopathy² and has a list price of $225 000 per annum.³ A recent study showed that tafamidis exceeds conventional cost-effectiveness thresholds.⁴ In this article, we describe our early experience in prescribing commercial tafamidis. All patients with an established diagnosis of TTR amyloid cardiomyopathy who presented to the Oregon Health & Science University Multidisciplinary Amyloidosis Program and received a prescription for commercial tafamidis were included in the analysis. We did not intentionally exclude women, but all of our consecutive cohort were men. Approval and a waiver of consent because of minimal risk for participants were obtained from the institutional review board of Oregon Health & Science University.

From May through November 2019, 50 consecutive patients (mean [SD] age, 76 [8] years) were prescribed tafamidis and 43 patients (86%) successfully obtained the drug. Only 1 patient (2%) did not have prescription insurance, while 38 patients (76%) had Medicare Part D, 6 (12%) had private insurance, 2 (4%) had Veterans Affairs insurance, and 3 (6%) had other types. Of the 7 (14%) who did not obtain tafamidis, 3 could not afford the out-of-pocket cost, 2 declined further attempts at drug procurement, 1 died before receiving tafamidis, and 1 elected to enroll in a research study. The mean (SD) cost of a 30-day supply of tafamidis was $23 485 ($2). All prescriptions required prior authorization (3 patients [6%] required a prior authorization appeal). Prior to financial assistance, the median and mean (SD) 30-day out-of-pocket costs of tafamidis were $1909 (range, $250-$3144) and $3082 ($5216), respectively (insurers covered a mean [SD] 89% [17%] of the total tafamidis cost). Fifteen patients (30%) qualified for copayment assistance from a foundation and an additional 13 (26%) received financial assistance from the manufacturer (Table). All patients who qualified for financial assistance paid $0, while the median and mean (SD) 30-day out-of-pocket costs of tafamidis for patients without financial assistance were $250 (range, $39-$1763) and $1683 ($858), respectively (Figure). The median time from prescribing to mailing tafamidis was 26 (range, 12-78) days.

Table. Characteristics of Patient Assistance Programs Active During the Study Period.

Eligibility criteria	Pfizer Vyndalinka	PAN Foundation	HealthWell Foundation	TAF
Disease covered under program	Yes	Yes	Yes	Yes
Medication has to be covered by insurance	Case by caseb	Yes	Yes	Yes
Medicare only	NA	Yes	No	No
Income falls at or below federal poverty level, %c	500	500	400-500	Varies (665 as of December 2019)
US citizen or permanent resident	Yes	No	NA	Yes
Must receive treatment in the US	Yes	Yes	Yes	Yes
Assistance amount (per year), $	Free medication	7800	8000	Varies
Approved period	Up to 1 y	Up to 1 y	Up to 1 y	Up to 1 y
Renewal	Yes	Varies depends on funding	Varies depends on funding	Varies depends on funding

Abbreviations: NA, not applicable; PAN, Patient Access Foundation; TAF, The Assistance Fund.

^aPfizer has 2 options: an assistance program or a copay card.

^bBased on patients’ experience, an appeal resulting in successful assistance from Pfizer is possible.

^cFederal poverty level for 2019 is $12 490 for 1 person and $16 910 for 2 people.

Figure. Tafamidis Out-of-Pocket Costs.

Distribution of tafamidis out-of-pocket costs for all patients before any assistance programs were applied to and after 28 patients (56%) received financial assistance.

Our initial experience prescribing tafamidis demonstrates that the current system depends heavily on copayment assistance programs. Regulations instituted in 2005 by the US Office of the Inspector General and the US Department of Health and Human Services aimed to ensure compliance of the patient assistance programs with the Anti-Kickback Statute.⁵ The most pertinent regulation to patients is that the assistance should be based on reasonable and consistent measures of financial need, requiring certain thresholds to be put in place under which patients may qualify for assistance. There are many elderly individuals who do not qualify for assistance based on these thresholds but have other preexisting competing financial commitments that prohibit them being able to afford the costly copayments. In addition to these eligibility stipulations, the funds in these nonprofit foundations are liable to run out. We have experienced periods during which programs have closed temporarily, causing tremendous uncertainty for patients. In our experience, some of the patients who were initially denied manufacturer assistance had success with an additional level of appeal. In these cases, patients directly appealed to the manufacturer with more granular explanations of unaccounted financial obligations and hardship that may not be captured on an income tax report. Finally, we have an integrated multidisciplinary amyloidosis program, a specialty pharmacy, and a dedicated pharmacist who spent an average of 1 hour per patient to ensure they can afford tafamidis. As such, our experience might not be easily applicable to other health care settings.