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

Masri, Ahmad; Chen, Hongya; Wong, Chan‐Yuan; Fischer, K; Karam, Chafic; Gellad, Walid F.; Heitner, Stephen B.

doi:10.1001/jamacardio.2020.1738

Cited by 24 publications

(11 citation statements)

References 3 publications

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“…Currently in the US, the cost of tafamidis treatment is covered through various methods, including financial assistance programs that patients may qualify for. 47 Future studies may investigate the impact that varying modes of coverage for tafamidis costs and adverse events associated with tafamidis have on adherence to the medication.…”

Section: Discussionmentioning

confidence: 99%

Baseline Characteristics and Secondary Medication Adherence Patterns Among Patients Receiving Tafamidis Prescriptions: A Retrospective Analysis Using a National Specialty Pharmacy Dispensing Database

Roy¹,

Peterson²,

Marchant³

et al. 2022

PPA

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Introduction Transthyretin amyloid cardiomyopathy (ATTR-CM) is a serious, underrecognized condition, which leads to heart failure and early mortality if left untreated. Until recently, heart transplantation was the only treatment for ATTR-CM. Regulatory approval of tafamidis transformed treatment for patients. In the phase 3 Transthyretin Amyloidosis Cardiomyopathy Clinical Trial (ATTR-ACT), which established the safety and efficacy of tafamidis, medication adherence was high with 97.2% of patients taking ≥80% of scheduled doses. Evidence of real-world adherence to cardiology drugs demonstrates low adherence and suboptimal outcomes; however, real-world adherence to tafamidis has not been investigated. The main objective of this study was to describe adherence patterns of patients filling tafamidis in the Symphony Health database. Methods This retrospective analysis of the Symphony Health Solutions claims database used secondary adherence measures, including modified medication possession ratio (MPRm), days between fills adherence rate, and compliance rate, to assess adherence patterns of 2020 patients filling tafamidis free acid 61-mg capsules or tafamidis meglumine 4x20-mg capsules from June 1, 2019 to August 31, 2020. Results Patients receiving a tafamidis formulation had characteristics consistent with the expected patient population; 71.6% were aged 75–84 years, 83.2% were male, and the highest proportion resided in the Northeast region (30.5%) of the United States. Adherence for tafamidis was high, as 75% to 100% of the patients across subgroups met or exceeded the commonly defined adherence threshold of 80%. Median number of refills ordered and received was six refills per patient. Most patients received refills with no gap (n=1633) or a gap <30 days (n=1267/1317 patients). Adherence was high across follow-up time, sex, and age subgroups. Adherence varied by geographic region, with the Northeast being significantly higher than the Midwest (mean MPRm 94.41% vs 88.21%, p=0.0007). Conclusion These results provide evidence that real-world adherence to tafamidis in patients with ATTR-CM is high.

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Section: Discussionmentioning

confidence: 99%

Baseline Characteristics and Secondary Medication Adherence Patterns Among Patients Receiving Tafamidis Prescriptions: A Retrospective Analysis Using a National Specialty Pharmacy Dispensing Database

Roy¹,

Peterson²,

Marchant³

et al. 2022

PPA

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“…There has been considerable debate regarding the costs of treatments for ATTR-CA, and particularly of tafamidis [76][77][78] , recently labeled as "the most expensive cardiac medication in history" [79] . In the words of experts, "unreasonable pricing decisions have been made for this pharmaceutical, considerably above typically accepted cost-effectiveness thresholds" [76] .…”

Section: Cost-effectiveness Of Diagnosing and Treating Attr-ca Later ...mentioning

confidence: 99%

Cardiac amyloidosis: a changing epidemiology with open challenges

Canepa¹,

Vianello²,

Porcari³

et al. 2022

Vessel Plus

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Cardiac amyloidosis (CA) is increasingly diagnosed due to the advancements made in diagnostics and therapeutics in the last decades, particularly in the field of transthyretin-related CA. Studies that have used bone scintigraphy for screening at-risk conditions have shown that about one out of ten patients with heart failure with preserved ejection fraction (HFpEF), aortic stenosis undergoing valve replacement, or hypertrophic cardiomyopathy (HCM) diagnosed later in life might have an underlying or concomitant CA. At the same time, the epidemiology of these conditions is also rapidly evolving. HFpEF has become the leading form of heart failure, and HFpEF patients are increasingly cared for in non-cardiology settings due to their older age and substantial burden of comorbidities. Aortic stenosis is increasingly treated percutaneously at earlier stages of the disease, determining a significant gain in survival. Hypertrophic cardiomyopathy is nowadays mostly diagnosed in middle-aged adults with near-normal life expectancy, with a greater chance of misdiagnosing CA as HCM or of an overlap between the two conditions. In all these contexts, the therapeutic and prognostic implications of diagnosing CA will have to be further investigated. Meanwhile, the diagnostic workup of patients with suspected CA should always be completed with the systematic exclusion of a plasma cell dyscrasia, the acquisition of tomographic imaging at bone scintigraphy, and the completion of genetic testing for transthyretin-related forms.

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“…Their high prices at market entry (and, frequently, high out-of-pocket costs for patients) restrict access and undermine the stated purpose of the Orphan Drug Act to expedite access to effective treatments. The lack of competition leads to substantial negotiating power for manufacturers upon approval; rebates and discounts are often small (5%) compared with other brand name drugs (20–40%) [ 16 •, 31 ]. Finally, as with the case of transthyretin cardiomyopathy, the frequency of diagnosis may increase considerably after a treatment is available, so the targeted disease may not be “rare” after all [ 30 ].…”

Section: What Are Orphan Drugs and Why Are They So Expensive?mentioning

confidence: 99%

The Price of Progress: Cost, Access, and Adoption of Novel Cardiovascular Drugs in Clinical Practice

2021

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Purpose of Review The launch of new effective and safe cardiovascular drugs has produced large gains in health outcomes for several cardiovascular conditions. But this innovation comes at the cost of rapidly increasing pharmaceutical spending and high out-of-pocket costs. Recent Findings In the USA, manufacturers are able to set prices according to what the market will bear rather than value to patients or society, with a complicated system of discounts and rebates obscuring the final price borne by payors. Some of these costs are passed on to patients in the form of co-payments or co-insurance, making these effective but high-cost medications unaffordable for many patients. Orphan drugs developed to treat rare diseases—for which manufactures are presented substantial financial and regulatory benefits—are particularly problematic, as they typically enter the market at very high prices compared with drugs for other indications. Summary Systematic cost-effectiveness analyses from the healthcare sector or societal perspectives can help identify the value-based price of a medication at market entry as well as later in the lifecycle of the drug when more data on effectiveness and safety becomes available. Despite bipartisan support, legislative progress on drug pricing has been slow. Clinicians should know the cost of the drugs they prescribe frequently, use generics where feasible, and regularly discuss out-of-pocket costs with patients to pre-empt cost-related non-adherence.

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

Abstract: 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.

Cited by 24 publications

References 3 publications

Baseline Characteristics and Secondary Medication Adherence Patterns Among Patients Receiving Tafamidis Prescriptions: A Retrospective Analysis Using a National Specialty Pharmacy Dispensing Database

Baseline Characteristics and Secondary Medication Adherence Patterns Among Patients Receiving Tafamidis Prescriptions: A Retrospective Analysis Using a National Specialty Pharmacy Dispensing Database

Cardiac amyloidosis: a changing epidemiology with open challenges

The Price of Progress: Cost, Access, and Adoption of Novel Cardiovascular Drugs in Clinical Practice

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