The Financial Toxicity of FDA-Drugs Approved for Malignant Hematology

Abstract

Background: Many Food and Drug Administration (FDA) drugs (FDADs) are available now for patients with malignant hematology (MH). Still,the high prices of FDADs for MH affect the care of these patients and our health care system. Here, we estimated one monthly supply price (OMSP) for FDADs for MH approved between 2011 and 2021. Also, we reported the total cost (TC) of FDADs by multiplying OMSP times the assumed duration of therapy based on the available overall survival (OS) (OMSP x OS (months)). Methods: We performed a retrospective review of clinical trials (CTs) published from January 2011 to May 2021 that led to FDA approval of hematological malignancy drugs (Leukemias, lymphomas, Multiple Myeloma (MM), Myelodysplastic Syndrome (MDS), and Myeloproliferative Neoplasm (MPN)). The drugs investigated were selected using FDA Databases, 'Oncology (Cancer) / Hematologic Malignancies Approval Notifications' and 'Novel Drug Approvals'. CTs were also reviewed for demographic data on ClinicalTrials.gov (CT.gov) and Primary Literature on drug approval. FDADs were divided into disease groups: acute myeloid leukemia (AML), chronic myeloid leukemia (CML), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), MDS+MPN, MM, and lymphoma. We compared prices between drug.com and UpToDate using the lowest price for each drug (the percentage change was low 0.18). We calculated the median of OMSP for each group, compared the median of OMSP between oral (PO), subcutaneous (SQ), and intravenous (IV) FDADs, and calculated TC for each group. For non-reached OS, we used 60 months to calculate TC. Results: Sixty-one FDADs were identified; 9,4,4,7 and 4 for AML, CML, ALL, CLL, and MDS+MPN, respectively. For MM and lymphoma, we identified 11 and 22, FDADs, respectively. The median of PO, SQ, and IV FDADs were 29, 4, and 30, respectively (some drugs were approved for more than one group). The median of OMSP for AML, CML, ALL, CLL, and MDS+MPN were $30,923 [range, $5,989-36,099], $19,343 [range, $20,834-22,570], $75,839 [range, $48,917-93,660] (excluding chimeric antigen receptor-T cell (CAR-T) therapy), $15,263 [range, $3,532- 23,091], and $15,284 [range, $8,060- 24,157], respectively. The median of OMSP for MM and lymphoma excluding CAR-T were $20,514 [range, $2,090-35,276 ] and $18,434 [range, $6,834-34,351], respectively. The median of OMSP for PO, SQ, and IV FDADs were $18,291 [range, $2,628-31,686], $18,370 [range, $6,934-22,583] and $24,691 [range, $2,090-93,660] (excluding CAR-T cell therapy), respectively. All FDADs for ALL patients were IV. The median OMSP for FDADs approved from (2011 to 2016) and (2016 to 2021) were $22,233 (range, $8,300-23,091) and $24,517 (range, $2,352-86,253), respectively. The median TC for AML, CML, ALL, CLL, and MDS+MPN were $351,327 (range, $196,380-791,041), $1,160,597 (range, $1,097,482-1,354,993), $547,091 (range, $244,587-$985,915), $971,160 (range, $564,720-3,456,000), and $1,114,038 (range, $255,502-1,449,447), respectively. OS was not reached for all CML drugs, in 5/7 (71%) of CLL drugs and 12/61 (34%) of all FDADs. The median TC for MM and lymphoma were $607,031 [range, $18,813-1,743,706 ] and $884,900 [range, $183,817-9,116,360], respectively. Excluding CAR-T drugs, The highest OMSP was for ALL but not TC due to the short median OS (10 months) (Figure 1). There were 5 CAR-T products approved between August 2017 and March 2021 (tisagenlecleucel, axicabtagene ciloleucel, lisocabtagene maraleucel, brexucabtagene autoleucel and idecabtagene vicleucel). The median cost was $432,055 (range, $373,000-475,000), and the estimated OMSP was $21,635 (range,$6,216-39,583). The median household monthly income was $6,558 in 2020 in the United States (US). In 2020, 8.6% of US population (28 million) did not have helath insurance at any point during the year. There are currently no generic drug products of these FDADs available in the US. Conclusion: The cost-effectiveness of CAR-T products is relatively similar to other FDADs. Patients with malignant hematologic disorders are affected by treatment-related financial harm. Although the costs used for this analyses may be modulated by many factors where the actual cost may not be what is reported in the sources used, the financial impact is still significant for many patients. There is a need for strategies to lower the costs. The value of drugs should be considered in formulating treatment guidelines. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal