Survival, cost and added therapeutic benefit of drugs granted early access through the French temporary authorization for use program in solid tumors from 2009 to 2019. Issue 8 (7th July 2022)

• Record Type: Journal Article
• Authors: Pham, Fiona Y.‐V.; Jacquet, Emmanuelle; Taleb, Amina; Monard, Adrien; Kerouani‐Lafaye, Ghania; Turcry, Florence; Brunel, Liora; Grudé, Françoise; Yoldjian, Isabelle; Sainte‐Marie, Isabelle; Boudali, Lotfi; Blay, Jean‐Yves; Albin, Nicolas
• Is Part Of: International journal of cancer. Volume 151:Issue 8(2022)
• Page Start: 1345
• Page End: 1354
• Publication Date: 2022-07-07
• Subjects: added therapeutic benefit -- drug costs -- early access program -- health technology assessment -- survival outcomes; Cancer -- Periodicals; Cancer -- Prevention -- Periodicals; 616.994
• DOI: 10.1002/ijc.34129
• Languages: English
• ISSNs: 0020-7136

Abstract

Abstract: Decisions on market authorization (MA) and reimbursement have different durations across countries because of health technology assessment (HTA) procedures and negotiations between manufacturers and national authorities. To overcome this delay, France has implemented a Temporary Authorization for Use (ATU) program that allows early access to drugs before MA, in order to treat patients with unmet medical needs. The objectives of our study were to establish the added therapeutic benefit (ATB) of ATUs for solid tumors and to investigate the correlations between three tools evaluating ATB and survival outcomes and drug costs. Data on ATUs granted from January 2009 to December 2019 to treat solid tumors were analyzed. An assessment of their ATB was conducted using the American Society of Clinical Oncology‐Value Framework (ASCO‐VF), the European Society for Medical Oncology‐Magnitude Clinical Benefit Scale (ESMO‐MCBS) and the French HTA criterion, clinical added value (CAV). The latter score determines reimbursement and national market access. Thirty‐five drugs in 39 indications were granted ATUs. All of them obtained MA and derived a clinical benefit to be reimbursed by the Social Security. Twenty‐eight (71.8%) had CAV compared to preexisting therapies. 24/38 (63.2%) had a 4‐5 ESMO‐MCBS score and 19/33 (57.6%) had an ASCO‐VF score over 45. No correlations were found between cost, PFS, OS, CAV and ASCO‐VF score, while high ESMO‐MCBS scores were correlated to OS. In conclusion, many patients were treated with innovations before MA thanks to ATU, although there are discrepancies between ATB scales, hence the importance of international collaboration in the evaluation of innovative therapies. What's new?: In 1994, France implemented a Temporary Authorization for Use (ATU) program for early drug access before marketing authorization. Since its inception, the program has enabled thousands of cancer patients to be treated with innovative therapies. In our study, ATUs granted for the treatment of solid tumors over the period 2009‐2019 were assessed for added therapeutic benefit (ATB). Of the 39 indications granted ATUs, all obtained marketing authorization and reimbursement. The majority had significant ATB. Analyses further indicate that ATB evaluation tools differ markedly. Thus, while the ATU program is successful, challenges remain for international harmonization of measurements in clinical benefit.