Added therapeutic benefit regarding ESMO-MCBS and the French health technology assessment of drugs granted early access program

Pham, Fiona Y.‐V.; Jacquet, Emmanuelle; Monard, Adrien; Brunel, Liora; Blay, J-Y.; Albin, Nicolas

doi:10.1016/j.annonc.2022.02.002

Cited by 5 publications

(2 citation statements)

References 5 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The scale most widely utilized with HTA applications in cancer is the Magnitude of Clinical Benefit Scale (MCBS) of the European Society for Medical Oncology (ESMO) [ 110 , 111 ]. Validation of MCBS as a tool to harmonize regulatory decisions as well as enabling HTA decisions has been provided across multiple countries, including the UK [ 112 ], Israel [ 113 ], Austria [ 114 ], France [ 115 , 116 ], Switzerland [ 117 ] and Kazakhstan [ 9 ]. MCBS provides explicit scores based on a transparent methodology, helping to discern high- versus low-value medicines.…”

Section: What Could Be Donementioning

confidence: 99%

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

et al. 2022

View full text Add to dashboard Cite

Background: Advances in cancer medicines have resulted in tangible health impacts, but the magnitude of benefits of approved cancer medicines could vary greatly. Health Technology Assessment (HTA) is a multidisciplinary process used to inform resource allocation through a systematic value assessment of health technology. This paper reviews the challenges in conducting HTA for cancer medicines arising from oncology trial designs and uncertainties of safety-efficacy data. Methods: Multiple databases (PubMed, Scopus and Google Scholar) and grey literature (public health agencies and governmental reports) were searched to inform this policy narrative review. Results: A lack of robust efficacy-safety data from clinical trials and other relevant sources of evidence has made HTA for cancer medicines challenging. The approval of cancer medicines through expedited pathways has increased in recent years, in which surrogate endpoints or biomarkers for patient selection have been widely used. Using these surrogate endpoints has created uncertainties in translating surrogate measures into patient-centric clinically (survival and quality of life) and economically (cost-effectiveness and budget impact) meaningful outcomes, with potential effects on diverting scarce health resources to low-value or detrimental interventions. Potential solutions include policy harmonization between regulatory and HTA authorities, commitment to generating robust post-marketing efficacy-safety data, managing uncertainties through risk-sharing agreements, and using value frameworks. Conclusion: A lack of robust efficacy-safety data is a central problem for conducting HTA of cancer medicines, potentially resulting in misinformed resource allocation.

show abstract

Section: What Could Be Donementioning

confidence: 99%

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

et al. 2022

View full text Add to dashboard Cite

show abstract

“…We previously reported a synthesis of the ATB of drugs granted ATUs according to ESMO‐MCBS and HAS assessments, which indicated that ATU was a successful program in giving early drug access to patients in medical need 19 . In continuity with this previous work, the objective of the present study was to assess the ATB of drugs granted the ATU program in oncology over the past 11 years, according to the French HTA criteria (ACB and CAV), the ESMO‐MCBS and the ASCO‐VF.…”

Section: Introductionmentioning

confidence: 99%

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

Pham¹,

Jacquet

Taleb³

et al. 2022

Intl Journal of Cancer

View full text Add to dashboard Cite

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.

show abstract

Les nouveaux dispositifs d’accès précoce aux innovations thérapeutiques

Dory,

Gourieux

2023

Revue des Maladies Respiratoires Actualités

View full text Add to dashboard Cite

Added therapeutic benefit regarding ESMO-MCBS and the French health technology assessment of drugs granted early access program

Cited by 5 publications

References 5 publications

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments

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

Les nouveaux dispositifs d’accès précoce aux innovations thérapeutiques

Contact Info

Product

Resources

About