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Review

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

1
European Institute of Oncology, IRCCS, 20141 Milan, Italy
2
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, USA
3
Department of Pharmaceutical Sciences, Università Del Piemonte Orientale, 28100 Novara, Italy
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Department of Health Products Policy and Standards, World Health Organization, 1211 Geneva, Switzerland
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Medical Oncology Service, Hospital Sírio-Libanês, São Paulo 01308-050, Brazil
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Tata Memorial Centre, Homi Bhabha National Institute, Mumbai 400094, India
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Oncomédica C. A., Caracas 1040, Venezuela
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Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital and Harvard Medical School, Boston, MA 1620, USA
9
Department of Oncology and Hemato-Oncology, University of Milan, 20122 Milan, Italy
*
Author to whom correspondence should be addressed.
Curr. Oncol. 2022, 29(8), 5774-5791; https://doi.org/10.3390/curroncol29080455
Received: 18 June 2022 / Revised: 10 August 2022 / Accepted: 11 August 2022 / Published: 16 August 2022
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. View Full-Text
Keywords: cancer medicines; health technology assessment; value; dis-investments; MCBS; health economics; accelerated approval cancer medicines; health technology assessment; value; dis-investments; MCBS; health economics; accelerated approval
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MDPI and ACS Style

Trapani, D.; Tay-Teo, K.; Tesch, M.E.; Roitberg, F.; Sengar, M.; Altuna, S.C.; Hassett, M.J.; Genazzani, A.A.; Kesselheim, A.S.; Curigliano, G. Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments. Curr. Oncol. 2022, 29, 5774-5791. https://doi.org/10.3390/curroncol29080455

AMA Style

Trapani D, Tay-Teo K, Tesch ME, Roitberg F, Sengar M, Altuna SC, Hassett MJ, Genazzani AA, Kesselheim AS, Curigliano G. Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments. Current Oncology. 2022; 29(8):5774-5791. https://doi.org/10.3390/curroncol29080455

Chicago/Turabian Style

Trapani, Dario, Kiu Tay-Teo, Megan E. Tesch, Felipe Roitberg, Manju Sengar, Sara C. Altuna, Michael J. Hassett, Armando A. Genazzani, Aaron S. Kesselheim, and Giuseppe Curigliano. 2022. "Implications of Oncology Trial Design and Uncertainties in Efficacy-Safety Data on Health Technology Assessments" Current Oncology 29, no. 8: 5774-5791. https://doi.org/10.3390/curroncol29080455

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