Skip pattern approach toward the early access of innovative anticancer drugs

ESMO Open

article

G. Apolone¹  A. Ardizzoni²  A. Biondi³  A. Bortolami⁴  C. Cardone⁵  C.M. Ciniselli⁶  P. Conte⁷  C. Crippa⁸  F. de Braud⁹  M. Duca⁹  S. Gori^1,10  G. Gritti^1,11  A. Inno^1,10  R. Luksch^1,12  F. Lussana^1,11  M. Maio^1,13  G. Pasello^1,14  F. Perrone^1,16  A. Rambaldi^1,17  G. Rossi^1,19  D. Signorelli⁹  G. Soverini^1,19  M. Valente^1,13  P. Verderio⁶  G. Buzzetti^2,21 

[1] Scientific Directorate, Fondazione IRCCS Istituto Nazionale dei Tumori;Department of Medical Oncology, IRCCS Azienda Ospedaliero-Universitaria Policlinico Sant"Orsola-Malpighi;Department of Pediatrics, University of Milano Bicocca—Fondazione MBBM/Ospedale San Gerardo;Rete Oncologica Veneta, Istituto Oncologico Veneto;Experimental Clinical Abdominal Oncology, Istituto Nazionale Tumori-IRCCS-Fondazione G. Pascale;Unit of Bioinformatics and Biostatistics, Fondazione IRCCS Istituto Nazionale dei Tumori;Istituto Oncologico Veneto;Department of Hemathology;Department of Medical Oncology & Haematology, Fondazione IRCCS Istituto Nazionale dei Tumori;Department of Oncology, IRCCS Sacro Cuore Don Calabria Hospital of Negrar;Hematology and Bone Marrow Transplantation Unit;Department of Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori;Center for Immuno-Oncology, Medical Oncology and Immunotherapy, University Hospital of Siena;Department of Surgery, Oncology and Gastroenterology, University of Padova;Medical Oncology 2, Istituto Oncologico Veneto IRCCS;Clinical Trials Unit, National Cancer Institute of Naples;Department of Oncology-Hematology, University of Milan;Department of Oncology and Hematology;Deparment of Hematology ASST Spedali Civili di Brescia;Niguarda Cancer Center-Grande Ospedale Metropolitano Niguarda;Dephaforum

关键词: early access priority;    innovative anticancer treatments;    unmet medical need;    added benefit;    quality of evidence;   

DOI  :  10.1016/j.esmoop.2021.100227

学科分类：社会科学、人文和艺术（综合）

来源: BMJ Publishing Group

【 摘 要 】

Background With the rapid development of innovative anticancer treatments, the optimization of tools able to accelerate the access of new drugs to the market by the regulatory authority is a major issue. The aim of the project was to propose a reliable methodological pathway for the assessment of clinical value of new therapeutic innovative options, to objectively identify drugs which deserve early access (EA) priority for solid and possibly in other cancer scenarios, such as the hematological ones.Materials and methods After a comprehensive review of the European Public Assessment Report of 21 drugs, to which innovation had previously been attributed by the Italian Medicines Agency (Agenzia Italiana del Farmaco, AIFA), an expert panel formulated an algorithm for the balanced use of three parameters: Unmet Medical Need (UMN) according to AIFA criteria, Added Benefit (AB) according to the European Society for Medical Oncology's Magnitude of Clinical Benefit Scale (ESMO-MCBS) criteria and Quality of Evidence (QE) assessed by the Grades of Recommendation Assessment, Development and Evaluation (GRADE) method. By sequentially combining the above indicators, a final priority status (i.e. EA or not) was obtained using the skip pattern approach (SPA).Results By applying the SPA to the non-curative setting in solid cancers, the EA status was obtained by 5 out of 14 investigated drugs (36%); by enhancing the role of some categories of the UMN, additional 4 drugs, for a total of 9 (64%), reached the EA status: 2 and 3 drugs were excluded for not achieving an adequate score according to AB and QE criteria, respectively. For hematology cancer, only the UMN criteria were found to be adequate.Conclusions The use of this model may represent a reliable tool for assessment available to the various stakeholders involved in the EA process and may help regulatory agencies in a more comprehensive and objective definition of new treatments' value in these contexts. Its generalizability in other national contexts needs further evaluation.

【 授权许可】

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