Skip pattern approach toward the early access of innovative anticancer drugs

G Apolone, A Ardizzoni, A Biondi, A Bortolami, C Cardone, C M Ciniselli, P Conte, C Crippa, F de Braud, M Duca, S Gori, G Gritti, A Inno, R Luksch, F Lussana, M Maio, G Pasello, F Perrone, A Rambaldi, G RossiD Signorelli, G Soverini, M Valente, P Verderio, G Buzzetti

Research output: Contribution to journalReview articlepeer-review


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.

Original languageEnglish
Pages (from-to)100227
JournalESMO Open
Issue number4
Publication statusPublished - Aug 2021


  • Antineoplastic Agents/therapeutic use
  • Government Agencies
  • Humans
  • Italy
  • Neoplasms/drug therapy


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