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Published on: December 9, 2015
Skip pattern approach toward the early access of innovative anticancer drugs
G Apolone1, A Ardizzoni2, A Biondi3
1Scientific Directorate, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
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.
Insights
This study developed a method to prioritize new cancer drugs for early access (EA). The algorithm uses unmet medical need, added benefit, and quality of evidence to objectively identify valuable treatments for regulatory approval.
Area of Science:
- Oncology
- Pharmacoeconomics
- Regulatory Science
Background:
- Optimizing tools for accelerated regulatory approval of innovative anticancer treatments is crucial.
- Assessing the clinical value of new therapeutic options is essential for early access (EA) prioritization.
- This project aimed to establish a reliable pathway for evaluating innovative cancer drugs.
Purpose of the Study:
- To propose a methodological pathway for assessing the clinical value of innovative anticancer therapies.
- To objectively identify drugs deserving early access (EA) priority for solid and hematological cancers.
- To support regulatory authorities in drug approval processes.
Main Methods:
- A comprehensive review of 21 European Public Assessment Reports for innovative drugs.
- Formulation of an algorithm by an expert panel using Unmet Medical Need (UMN), Added Benefit (AB), and Quality of Evidence (QE) criteria.
- Sequential combination of UMN, AB, and QE using the skip pattern approach (SPA) to determine EA status.
Main Results:
- Applying SPA to solid cancers: 36% (5/14) achieved EA status; enhancing UMN criteria increased this to 64% (9/14).
- Two drugs were excluded based on inadequate Added Benefit (AB) scores.
- Three drugs were excluded due to insufficient Quality of Evidence (QE) scores.
- For hematological cancers, only UMN criteria were deemed adequate.
Conclusions:
- The developed model offers a reliable tool for stakeholders and regulatory agencies to objectively assess the value of new cancer treatments.
- The model can aid in a more comprehensive and objective definition of treatment value for early access decisions.
- Further evaluation is needed to assess the generalizability of this model in other national contexts.
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