Oncology

Innovativeness of drugs, value to be measured on the basis of patients' clinical outcomes

Between the new Aifa criteria and the implementation of Ema's Health Technology Assessment regulation: the proposals of a multidisciplinary working group

by Carmine Pinto *, Giuseppe Curigliano **, Giovanni Pappagallo ***

9 June 2026

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2' min read

Translated by AI

Versione italiana

Key points

Evaluation of innovativeness and new criteria
The proposals

2' min read

Translated by AI

Versione italiana

The recognition of drug innovativeness is one of the central tools through which the Italian Medicines Agency (Aifa) guides early access to therapies, promotes clinical value and governs the allocation of public resources. With Determination No. 519 of 31 March 2017, the Agency defined a multidimensional assessment model based on three pivotal criteria: therapeutic need, added therapeutic value and quality of evidence.

In the period 2017-2024, Aifa evaluated more than 160 oncohaematological therapeutic indications, recognising full innovativeness in 36% of cases. The retrospective analysis of these decisions shows that the added therapeutic value was the main determinant of the evaluation outcome, even more than the therapeutic need considered in isolation.

Evaluation of innovativeness and new criteria

In this context, the recent evolution of the Aifa institutional set-up - with the establishment of a (single) Scientific and Economic Commission (CSE) and the publication of updated criteria for the recognition of innovativeness - together with the implementation of the HTA Regulation at European level, offer the opportunity for a critical and prospective reflection. In particular, on the parameters used to define 'added therapeutic advantage' in relation to endpoints not based on overall survival (OS) in the treatment of early-stage malignancies.

In order to analyse this field, a multidisciplinary working group was set up - including experts in oncology clinics, research methodology, regulatory procedures and health economics - in which, in addition to the authors, Francesco De Lorenzo, Maria Carmela Piccirillo and Entela Xoxi also participated, in the context of the Project 'Oncology Early Asset Consensus Pathway with NGT (Nominal Group Technique) Methodology', with the noncontributing contribution of Astrazeneca.

The proposals

Integrating the different disciplinary perspectives, the Multidisciplinary Working Group reached a shared consensus on the following summarising elements:

- The value of a treatment must be measured by the clinical outcomes relevant to the patient, regardless of the degree of pharmacological novelty of the underlying mechanism.

- In recent years, more sophisticated measurement tools have expanded the range of endpoints that can be used, many of which are pathology-specific and informative in the early stages of disease.

- These include, for example: (i) minimal residual disease assessed by liquid biopsy; (ii) invasive disease-free survival in breast cancer; (iii) radiographic progression-free survival in prostate cancer.

- These endpoints, although not validated as surrogates for overall survival, have a direct impact on clinical decisions, follow-up treatment planning and care pathway organisation.

- The quantification of these parameters on the overall therapeutic strategy, their economic and social impact, represents valuable information to support the decisions of the CSE in an integrated evaluation logic.

- The importance of quality of life and patient-reported outcomes (Patient-Reported Outcomes, PROs) must be considered structural components of the assessment of therapeutic benefit and not ancillary elements.

- The full implementation of the HTA Regulation-Ema, with the introduction of the Joint Clinical Assessment and the consideration of ethical, social and economic domains at national level, offers a concrete opportunity for a systematic valorisation of these dimensions.

* Medical Oncology Director, Comprehensive Cancer Centre, Ausl-Irccs of Reggio Emilia
** Department of Oncology and Haemato-Oncology, University of Milan; European Institute of Oncology, Irccs Milan
***Scientific Coordinator School of Clinical Research Methodology, Irccs Sacro Cuore Don Calabria, Negrar di Valpolicella

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