Using GRADE methodology to assess innovation of new medicinal products in Italy

Abstract

Aim: In April 2017 the Italian Medicine Agency (AIFA) developed new criteria to grant any new medicinal product with an innovative designation. The aim of this study is to describe this new model and how it works. Methods: A retrospective descriptive analysis was performed on the results of the assessment process of innovativeness of new medicinal products (or therapeutic indications) based on the AIFA's new innovation criteria (therapeutic need, added therapeutic value and quality of clinical evidence through GRADE methodology) carried out between April 2017 and February 2019 and made publicly available on the AIFA website starting from January 2018. Results: A total of 37 full reports (22 for oncological indications) explaining the rationale for the AIFA's decision is made publicly available on the agency's website. A total of 12 therapeutic indications (5 oncological) were evaluated as fully innovative, 13 indications (11 oncological) were evaluated as conditionally innovative, while 12 indications (6 oncological) as non-innovative. Conclusion:The new AIFA innovation criteria resulted in a much more flexible and transparent model to define and assess what constitutes a therapeutic innovation. In particular, the choice of AIFA to use the GRADE methodology to evaluate the quality of clinical evidence within a process of drug innovativeness assessment is essential for the early identification of the discrepancy between the need for patients of a rapid access to innovative therapies and the available clinical data needed to make decisions on drug innovativeness.

Key drivers of innovativeness appraisal for medicines: the Italian experience after the adoption of the new ranking system

“…The choice of GRADE methodology was aimed at improving the transparency and reproducibility of the appraisal process; this structured and flexible methodological tool provides a systematic approach in the assessment and is meant to minimise biases and improve consistency of the decisions. 5 The innovativeness is appraised per indication, and the innovativeness status lasts 3 years. The appraisal model represents a common framework for all indications, even if safeguard clauses are provided for rare indications where the quality of the evidence is more likely to be lower.…”

“…The choice of GRADE methodology was aimed at improving the transparency and reproducibility of the appraisal process; this structured and flexible methodological tool provides a systematic approach in the assessment and is meant to minimise biases and improve consistency of the decisions. 5 …”

Potential approaches for the pricing of cancer medicines across Europe to enhance the sustainability of healthcare systems and the implications

“…Current proposals to address concerns include establishing meaningful minimum effectiveness levels for new oncology medicines to be funded at higher prices than current standards, first proposed in the United Kingdom in 2000 [40,41], defining what is meant by innovation [42], and implementing fair and transparent approaches toward the pricing of new oncology medicines proposed by European payers, the World Health Organization (WHO) and others although there are concerns with issues including fully costing R. & D [43][44][45]. Potential proposals also include re-looking at managed entry agreements (MEAs) with countries concerned they may not always be getting optimal discounts in practice with rebates continuing to be confidential [46,47]; however, balanced against increased opportunities of reimbursement with such schemes combined with the potential to gain valuable evidence of the performance new oncology medicines in routine clinical care [46,48].…”

Non-Price-Related Determinants of Value and Access for Novel Non-small Cell Lung Cancer Treatments: A Cross-Country Review of HTA Decision Making

“…The definition of innovation and novelty was not clear even in those HTAs where the agency explicitly considered these factors, which may explain some of the variability between agency decisions. Research is underway on how best to define and reward medical innovation [34,35], which may clarify and qualify the value of innovation in future reimbursement decisions.…”