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The independent research on drugs

The promotion of independent research on drugs represents one of the strategic tasks assigned to the Italian Medicines Agency by legislation. The general aim of the program is to support clinical research on drugs in areas of interest for the National Health Service (NHS) and where commercial support is normally insufficient.

There is not only a concern for patient populations normally excluded by clinical studies on efficacy and safety, such as children, pregnant women and the elderly. There is also a need to obtain more information on research issues less explored in commercial research, such as clinically relevant end points, relative efficacy of drugs (including the assessment of multimodal strategies), and long term follow up on efficacy and safety of therapies.

AIFA set up the program on independent research in 2005, and five calls for proposals (2005, 2006, 2007, 2008 and 2009) have already been launched. The call for proposals is aimed at investigators working in public (e.g., NHS, universities, etc.) or non-profit organisations (e.g. scientific foundations, patient associations, etc.). For the first three years, three main areas of drug research were included in the program:

  • Area 1. Orphan drugs for the treatment of rare diseases and drugs for non-responders.
  • Area 2. Head to head comparison of drugs and therapeutic strategies.
  • Area 3. Strategies to improve the appropriateness of drug use and pharmacoepidemiology studies.

Lack of support in the area of rare diseases generally stems from the limited segment of patient populations involved. Comparative studies, especially when generics are included, and strategies aimed at providing effective and independent information to GPs and patients, also suffer from a considerable degree of neglect.

The 2008 call for proposal and the 2009 one, which at the time being is on going, included areas number 2 and 3 of the program.

The AIFA fund

An innovative aspect of the program is represented by the mechanism of funding of the independent research: an ad hoc fund was set up, requiring pharmaceutical companies to contribute 5% of their yearly expenditure devoted to promotional initiatives (e.g., seminars, workshops, etc.) aimed at physicians. This ad hoc fund consists of about 40 million Euro each year and it guarantees not only the AIFA research program funding, but other AIFA-related activities as well (independent drug information, reimbursement of orphan drugs, and “life saving” drugs, not yet marketed). An independent scientific committee (Research and Development Committee, R&D) has been founded, in order to coordinate the different aspects of the public scientific research. Moreover, the R&D committee plays a fundamental role in proposing research areas where to address the public funded research, in conducting the first phase of the selection process, and in supervising the implementation of the projects.

The selection process

The evaluation procedure mirrors the accredited standards of internationally recognised scientific institutions. The evaluation of projects is based on the following criteria:

  • Relevance of the expected results for the clinical practice within the NHS;
  • Scientific validity, in order to select projects with the highest scientific merit;
  • Potential impact on the regulatory activity of AIFA, with specific attention to guide the decision about drug reimbursement and use limitations within the NHS;
  • Lack of commercial interest for the objectives of the study, in order to use available resources on important but neglected areas of interest.

The project to be funded are selected via a two-step review process. In the first step, researchers are required to submit a “letter of intent” (i.e. a synthesis of the study protocol) which is evaluated by the R&D Committee. Investigators passing to the second phase of the evaluation are required to present a fully detailed study protocol.

The evaluation of the study protocols entailed the organisation of independent study sessions, involving more than 20 experts (half from Italian institutions and half international). The R&D Committee members can not participate the study sessions in this way guaranteeing the independence of the evaluation procedure. At least two written comments are obtained for each study protocol before the study session meeting and thoroughly reviewed, together with each protocol, during the study session plenary discussion. Finally, the average of each expert’s vote is calculated giving the project’s final score. Study protocols are then ranked on the basis of the final score and, starting with the highest score, the available funds (35 million Euro in 2005, 31 in 2006, 13 in 2007 and more than 11 in 2008) are distributed accordingly.

 

Foto di Mario Melazzini

Pre-Authorization Department

DG Regency: Mario Melazzini
E-mail: segr.tecnica.dg@aifa.gov.it (for administrative correspondence with the DG's office)
           segr.scientifica.dg@aifa.gov.it (for scientific correspondence with the DG's office)

 

Indipendent Research Office

Head of Office: Sandra Petraglia
E-mail: s.petraglia@aifa.gov.it

Data aggiornamento: December 2016
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