Claudia Garimberti -

Law 326/2003 provided for the establishment of a national fund at AIFA for the use of orphan drugs and for the treatment of rare diseases and to drugs that represent a hope of therapy, pending commercialisation, for particular and serious diseases. The fund is made by the 50% of the contribution that pharmaceutical companies pay to AIFA on an annual basis.

Inclusion criteria to access the AIFA fund are the following:

  • Medicinal products for rare and severe diseases required for individual cases on a nominal basis under conditions of urgency
  • Absence of valid therapeutic alternative in a condition of maximum or important therapeutic need
  • Medicinal products/indications for which published efficacy data from phase II clinical trials are available or, in the case of rare diseases, evidence of similar relevance, and which represent, in the applicant’s view, a treatment opportunity for the individual patient for whom the request is made, assessed on the basis of clinical conditions, stage and rapid progression of the disease
  • Denial and/or inability to place the patient in a compassionate use program or a clinical trial.

Exclusion criteria are the following:

  • Medicinal products for rare and severe diseases, required for repeated uses for a specific indication.
  • Medicinal products/indications available through other regulatory procedures:
    • Medicinal products/indications reimbursed by the Italian NHS (class A, H)
    • Medicinal products included in the list provided for by Law 648/96 for the requested indication
    • Medicinal products/indications in C(nn) or C class of reimbursement
    • Presence of compassionate use programs for the same clinical condition
    • Possibility of inclusion in an ongoing clinical trial with the drug for the same indication
  • Medicinal products available in Italy for different indications from those objects of request and not under EMA evaluation
  • Medicinal products which were rejected by EMA CHMP for the requested indication, or withdrawn by the Company following an ongoing negative evaluation by EMA CHMP
  • Medicinal products/indications which were rejected by the AIFA CTS for the inclusion in the 648/96 List.

The request to access the AIFA fund can be submitted by centres with documented experience and for nominal use.

Should the requests for single uses become repetitive and systematic, considering the phase of clinical development or access to the market for the specific medicinal product/ indication, AIFA will assess the possibility of:

  1. activating a compassionate use program by the company that owns the medicinal product;
  2. starting clinical trials;
  3. starting the assessment procedure for the inclusion of the drug in the L 648/96 list by the Technical- Scientific Commission.

Regulatory Pharma Net has extensive experience in advising and devising strategies to facilitate early accesses to orphan drugs for patients with rare and severe diseases.

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