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Focus on Regulation

New rules for expanded access in Italy

Fast track for orphan diseases and rare forms of cancers

By Ministerial Decree of 7 September 2017 (DM 7 September 2017), published in the Italian Official Journal on 2nd  November2017, the Ministry of Health enacted new rules on expanded access to medicinal products, for which the authorisation procedure is still on-going, replacing the former Ministerial Decree of 8 May 2003, ruling on the same matter.

Under certain conditions, results of Phase I clinical trials may now be sufficient for requesting approval of an expanded access program intended to treat orphan diseases or rare cancers.

Overview of early access of medicinal products in Italy

In the European Union, early access may be achieved through “compassionate use programs“, which are contemplated by Article 83 of Regulation (EC) No 726/2004, on centrally approved medicinal products, while specific regulation of these is left to the Member States.

According to the Guideline on Compassionate Use of the European Medicines Agency, compassionate use programs should apply to group of patients and should be kept distinct from a named-patient supply (as meant in Article 5 of Directive 2001/83/EC). Read more

Expanded access under the old regulation

Expanded access according to Ministerial Decree of 8 May 2003 could be sought if “there were no valid therapeutic alternative for the treatment of serious diseases, or rare diseases, or pathological conditions that put the life of the patient at risk“.

It was furthermore required that clinical trials were on-going (phase III) or have been concluded (phase II or III). Read more

The new regulation – what it is not going to change

DM 7 September 2017 contains a detailed list of experimental medicinal products and medical conditions that may justify a request for an expanded access program.

In particular, it is now made clear that the expanded access may be sought for non-authorised products, as well as for medicinal products that are authorised in Italy or abroad but for a different therapeutic use, or which has been authorised abroad but not in Italy. Expanded access may be requested also for advanced therapy medicinal products (ATMPs).  Read more

Fast track for orphan diseases and rare cancers

DM 7 September 2017 lays down new and special conditions for expanded access in the case of orphan diseases or rare cancers.

While for other medical conditions results of Phase III, concluded or ongoing, are needed (or at least results of Phase II should be available, if the use is for life threatening or serious diseases), results of Phase I clinical trials may now be sufficient for orphan diseases or rare cancers. Read more

Declaration from the pharmaceutical company on the activation of an expanded access program

Under the new regulation, “Pharmaceutical companies, which intend to activate an expanded access program in Italy, shall inform AIFA on the date of activation, as well as on the date of termination of the program, specifying the medicinal product that they wish to make available free of charge pursuant to the [expanded access] regulation, without prejudice to the regulatory or safety grounds that may require a termination of the supply by operation of law“.

This provision is new and requires pharmaceutical companies to communicate the initiation and termination of an expanded access program. Additional regulation from AIFA, as to the formalities and procedures that must be followed by pharmaceutical companies for the communication, could be useful.