1996: Introduction of law in Italy for early access to drugs (648/1996) to support rare disease patients

Nominated by: Federchimica Assobiotec 

Organisation in nomination: Government of Italy

Early access programmes for novel medicines can benefit patients suffering from serious illnesses for which effective therapeutics are not yet available. For rare diseases, this is particularly important where no therapeutic alternatives are available, and the disease itself may be life-threatening or have a serious impact on the quality of life.  Access to such novel medicines outside clinical trials and prior to formal approval/reimbursement enables substantially greater benefits for citizens.

In 1996, Italy introduced in the national legislation Law 648/96, which allowed the reimbursement of certain medicinal products if no other therapeutic alternative was available, and provided that results phase II clinical trials, with favourable evaluation in terms of efficacy and safety, were available.  Eligible products were to be either:

  • Approved for commercialisation in other States, but not in Italy;
  • Not yet approved, but already subject to clinical trials;
  • To be used for a therapeutic indication different from that/those already authorized (off-label use).

Subsequently, Italy issued legislation regulating compassionate use and therapeutic use outside clinical trials (Ministerial Decree 8th May 2003, now replaced by Ministerial Decree 7th September 2017); and a Law (no. 326 of 24th November 2003) which established a fund covering named-patient reimbursement for not yet marketed “orphan medicinal products for rare diseases and medicinal products that represent a hope of cure for specific and serious diseases”.

These forms of early access to medicinal products can be considered one of the pillars on which the adaptive pathways approach is built. Adaptive pathways is a scientific concept for medicine development and data generation which allows for early and progressive patient access to a medicine. The approach makes use of the existing regulatory framework for medicines, and brings together different stakeholders such as regulators, Health Technology Assessment (HTA) bodies, medicine developers, health professionals and patients to agree on a prospective plan that allows the generation of data on a drug throughout its life cycle in areas of high unmet medical need, where it is difficult to collect data via traditional routes and where large clinical trials would unnecessarily expose patients who are unlikely to benefit from the medicine.


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