Claudia Garimberti -

On 27th November 2021, a new piece of legislation on rare diseases was published in the Italian Official Journal (Law 175/2021: “Provisions for the treatment of rare diseases and for the support of research and production of orphan drugs”).

The main goal of the new law is to safeguard the right to health of people suffering from rare diseases through the following measures:

  1. even supply of services and medicinal products, including orphan drugs, throughout Italy;
  2. coordination and periodic update of the essential levels of care (LEA) and of the list of rare diseases;
  3. coordination, reorganisation and strengthening of the national network for the prevention, surveillance, diagnosis and treatment of rare diseases;
  4. support for research.

Some particularly interesting points are the following:

  • Faster time to access for orphan drugs.

Orphan drugs must be made available by the Regions for patients with rare diseases even before their formal inclusion in the local formularies.

  • Personalised diagnostic therapeutic plans (PDTA, piano diagnostico terapeutico assistenziale).

The NHS pays for all the necessary treatments included in the personalised diagnostic therapeutic plans. As a derogation from the Ministerial Decree dated 11 February 1997, even medicinal products that are marketed in other countries, but not in Italy, may be imported and even used off label (i.e. even for uses/indications not approved in the country of origin), provided that they are included in a rare disease diagnostic therapeutic plan. The drugs must be requested by a hospital, even if they will be used for home care and the cost is covered by the National Health Service.

  • Starting from 2022, the AIFA national fund instituted by Law 326/2003 has been supplemented by the equivalent of an additional 2% of companies’ promotional expenditure to support:
    • Pre-clinical and clinical studies on rare diseases;
    • Observational studies and compassionate use registries;
    • Surveillance programmes for orphan drugs and other innovative treatments authorised and marketed despite uncertainties on their medium- and long-term efficacy and safety;
    • Research and development of orphan plasma-derived products;
    • Development of neonatal screening tests for the diagnosis of rare diseases for which a cure is available or at an advance development stage.

Regulatory Pharma Net is constantly monitoring the changes to the Italian legislation on medicinal products including Orphan Drugs and supports companies in the definition and implementation of the best strategies to access the Italian market.

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