Law 10 November 2021, n. 175: in the Official Gazette the new provisions for the treatment of rare diseases and for the support of research and production of orphan drugs

On November 27, 2021, Law No. 175/2021, setting forth "Provisions for the treatment of rare diseases and for the support of research and production of orphan drugs." was published in the Official Gazette of the Italian Republic. The law will come into force December 12, 2021, although the application of some provisions will be effective only after the issuance of specific ministerial decrees.

Law 175/2021 was issued with the aim of strengthening the protection of the right to health of people affected by rare diseases, through rules ensuring uniformity in the supply of medicines, as well as through support for research.

Rare diseases and orphan drugs

Law 175/2021 provides a definition of rare diseases anchored to the concept of "low prevalence", corresponding to a percentage of less than five individuals in ten thousand. The law specifies that rare diseases also include ultra-rare diseases, characterized by a prevalence of less than one individual in fifty thousand.

Law 175/2021 also incorporates the definition of orphan drug provided by Regulation (EC) No 141/2000. Namely, A medicinal product shall be designated as an orphan medicinal product if its sponsor can establish:

1. that it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10 thousand persons in the Community when the application is made, or that it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in the Community and that without incentives it is unlikely that the marketing of the medicinal product in the Community would generate sufficient return to justify the necessary investment; and
2. that there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Community or, if such method exists, that the medicinal product will be of significant benefit to those affected by that condition.

Benefits paid by the National Health Service

Law 175/2021 helps to define the essential levels of care for rare diseases. In particular, it provides that the followings are fully charged to the National Health Service:

1. health treatments which included in the personalized diagnostic therapeutic care plan and indicated as essential, such as diagnostic monitoring, clinical tests, pharmacological therapies, palliative care and health services;
2. medical devices and healthcare equipment, which are included in the personalised therapeutic diagnostic plans, including ordinary and extraordinary maintenance and any training for their use.

Access to medicines

Class A and H drugs prescribed for the care of patients with a rare disease may be dispensed by:

1. health care pharmacies;
2. territorial health authorities to which the patient belongs;
3. public and private pharmacies affiliated with the National Health Service.

As an exception to the provisions on pharmaceutical prescriptions, for prescriptions regarding a rare disease, the number of items per prescription may be higher than three when provided for by the patient's Personalized Diagnostic-Therapeutic-Assistance Plan.

Imports from abroad for uses not permitted in the country of origin

As an exception to the provisions that regulate the importation of medicines from abroad, the importation of medicines marketed in other countries will be allowed, even for uses not allowed in the country of origin, as long as they are included in the Personalized Diagnostic-Therapeutic Care Plan.

The importation shall be requested by an hospital, even if the drugs are used for home care, and the cost will be charged to the National Health Service.

Funding for research into rare diseases and the development of orphan drugs

As from 2022, the national fund for the use of orphan drugs for rare diseases and drugs that represent a hope of cure, pending marketing, for particular and serious pathologies, will be supplemented with an additional payment equal to 2 per cent of the expenses self-certified by 30 April of each year by pharmaceutical companies on the total amount of expenditure incurred in the previous year for promotion activities aimed at healthcare personnel.

These additional resources will be used to fund:

1. preclinical and clinical studies promoted in the field of rare diseases;
2. observational studies and registries of compassionate use of drugs not yet marketed in Italy;
3. surveillance programmes on orphan medicinal products and other innovative treatments placed on the market on the basis of biological hypotheses and initial evidence of efficacy, but without firm knowledge of the efficacy and safety of their use in the medium and long term;
4. research and development of plasma-derived orphan drugs;
5. projects for the development of neonatal screening tests for the diagnosis of rare diseases for which a cure is available or at a proven advanced stage of development.

Tax incentive equal to 65% of the expenses incurred up to a maximum annual amount of €200,000 for each beneficiary, up to a total annual expenditure limit of €10 million

Law 175/2021 establishes a tax incentive in favor of public or private entities that (i) carry out activities for the development of therapeutic protocols on rare diseases or the production of orphan drugs, and (ii) those that finance research projects on rare diseases or orphan drugs carried out by public or private research entities.

The incentive is granted, starting from 2022, in the form of a tax credit, equal to sixty-five per cent of the expenses incurred for the start-up and implementation of research projects, up to a maximum annual amount of €200,000 for each beneficiary, within the overall spending limit of €10 million per year.

Within six months of the entry into force of Law 175/2021, the Ministry of Health shall issue a decree to regulate the methods of implementation of the incentive.

Support for pharmaceutical and biotechnology companies

As from 2022, pharmaceutical and biotechnology companies intending to carry out studies aimed at the discovery, registration and production of orphan drugs or other highly innovative treatments will be able to benefit from support to research activity referred to in the Decree of the Minister of Education, Universities and Research no. 593 of 26 July 2016

Within six months from the entry into force of Law 175/2021, the Minister of Universities and Research must issue a decree to implement the support measures.