The Consolidated Act on Rare Diseases is finally approved
After three years, Italy has finally adopted its first law on rare diseases. On November 3, 2021, the Italian Senate unanimously approved the Consolidated Act on Rare Diseases, or “Testo Unico sulle malattie rare” (the “Act”).
The aim of the Act, which consists of 16 articles, is both, to make the treatment of rare diseases uniform throughout the Italian territory and to promote progress in research, preserving the good practices developed in recent years.
Further decrees are needed for the Act to be implemented. Those should be adopted by the government in the coming months to ensure that the provisions are operational.
THE MAIN PROVISIONS OF THE ACT
1) Definition of rare disease and orphan drug
According to the Act, “rare diseases are defined as those diseases, including genetic ones, that present a low prevalence,” where “low prevalence” means a prevalence of fewer than five individuals out of ten thousand. The field of rare diseases also includes ultra-rare diseases, i.e., those characterized by a prevalence of fewer than one individual in fifty thousand. This definition also applies to rare cancers.
In accordance with the criteria established by Section 3 of Regulation (EC) No. 141/2000 of the European Parliament and of the Council of December 16, 1999 on orphan medicinal products, a drug shall be defined as an orphan drug if (i) either it is intended for the diagnosis, prophylaxis, or treatment of a life-threatening or chronically debilitating disease affecting no more than five out of every ten thousand individuals at the time the application for orphan drug designation is made; or (as an alternative to the requirement above) it is unlikely that, in the absence of incentives, the commercialization of such a drug would be sufficiently profitable to justify the necessary investment; and (ii) there are no satisfactory methods of diagnosis, prophylaxis, or treatment of the diseases referred to in the previous point, or if there are the drug has significant beneficial effects for individuals affected by such diseases.
Moreover, in order to overcome regional disparities in access to orphan drugs, the Act provides that, once approved by the Italian Medicines Agency (“AIFA” or “Agency”), the drugs shall be made immediately available by the regions, i.e., without having to wait for their inclusion in Hospital Therapeutic Handbooks or other similar lists set up by the relevant regional or local authorities. Moreover, pursuant to Section 10, paragraph 5 of Law Decree No. 158/2012, those handbooks must be updated at least every six months.
This is an important measure aimed at guaranteeing faster access to orphan drugs (already provided for innovative drugs) and fewer regional disparities; previously, patient access varied at the regional level due to the different ways in which a new drug was introduced into normal clinical practice through Regional Therapeutic Handbooks and, at the local level, through Hospital Therapeutic Handbooks.
Finally, as a derogation to the Ministerial Decree of February 11, 1997, the Act allows the importation of drugs that are already on the market in other countries, even for uses not authorized in the country of origin, provided that they are included in the diagnostic and therapeutic plans as well as in the list under Law 648/1996. These drugs must be requested by a hospital, even if used for home care, and their costs are paid by the National Health Service (“NHS”).
2) Diagnostic and therapeutic plans and Essential Levels of Care (LEA) for rare diseases
A diagnostic and therapeutic plan includes the treatment and monitoring required by a person affected by a rare disease. The plan, along with an expenditure forecast, is shared with the services in the national network for rare diseases that are responsible for implementing it after sharing it with the patient or their family.
Health treatments included in the LEA or qualified as life-saving—covered in a personalized diagnostic and therapeutic plan and indicated as essential—are charged to the NHS. These include but are not limited to health and social services, different types of care (including palliative care and rehabilitation), drugs, and medical devices.
Moreover, the Act explicitly requires timely updating of LEA and stipulates that, pending the conclusion of the procedure for updating LEA, the Minister of Health, in agreement with the Minister of Economy and Finance, shall issue a decree to update the list of rare diseases identified by the National Center for Rare Diseases of the National Superior Institute of Health, as well as the necessary treatments for rare diseases.
3) Solidarity Fund for people with rare diseases
The Act also provides for the establishment of a Solidarity Fund for people with rare diseases, with an initial budget of EUR 1 million per year starting from 2022, aimed at financing measures to support care and assistance work for people affected by these diseases who are considered 100% disabled and in serious condition and in need of ongoing assistance.
The Fund shall be established by decree within three months of the effective date of the Act.
4) AIFA 5% National Fund
Beginning in 2022, the AIFA 5% National Fund, created to finance orphan drugs that may be used to treat rare diseases and drugs which represent a hope of cure, while they await authorization for marketing, will receive an additional contribution equal to 2% of the expenses for promotional activities to healthcare professionals, self-certified by pharmaceutical companies each year. Notably, this fund is currently financed with 50% of the contributions paid by pharmaceutical companies, which correspond to 5% of the expenses for promotional activities addressed to healthcare professionals, self-certified by the same companies. Following the Act, the AIFA 5% National Fund will receive 4.5% of that amount (instead of the current 2.5%).
5) Tax incentives
In order to encourage research aimed at the development of therapeutic protocols for rare diseases and orphan drugs, beginning in 2022 a tax credit is provided for public and private entities that carry out these activities or finance research projects. That credit shall be equal to 65% of the expenses incurred for the launch and implementation of such research projects, up to a maximum annual amount of EUR 200,000 for each beneficiary, with an overall spending limit of EUR 10 million per year.
The Act also provides pharmaceutical and biotechnology companies that intend to carry out studies aimed at the discovery, registration, or production of orphan drugs or other highly innovative treatments the opportunity to benefit from the measures provided by the Decree of the Minister of Education, University and Research of July 26, 2016 No. 593 (concerning financial grants), starting in the year 2022.
6) Information on rare diseases
As part of the information and communication activities covered under current legislation, the Ministry of Health shall promote useful actions to provide timely and accurate information to patients and their families and raise public awareness of rare diseases.
Within six months from the date the Act takes effect, regional and interregional coordination centers shall be equipped with appropriate tools to provide people with rare diseases the information they need to access and use in an optimal way the regional and interregional networks of care the centers coordinate, as well as to guide people affected by these diseases, including with regard to care offered by regions other than their regions of residence.