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Rare diseases and orphan drugs: a healthcare priority, a Recordati  priority. 

The Group is dedicated to caring for the most vulnerable.  The claim “Focused on the Few” expresses Recordati’s conviction that every single patient should have access to the best possible treatment.
Rare diseases are predominantly genetic disorders that can affect patients of any age, gender and ethnicity, and involve every category of medical specialisation. These are chronic, often fatal or severely debilitating diseases that have a huge impact on patients, their families and society. To treat these diseases, specialist medical products known as “orphan drugs” are developed. 

A disease is defined as rare when its prevalence, understood as the number of cases in a specific population, does not exceed a set threshold. In Europe, this threshold is 0.05% of the population, corresponding to 5 cases in every 10,000 people, while in America the threshold is less than 200,000 people in the country’s entire population. Over 30 million people are affected in Europe alone. There are more than 7,000 known rare diseases, but today approved treatments exist for just 10% of these. The number of patients is so small that a rare disease is often not “adopted” by the pharmaceutical industry and hence the expression “orphan drug”. 

Due to the broad spectrum of existing diseases and the scarcity of available information, physicians may never examine a patient with a rare disease in their entire career. For this reason, there is always the risk that when a child is born with a rare disease, a correct diagnosis may not be made and and timely appropriate treatment treatment may not be provided. The limited number of patients and scarcity of relevant knowledge and expertise characterise rare diseases. In order to guarantee that the scarce knowledge and available resources are made available, these are often shared through international cooperation channels. In order to provide assistance to persons affected by a rare disease and encourage pharmaceutical and biotechnology companies to invest in treatments for rare diseases, governments have introduced various legal and financial incentives.

The Recordati group operates in the rare diseases segment worldwide through Recordati Rare Diseases, its dedicated group of subsidiaries which make our specialty pharmaceuticals for rare diseases available directly in Europe, the Middle East, the United States, Canada, Russia, Australia, Japan and certain countries in Latin America (Brazil, Mexico and Colombia) and through highly qualified distributors in other areas, covering over 100 countries around the world.

The Group has developed a direct distribution and packaging system capable of efficiently providing very small quantities of specialised products to people all around the world very quickly. Recordati manages a GMP-certified site in Nanterre (Paris) that is entirely dedicated to packaging, storage and shipment of products for rare diseases to all countries.

The activities carried out by Recordati Rare Diseases include support for patient associations for people affected by rare diseases, which help patients and their families, facilitating access to orphan drugs and treatment centres. Recordati's orphan drug specialists actively collaborate with the medical community to facilitate dialogue between hospitals with limited expertise in rare diseases and specialist medical centres able to diagnose and treat rare conditions in an appropriate manner. Through Recordati Rare Diseases Foundation, the Group is committed to improving diagnosis and treatment of rare diseases, also through targeted training courses.
Recordati Rare Diseases Fondation D’Entreprise 

Working in the field of rare diseases means we have an important responsibility towards patients and healthcare professionals and lies at the heart of Recordati’s commitment.
The Recordati Rare Diseases Foundation was established to provide unconditional support to training for the scientific community in the field of rare diseases. These high-level courses are organised under the supervision of an independent scientific committee. The overall aim is to share experience in the diagnosis, management and outcome of rare disorders where individual knowledge is by its nature limited.
The Foundation gives specialists the opportunity to broaden their expertise, develop new ideas and establish scientific relationships.
A number of live events are held each year bringing together clinicians and scientists from all over the world to discuss innovations and new diagnostic and management strategies.
During the COVID-19 pandemic and consequent lockdowns, courses have been postponed and virtual meetings organised to keep up to date with developments in the scientific community.
The Foundation also offers online e-learning courses aimed at providing physicians worldwide with clinically useful information and updating them on current knowledge and treatment recommendations.

Also in the context of facilitating access to treatments, the US company Recordati Rare Diseases inc. has developed two separate programmes to provide assistance to patients that qualify to receive support for the costs related to its products: the Patient Assistance Program (PAP) and the Co-Pay Assistance Program (CAP):
  • Patient Assistance Program (PAP): this programme enables Recordati Rare Diseases to supply products to medical professionals or hospitals which require free product in order to treat patients who do not have adequate medical insurance to cover the cost of the drug and are able to demonstrate financial need. A case-by-case assessment is carried out by a third party on behalf of Recordati Rare Diseases in order to substantiate eligibility and register patients in the programme.
  • Co-Pay Assistance (CPA): this support programme, available for certain products, is administered through a third party on behalf of Recordati Rare Diseases and provides financial support to insured patients for all or part of their financial responsibilities not covered by their insurance plan. In order to benefit from this assistance, patients must fulfil certain eligibility requirements, and have a valid medical prescription for the product. 
Additional information can be found in the Annual Report and in Rare Diseases website page