In this section we list all the relevant issues on which Fondazione Telethon has expressed its position and the relevant documents.
Research integrity
We work to preserve and promote the responsible conduct of research (RCR) within the Telethon-funded research community. Research integrity (RI) is the basis of RCR and standard principles of RI are shared within the international scientific community, as laid out by the European Code of Conduct for Research Integrity. In line with these principles, we drew up a RI Policy for Telethon-funded scientists working in Institutions all over Italy.
Animal testing
Animal testing is still an indispensable tool of biomedical research, despite efforts to limit its use in accordance with the R-principle (Replacement, Refinement, Reduction). However, it is a widely debated and sometimes controversial issue. Read our official position and answers to the most common doubts on this issue.
Open access
Our mission is to support and promote scientific, basic and clinical research aimed at finding ways to treat genetic diseases. Optimising the dissemination of Telethon-funded studies published in the major international peer-reviewed journals through free online access for everyone is the best way to ensure that this research is available not only to the scientific community, but also to Telethon’s main stakeholders such as patients and donors. That’s why we promote free access to our scientific publication
Access to affordable medicines for rare diseases
If a therapy exists, it is not acceptable for it not to be available to a patient who needs it, regardless of how rare their disease is. That is why we are involved in many international multi-stakeholder initiatives, to keep the attention of institutions on the issue high and to try to break down major economic and regulatory barriers. To learn more, read the following documents:
Pharmaceutical legislation
Since 2023, we have become the first not-for profit marketing authorization holder of an ATMP for a ultra-rare disease in the EU. On the occasion of the EU Commissions proposal on the Revision of the EU general pharmaceuticals legislation, we shared our comments, in particular about three main issues: orphan medicinal products addressing a high unmet medical need; early access of these products; European Medical Agency structure. Find more details here.
Ensuring access to the therapies we develop
When a patient cannot access one of our therapies, our first commitment is clear: to do everything possible to help them obtain it through the right channels. We activate all available channels to persuade governments, regulators and payers to ensure that those responsible step in and do their part, and we stand alongside patient organisations to help patients and families navigate the pathways available in their country.
For the therapies we develop and distribute, this may also include exceptional, case-by-case support: the provision of a therapy on a pro bono basis, governed by a defined policy and transparent criteria, and covering the therapy itself only. To learn more, read our policy >
