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Fetro. Rare Dis Orphan Drugs J 2023;2:7 https://dx.doi.org/10.20517/rdodj.2023.06 Page 13 of 16

libraries, associated pharmacological data). Although there are open-access databases that can be used for
searching chemical, biological, and pharmacological data, it is not always easy to search into such specific
databases. Another challenge is to have access to shelved industry compounds and their trial data when
companies are reluctant to. It is then critical to simplify the procedures for accessing public databases and
promote data sharing between public and private structures.

Improving awareness of intellectual property issues
DR can be done on shelved, failed compounds, live patents, and drugs sold on the market as well as generic
drugs. DR always relies on a previously known drug, but each situation has its own challenges when
considering collaborative research and development between pharmaceutical companies and academic
researchers. If the repurposing project comes from academic bodies, they will have to discuss and negotiate
with the pharmaceutical companies holders of the molecule involved in the project to convince them to
agree on the methods for developing and sharing intellectual property.

It is then important to provide all researchers with better training in legal aspects of the drug development
process. A proper DR project needs expert legal counsel to advise on the issues to identify risks and
opportunities when setting out on new projects.

Anticipating preclinical and clinical requirements
The real feasibility of the project, the financial and human resources to be deployed, and the time needed to
complete the project should always be analyzed and anticipated. There is a real need for tools or structures
capable of providing advice on the model of the Scientific Advice/protocol Assistance of the EMA.

Improving the regulatory procedures for market access
Market Authorization is a regulatory process at the European level and does not necessarily mean market
access in all Member States of the European Union. Some efforts should be made to get better alignment
and synergies between regulatory and payers’ requirements. Regulatory agencies assess new treatments’
benefits and harms to the exclusion of economic considerations, whereas payers focus on effectiveness and
economic consequences. In France, for instance, the doctrine of the High Authority for Health (HAS)
Transparency Commission (TC) needs to evolve both regarding the methodology of potentially admissible
trials and the more specific part of the so-called “relevant” clinical comparator. The HAS Transparency
Commission’s main task is to assess medicinal products in order to provide recommendations on
reimbursement decisions made by public authorities. Access to reimbursement in France follows Marketing
Authorization and requires pharmaceutical companies to submit an application file to the HAS
Transparency Committee (TC)

Creating a public-private partnership support structure on a European scale
The main recommendation made during the round table was the creation of a support structure capable of
managing the most complex aspects of repurposing projects. A trusted third party is useful for initiating
collaborations and providing the infrastructure and resources for academia and industry to work together.
Such a support structure would have dedicated financial resources and expertise at all stages of development
until the Marketing Authorization. It could even become the MA holder for the repurposed drug if need be.
The Innovative Health Initiative (IHI) is a public-private partnership between the European Union and the
European life science industries whose main objectives are to translate research and innovation into tangible
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benefits for patients. Such a structure could be used as a model applied to DR .