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Page 8 of 16 Fetro. Rare Dis Orphan Drugs J 2023;2:7 https://dx.doi.org/10.20517/rdodj.2023.06

form of market exclusivity, as in the case of orphan drugs (7 years in the United States, 10 years in Europe).

Patents can cover the existing product, its underlying chemical or biological make-up, as well as new
indications, dosage regimes, or mechanisms of delivery. Supplementary protection certificates are
intellectual property rights that serve as an extension to a patent right. They all give the owner the right to
prevent others from making, using, or selling the drug without permission. Patent protection is critical for
developing a new therapy.

It is challenging to obtain strong patent protection when repurposing compounds. By definition, the
structure of a repurposed drug is known and a novel patent claim to the active pharmaceutical ingredient is
not possible. Composition of matter (COM) claims, which exclude any use of the COM, are one of the
classic and powerful claim types. They cannot be proposed to be repurposed drugs in the vast majority of
cases. Repurposed drugs are eligible for patent protection through use claims or method of treatment
(MOT) claims. MOT patents may be perceived as second-tier to composition patents; however, it seems to
[28]
be, in some cases, a good way of protection and commercial success for some companies .
Intellectual property (IP) position is critical in ensuring the ability to achieve a collaboration based on
partnership and dialogue. IP may fall into two major categories, leading to different issues: it can be active
or expired.

IP is still in force
Alpelisib is an example of a successful academia-industry DR collaboration in this category leading to a
treatment for patients, although the company was not interested in its development at the very beginning.
Alpelisib was being evaluated for breast cancer at that time.

If an academic researcher has discovered a new indication for an approved or investigational drug, then
early engagement with the potential industry partner is highly recommended since the company is the
patent owner. It will be a crucial step in the process. The objective is twofold: to know if the company is
interested in the development and to obtain access to the drug/compound and potential funding of studies
performed under a material transfer agreement (MTA).

Pharmaceutical companies frequently engage in preclinical research collaboration with academics. Complex
knowledge transfer processes and substantial challenges in terms of IP may arise. Generating new IP has to
be discussed, anticipated, and validated in a consortium agreement in order to organize the rights and
[7]
obligations of all stakeholders and protect the researchers’ discovery and IP . If the company holding the IP
agrees to provide the drug but refuses to provide funding to support preclinical or clinical programs, it will
be difficult for the researcher alone to move forward with the drug, except if new patent protection can be
obtained. New formulations or drug forms for a known active pharmaceutical ingredient (API) may be
developed for a new indication and provide academics with an opportunity for patent protection. Academic
investigators may also decide to perform clinical studies without the support of the company and with no
guarantee -even if the trial is successful- of a newly approved indication. Repurposing an approved API for
a new indication would only benefit the original manufacturer who owns the IP for the composition.
Scientific and nonscientific understandable reasons may explain the company’s refusal, such as insufficient
remaining patent life, a lack of expertise and interest in the new therapeutic area, or anticipated regulatory
risks. A recent study explored innovative mechanisms to fund independent clinical research initiated and
led by researchers from academia with repurposed medicines . Focus is put on social impact bonds,
[29]
crowdfunding, or public-private partnerships to conduct expensive phase III clinical trials with, again, no