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Fetro. Rare Dis Orphan Drugs J 2023;2:7 https://dx.doi.org/10.20517/rdodj.2023.06 Page 11 of 16

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new projects , but it is only part of the puzzle. Market access strategy is critical to therapy launch success.
Landscape assessment is still more relevant in the rare disease ecosystem where literature and natural
history are limited. Close collaboration with clinicians is essential to better understand the burden of the
disease and patients’ needs and work together on the clinical development program to optimize pediatric
formulation and patient outcomes keeping in mind the expectations of regulatory agencies and payers.

A paper published in 2022 compares the price of Hemangiol® approved in IH and the price of its off-label
alternative, not approved in the indication, in Germany . It underlines the clinical development and
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registration program value taken into account by payers in the price obtained by the approved product. The
price difference is significant but relatively lower for Hemangiol® compared with two other orphan drugs
presented in the paper.

Although the results may not be generalized, they may foreshadow a positive return on investment for
Hemangiol®. In any case, it is a success story with an approved treatment covering an unmet medical need
for babies and infants with severe IH.

HOW
Implementing a relevant academia-industry collaboration requires a strategy to move forward. There are
many opportunities for industry to partner with academia and vice versa. There is a wide range of forms
varying in the extent of involvement and risk taken by the stakeholders. Identifying research opportunities,
applying to private-public calls for proposals, forming an early-stage partnership, developing new methods
for the design of small population clinical trials, understanding the natural history of the disease to better
treat it, or advancing real-world evidence to support payer decisions are just a few examples of it. How to do
it is the key question. A case-by-case answer is probably the best answer since there are so many different
factors to consider for complete and successful achievement. Such factors are related to the stakeholders
themselves, their profile, skills, interest, their willingness, and commitment, but they are also dependent on
the candidate repurposing drug, its IP and freedom to operate, its safety and toxicity profile, and the data
already accumulated toward gaining regulatory approval .
[27]

A brief overview of key suggestions and current initiatives in DR is given hereafter.

Educational program for academia
Academic researchers need to be guided and trained in IP, regulatory procedures, or market access to be
able to understand/challenge the constraints and expectations of the industry. Technology transfer offices
(TTOs) or neutral and trusted third parties (public or private) may support them to this end. First of all, it is
extremely important for academia to understand industry needs when dealing with practical problems to
which they can apply their knowledge. Academic research may be closely linked with practical implications
in an industrial environment . It is also important for academia to be aware of the pharmaceutical
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development of a drug and for the industry to be sensitized to the key role and importance of publications,
grant approvals, and international networking for academia, notably for Ph.D. students and young
researchers. There is a real need for mutual knowledge of expectations and constraints from both academia
and industry in order to facilitate interactions and enable fruitful collaboration. Both have to learn from
each other.

Pilot project from EMA and STAMP expert group
Another way of implementing relevant academia-industry collaborations is provided by some European
initiatives dealing with DR. In October 2021, the European Medicines Agency (EMA) and the Heads of