The market will not regulate itself when it comes to finding solutions to rare diseases, said Sandra Gallina, director at the European Commission’s DG SANTE, adding there is pressure to kickstart innovation on orphan drugs.
According to the head of the Commission’s service dealing with health, investments are needed to avoid a situation where some patients’ needs are not covered because they suffer from a rare condition.
“I’m not convinced about the fact that the market will take care of itself, meaning that we have a lot of pressure to find a solution for these unmet needs or rare diseases. So let me say, [there is] definitely [a need for] incentives for innovation,” said Gallina at a webinar on Monday (24 January).
She said that degenerative diseases had crawled up the list in the stakeholders’ consultations and that today there is less acceptance of limited treatments just because someone suffers from a rare disease.
“We have not been very successful in this. So we will need to tackle that. It is not a simple issue,” she added.
In an initiative already scheduled for this year, the EU executive will streamline the procedures linked to evaluating and authorising medicines for rare diseases and those that affect children.
As highlighted in its pharmaceutical strategy, the Commission wants to reorient a new system of pharmaceutical incentives to stimulate innovation in areas of unmet medical needs, such as neurodegenerative and rare diseases and pediatric cancer.
At the same webinar, CEO of Roche Pharmaceuticals, Bill Anderson, said that historically Europe’s impact on innovation has been strong.
“There is a challenge with valuing innovation. (…) The companies that are innovating tend to go and invest in the places where that innovation is most valued and reimbursed,” he said.
He added that there is a danger that sometimes people overlook the obvious, thinking, for instance, of some sophisticated industrial strategy. At the same time, what is key is a healthy environment and support for innovative medicines when available.
Both he and Stefan Oelrich, president of the Pharmaceuticals Division at Bayer A/G, highlighted funding opportunities as a critical points for pharma innovation in Europe, stressing how different funding mechanisms in the US provide better results.
“The access to capital is different in Europe than it is in the US. We will have to see how we can channel capital flows a little bit more effectively so that Europe is not just the place where we breed out all the good ideas, but where we can also capitalise on some of the value chains that should go with that,” said Oelrich.
Anderson also pointed out that a country like the US has historically shown more willingness to fund innovative medicines also when it comes to orphan drugs.
“These therapies can be really expensive to develop because if you have a disease that only affects maybe a few 100 people in the world, it still can cost tens of millions of dollars to develop it,” he said.
The EU’s Orphan Medicinal Products (OMP) regulation was adopted more than 20 years ago to regulate medicines that treat patients with rare diseases.
Speaking at an event in June 2021, Julia Wahl, managing economist at Copenhagen Economics, said the orphan drugs regulation had been successful in developing medicines, prompting more research in the field, and increasing the number of authorised treatments.
“But unmet needs still exist, and many rare disease patients still do not have treatment that they need,” she warned.
Copenhagen Economics was the knowledge partner that supported the European expert group on orphan drug incentives in developing its 14 proposals on addressing the unmet needs of rare disease patients.
[Edited by Gerardo Fortuna/ Alice Taylor]