Case Studies on Rare Disease Therapies Development – A Laboratory of Ideas for European Innovation Leadership

While the Roadmap of the Pharmaceutical Strategy for Europe focuses on European leadership and independence in pharmaceutical manufacturing, the EU should also enhance its ‘’innovation leadership”. Biopharmaceutical small to mid-sized companies and startups represent a laboratory of innovation and their potential should be nurtured by a forward- looking and predictable European ecosystem that encourages leading-edge technologies. This webinar brought together European policymakers, representatives of publicprivate partnerships as well as experts of the pharmaceutical industry and their case studies to talk about future ideas through an evidence-based approach to achieve Europe’s leadership in innovation in the healthcare sector.

Moderated by DR. ALEXANDER NATZ, Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), the panel had DR. PAUL RÜBIG, President of SME Connect and Board Member of the European Institute of Innovation and Technology (EIT); MARIA DA GRAÇA CARVALHO MEP, Member of the ITRE Committee; FABIO D’ATRI, Policy Officer, Unit B5 Medicines: policy, authorization and monitoring of the European Commission; ANNIK K-LAFLAMME, Vice-President Medical & Scientific Affairs, Global Medical & Scientific Affairs of Sobi; CLÉMENCE ROSS-VAN DORP, Former State Secretary for Health, Welfare and Sports Ambassador, Action Program “New Chances Topsector Life Sciences and Health”, Dutch Ministry of Economic Affairs; JULIEN PATRIS, Country Business Lead – Belgium & Luxembourg, Director Market Access & Policy – Europe, Canada, MEA (CEMEA) with the Alnylam Pharmaceuticals.

In his welcome speech, DR. PAUL RÜBIG highlighted the importance of development  and strengthening the health system during the pandemic. He also mentioned the crucial connection between business, education and research and believes that the cooperation between the public health system and SMEs could bring a lot of benefits and progress during the crisis.

MARIA DA GRAÇA CARVALHO MEP spoke about the Pharmaceutical Strategy that is going to be leading the pharmaceutical legislation for the next ten years as well as plans on strengthening existing frameworks. Rare diseases remains an area with a lot of unmet challenges that should be in the focus of the European Commission. She mentioned how much of a role Horizon 2020 had played to incentives R&D in the domain by supporting the founding of 200 projects covering rare diseases. Public funding remains crucial since single companies do not have enough resources to carry out the goals on their own. The answer could be the forging of the long-term Public Private Partnerships. Ms. Carvalho also noted how essential big data, available to the researchers, is to deliver results. She advocates for more Health Data Space, more power at the EU level on health and the establishing of an Agency for cooperation on health and biomedical research.

On the Commission side, FABIO D’ATRI clarified that the Pharmaceutical Strategy is designed to create a future proof regulatory framework and support industry in promoting research and technologies that actually reach patients and fulfil their therapeutic needs while addressing market failures. It will also take into account the weaknesses exposed by the pandemic and factor-in appropriate actions to strengthen the system. The Strategy will see very concrete long-term actions implicating change of legislation sided with proximate guidelines and pilot projects. The Commission will strenghten the Orphan legislation to activate SMEs in the Orpahn field and insentivise companies explore currently less profitable areas. Fabio D’Arti noted that now is the momentum for all actors to collaborate, to share ideas and shape the new Pharmaceutical Strategy.

ANNIK K-LAFLAMME spoke on behalf of Sobi, a biopharmaceutical international company with a focus on rare diseases headquatered in Stokholm. Annik introduced Sobi’s business model. “Innovation has a price”,- she remarked, – “but sustainable business models are just as important as incentives they are granted with”. As a European company, Sobi is commited to researching and developing treatments for the remaining 95% of rare diseases. She commended the collaboration with various stakeholders among which the Patient Association. The company sees the descripancy of the requirements as a main challenge that often delays release of the vital drugs.

JULIEN PATRIS introduced Alnylam Pharmaceuticals, which revolves around the same issue of rare diseases. Alnylam has been investing for nearly 20 years, launching two products without yet making a profit, and continues to increasingly invest in R&D to bring innovative products to market. Julien explained a few of their business models as well. They believe that reaching success is a shared responsibility and a new Social Contract between Biotech and Society is needed.

To conclude, CLÉMENCE ROSS-VAN DORP took the Netherlands as an example where a country greatly stimulates collaboration. There is still space for improvement. Clemence believes that steering is needed on successful variables that lead to successful innovation. The best way to locate those variables is by reaching out to science parks clusters, by analysing their most successful innovations and how these were achieved . She took note of the impact of the current scattered landscape of financing and the lack of a coherent approach to granting incentives. Clemence believes that team work must be promoted and more action lines are needed to inform SMEs on how to enter the ecosystem or the Public Private Partnership programmes as well as outline, what makes Public Private Partnerships a success.