Together For Rare Diseases and ERICA hosted the second instalment of a webinar series on public-private partnerships (PPPs) on rare disease research.
This webinar highlighted how industry contributes to PPPs beyond funding, and provided attendees with examples of how industry has brought value to research projects conducted with European Research Networks (ERNs) in recent years.
While several partnerships between ERNs and industry have launched in recent years, the value that pharmaceutical companies bring to rare disease research is not yet fully understood.
Our joint webinar series aims to bridge the knowledge gaps between ERNs and the pharmaceutical and biotech industries, fostering collaborations that can accelerate research and development in rare diseases.
Video
The video can also be found on YouTube.
Highlights
With our Chair, Sheela Upadhyaya, our panelists described the different contributions of industry in public-private partnerships.
Vinciane Pirard, senior director and rare disease expert at Sanofi, opened the panel by explaining how industry complements the research activities into rare diseases across Europe, positing that the high unmet needs and scarcity of resources makes collaboration in rare disease research all the more important. In addition to financial resources, industry brings complimentary skillsets, knowledge and competence, but central to success is a mutual understanding between researchers and industry representatives of objectives and the ‘end goal’, as well as the importance of building and sustaining trust throughout the partnership.
Industry, Vinciane Pirard continued, offers the following attributes:
- Scientific and regulatory expertise, access to infrastructure and assets
- Operational capabilities and capacity
- Collaborative leadership and innovation drive
- A patient-centred approach and tangible contribution to the research ecosystem
Although PPPs can be accompanied by practical and methodological challenges, understanding and tackling these obstacles is essential to moulding a research ecosystem.
Anton Ussi, Operations & Finance Director at EATRIS, the European Infrastructure for Translational Medicine, noted that industry partners can bring valuable insights into regulatory requirements and help guide research design to ensure research outcomes are more readily translatable.
Improving the number of regulatory-grade outcomes in academic research is another area where PPPs could make a tangible impact. Due to stringent requirements of regulatory bodies, there is a substantial loss of opportunities, funding and time that could be alleviated by several solutions. These include the development of collaborative frameworks between academia and industry, which can facilitate knowledge exchange, shared resources, and aligned research objectives.
Anton Ussi added that “industry helps deploy research into real world settings. If you want to get your research to patients, it will have to go through industry – who best know the regulatory rigour needed for confirmatory research processes. When you work with industry, you should be ready to move your research from an exploratory, creative mindset to a concentrated, goal-oriented setting. Collaborating with companies that understand industrial processes helps bring in a multidisciplinary mindset to ensure your publication is a beginning rather than an end.”
Using already collected data, rare disease specialists from ERN BOND – the European Reference Network on Rare Bone Diseases and Sanofi are collaborating in a pilot project aimed at better understanding the disease burden of osteogenesis imperfecta, a heritable skeletal disorder colloquially known as “brittle bone disease.”
The project kicked off in 2024, and Dr Luca Sangiorgi outlined how the partnership was structured and has advanced thanks to a milestone approach – combining tangible targets with more ambitious and mid/long-term goals.
The project will measure the impact of the disease on patient activity and quality of life using digital settings in real world settings, with a combined approach of gait analysis and sensors. The project partners will also jointly engage in early scientific dialogues with other stakeholders (such as regulators and HTA) to gain insights into approaches to foster patient access to innovative medicines.
Previous webinar from our series
In December 2024, Prof. Alberto Pereira, Dr Franz Schaefer and Katarzyna Mosiewicz outlined the work of ERNs, detailing how and where ERNs can serve as research partners to industry and presenting the resources, data repositories and clinical expertise available within ERNs. To learn more about our first webinar, click here.