Table 2 How Together4RD is addressing barriers to ERN-industry collaboration

Concerns on the part of some members of the ERN BoMS concerning Industry and ERN interactions: stemming from a lack of awareness of the nature such collaborations can take, and of the current extent of successful collaborations outside of the ERNs

Development of rare disease case studies to illustrate a range of examples in which networks (at national and international level) engage/have engaged with Companies for particular purposes

Disseminating these case studies to the BoMS, to show that win:win collaborations indeed exist

Concerns from the BoMS (and indeed from ERNs and patients themselves) over conflicts of interest

Initiating ERN-Industry engagement via Together4RD pilots, using the Code of Conflict developed by the WG on Legal and Ethical Issues, should appease concerns

Possible lack of awareness or clarity on the range of activities which could take place between ERNs and Industry

Utilising the case studies to more clearly distil the sorts of activities which could feasibly occur between ERNs (specifically, moving forwards) and Industry, to support the development of palatable pilot proposals the BoMS members would support

Concerns amongst BoMS that opening the door to ERN and Industry interactions would only benefit a few of the already more research mature ERNs

Together4RD will explore launching a multistakeholder forum to enable ERN and Industry interactions and advance strategic (and increasingly operational) collaborations across the board. Together4RD will, with the stakeholders involved, agree a preferred model for this

Administrative and bureaucratic efforts and time required to contract with Companies (exacerbated in the absence of a readily-available legal entity)

Planned development of standardised templates all ERNs could use (by making use of a designated HCP or Third Party) to contact with single Companies or multiple Companies

The fact that ERNs are not legal entities, and it appears that the European Commission is not seeking to make them so (in the near future at least)

The pilots showcased by Together4RD will demonstrate how either a consortium model or third party agreement may serve to deliver the results ERNs and Companies wish to see. It may be concluded that different Frameworks suit different sorts of activity, but either way, guidance -for now and for the future- will be proposed by Together4RD in the light of the pilot experiences

Inadequate funding for ERNs limits scope and ambition to engage in research in a meaningful way

Under the EU4Health programme, greater financial resources (provided through more amenable and appropriate and less bureaucratic grant processes) is already relieving pressure on the ERN coordination teams, which should serve to stabilise the core Network structures and services. Additional public funding is expected from the future RD Partnership and eventually from Member States to support ERN research activities and data collection. Coupled with a means of obtaining private funding for mutually-beneficial research activities, accelerated by Together4RD, and bolstered by a robust Code of Conduct issued by the Working Group on Ethics and Legal Issues, the hope is that resourcing becomes less of a barrier to ERNs fulfilling their potential

Lack of certainty on the part of ERNs of what they are able to do with Companies, and how to approach different activities

The disease-related pilots selected to be showcased by Together4RD will be closely followed and analysed, to distil good practices and lessons learned, which should serve to optimise all future interactions and should be illuminating for less-experienced Networks

ERNs and their potential are not always well understood—some companies, especially SMEs with limited European traction, are not aware of their existence or if they are, do not realise the breadth and depth of expertise ERNs offer

This Position Statement in itself should begin to raise awareness amongst the broader private sector. Foreseeably the Moonshot (and future RD Partnership, if Industry is able to eventually play a meaningful role) will also serve to boost this awareness-raising. The Together4RD pilot on a forum/fora by which ERNs and Industry can connect transparently and discuss needs and strategies with patients and other key stakeholders, should also address this challenge. And as Together4RD pilots are delivered, presumably increasingly word will spread of the ERNs in SME circles even outside of Europe

Limited basic research and Industry interest in the conditions addressed by a given ERN, which traditionally has therefore had limited research activity

Together4RD will offer benefits here firstly by simply networking the various stakeholder groups involved, and opening up the conversation. A pilot providing either a cross-ERN forum for strategic Industry discussion, or ERN-specific multistakeholder fora, will create a space for all ERNs and thus all disease domains to identify research gaps and meaningful patient-centred needs, and foreseeably make it easier to devise projects to begin to address these and build momentum for neglected conditions (which potentially could be supported through the future RD Partnership, Moonshot or other avenue)