A report on the EURORDIS Round Table of Companies, Barcelona October 16, by Maarten Kraaijenhagen 

Twice per year, EURORDIS organizes the European Round Table of Companies, bringing together industry, patient advocates, public officials and other experts to address patient needs.

Today, ERTC members include over 70 companies, from start-up biotechs to mid-sized and large pharmaceutical companies headquartered across the globe.

This year’s October meeting was held in Barcelona and the theme of the meeting was:
Patient engagement in the product life-cycle and community advisory boards (CABs).

Parallel Concerns and Possible Solutions
It was apparent from the start of the conference that the PARADIGM project is working to help address many of the concerns that were the subject of the day.  However, it was not completely clear how PARADIGM and the CABs relate to each other and if any synergies between them might be developed or formalized in the future. This will need further exploration.

The day was opened by Yann Le Cam, CEO of EURORDIS, who underscored the key activities for EURORDIS which are to empower patients through training, to advocate for patients with rare diseases at the national and international levels, and to engage patients in the product lifecycle, healthcare ecosystem and social policies.  Yann shared the outcomes of an International study amongst Rare Disease (RD) patients on the participation of RD patients in clinical research, showing the main obstacles to Rare Disease research: lack of public funding, lack of private funding and small patient populations. Yann then shortly mentioned PARADIGM as a best practice to continue to talk about the importance of the Community Advisory Boards (CABs).

Perceptions of patient engagement:

Outcomes of research on patient engagement from the pharmaceutical industry and patient association perspectives show that:

  • In about 5 years’ time, both patient associations and pharmaceutical companies are predicted to be more aligned in their desired distributions of patient engagement across the medicine lifecycle phases.
  • Both stakeholders find that legal and compliance are seen as a key barrier for pharmaceutical companies
  • Patient associations attributed the perception of patient engagement slowing down drug development as a barrier to engagement by pharmaceutical companies.  Pharmaceutical companies themselves did not identify this as a barrier.
  • Pharmaceutical industry respondents acknowledged that they have yet to fully integrate a patient-centric model (they are interested, but there are challenges in implementation!)

Early HTA discussions:

The importance of patient involvement in early HTA discussions was highlighted, which links very well to one of the three objectives of PARADIGM. Questions such as: “are patients stakeholders or are they the decision makers?” and “are patients target users of HTA and should they be involved as such?” were discussed.

Multi-stakeholder collaboration:

Vinciane Pirard from Sanofi-Genzyme presented on the objectives and status of project PARADIGM and made a good case for the necessity for collaboration between all the different stakeholders.

The importance of this was confirmed by representatives of patient organizations, pharma companies and regulators who all gave very compelling examples. A French regulator spoke about how big the impact had been of the French patients’ representatives’ organization (TRT5) in the field of HIV infections with regards to early drug access for patients in medical need and the continuous challenge of the benefit/risk assessment in drug regulation. Another example of collaboration between a patient organization and a pharmaceutical company was presented from both stakeholders’ angles which showed how crucial a multi-stakeholder approach is when trying to reach important goals all of which underlined the importance of Project PARADIGM.

Community Advisory Boards (CABs):

Community Advisory Boards have existed for some decades and originate from the different communities facing the HIV/AIDs epidemic. The first CABs developed in parallel with HIV/AIDS activism and drug development. In this area, patient engagement has proven a critical success factor in improving the outcomes of clinical research. CABs have evolved and could continue to play a critical role in the medicines development lifecycle. The principle of a CAB is proactive involvement from patients and companies involved in the development of new medicines and therapeutic strategies.

In order to achieve this a memorandum of understanding, which would serve as a conceptual, non-binding agreement between companies and EURORDIS, could be signed describing everyone’s responsibilities and duties. In addition, the EURORDIS Charter for Clinical Trials outlines a collaboration based on trust and transparency, and a commitment between the sponsor to work together with patient networks throughout the research and development process:

“The essence of the EURORDIS program for Community Advisory Boards is to ensure patient engagement is done in the right way, building on the experience of those who pioneered the activity”.

This quote highlights not only the importance of the patient engagement work done in the past, but also indicates how important it is that outcomes of current projects like PARADIGM and the implementation of CABs should be aligned with each other and with the multiple stakeholders involved, in order to create the best possible outcomes backed by shared consensus of all stakeholders involved. François Houÿez, EURORDIS, outlined the construct of the program and how to initiate participation.  Short to mid-term actions were laid out after which the session concluded and the break began.

Over the course of the afternoon sessions, the EURORDIS EUROCAB program was laid out from the perspective of two patient advocacy groups and two pharmaceutical companies who recently have been working together within the CABs. Key takeaways highlighted the benefits CABs can offer both to patients as well as to the companies involved.

Benefits for patients and patient organizations include a way for patients to contribute with their view and expertise in an independent structured, transparent way, where they can have an impact on all stages of medicine development to ensure that clinical research takes into account their real needs and ultimately enable access to treatments.

For companies the benefits were, amongst others, the opportunity to get the patients’ perspective on the clinical development process and the post-approval data generation plan, to get feedback on the company’s pipeline and on how the portfolio meets the needs of patients, but above all, the opportunity to exchange on all sorts of situations and issues.  In many cases, patient representatives can signal risks and opportunities that can avoid delay and failure in CT developments.

CABs are a way to establish a two-way dialogue and to build trust. They are a platform to exchange scientific information and to receive patient perspective on all elements of the development program at all stages of the medicines development continuum.   

In order to be successful, it (1) demands real and long-term commitment and support from the companies, including from higher management levels, (2) mutual trust (3) continued motivation and perseverance from all parties involved and 4) early engagement. Confidentiality, transparency and the willingness to share information are also key elements for success.

Defining Key Issues an Ongoing Process:   

After this, three breakout sessions were held, where relevant and outstanding issues with regards to the CABs and the EURORDIS Charter for Clinical Studies in Rare Diseases were discussed.  Key issues related to operationalizing the model were identified:

  • Legal issues related to the implementation of the Charter:  contract versus Statement of interest?
  • Industry safeguards with respect to ensuring confidentiality, intellectual property, anti-trust, standardization of templates/documents, fair market value payments, etc.
  • Transparency, conflicts of interests and funding

These were very useful and interactive sessions which also made clear that there’s still a lot of discussions and in clarity on certain topics, especially with regards to the legal status of the EURORDIS Charter for Clinical Studies in Rare Diseases, which is a requirement to engaging with a CAB. It was clear that these issues still need to be resolved before the CABs can successfully be introduced within the Rare Disease area with a buy-in of all stakeholders involved.  Next steps discussed included the formation of a working group inclusive of the industry to better understand and to solve for the identified challenges. There was a shared vision for the value of a CAB model.

Maarten Kraaijenhagen is Senior Medical Director Rare Disease at Pfizer