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HD InsightsWalking the HD Walk: Community Advisory Board

My connection with the HD community began in 1982, when I learned that the mysterious condition my mother had was Huntington’s disease and reached out for answers and support to the Norwegian Huntington Association (LHS). Located in my home country of Norway, the LHS was new at that time, and staffed solely by volunteers.

For me, connecting with them was a life-changing experience, meeting others who helped me understand what this disease was about for the first time. They helped guide me in learning how to cope — how to deal with caring for my mother and with the risk of having the same disease myself.

advisory board

In 2016, I became the President of the EHA (which later became a full-time paid position). In 2021, EHA formed a partnership with the International Huntington Association (IHA) and the Huntington Youth Organization (HDYO)
and set up the Huntington’s Disease Community Advisor Board (HD-CAB), a group of patient and family advisors who provide input to industry and other stakeholders in drug development.

While the HD-CAB members come from all over the world, the majority come from North America and Europe. Many of these HD-CAB members have served throughout my tenure. It’s been a really fantastic journey and I feel very privileged to be to be able to do this.

Community Advisory Board Origins and Mission

The HD-CABs are groups of 6-15 people from the HD community who serve as a sounding board and advisor to the trial sponsor, helping them with successful recruitment and retention. They advise sponsors on what will make potential trial participants more or less likely to enroll and what they will need in order to be motivated to see it through. It is part of my role to oversee these industry partnerships.

Our CAB members are all connected to their respective communities in different capacities. Some are running a blog, some are doing a lot of campaigning on social media, some are really active in their in their sustaining associations. They are not just connected to HD through their own status or their families’, but also through a broader HD community setting. We recruit them from that community and bring them together for trainings and online meetings to be prepared to serve as advisors to industry sponsors.

A goal of ours is to include family members who have varying relationships to Huntington’s disease — having symptomatic HD, having tested positive and being pre-symptomatic, or having tested gene-negative, being at-risk but not knowing their gene status, or being a carrier or a partner to somebody with Huntington’s disease. We especially want a lot of people who know that they are gene-positive, and people who have clinical trial experience.

We expanded our representation because throughout the years that I’ve been active on the European and international stage, there has been an explosion in the development of clinical programs, and more and more, we would be asked, “Do you have somebody we can consult on questions of patient perceptions of our intervention, or can you do it?”

We wanted to create a group that was able to give more profound input in this representation and provide excellence
in terms of organizing ourselves, training ourselves, and supporting ourselves to fulfill that role and be that coordinating body they wanted.

What Sponsors Want

We advertise or get the word out that we now have this CAB resource in the HD community, and also, of course, whenever and wherever we meet with industry. Often, companies just hear about us and contact us. We might be working with them informally through some regular calls, and we come to a point where we ask if they could use the services of a CAB.

Just last year, we had 37 people trained and available to serve on an HD-CAB, and we worked with six different companies. Meetings with sponsors may take place sporadically, as needed, but are heavy on the front end. One CAB member may be advising on just one, or up to three or four overlapping trials at any given time.

Patient input is not compulsory, but I think the regulators, both in the United States and certainly here in Europe, see the value. Optimally, we get involved early in the sponsor’s planning process to provide our knowledge and in-depth understanding of the implications the study might have for HD patients. Some of our input comes really early in the preclinical stages, but sometimes we come in at different points. Truly, we’re happy when people want to listen to our voices at whatever point. As the trials progresses, sponsors often come back to us because they need new input to answer new questions.

We really allow the company or the other sponsors to see into the HD community and the lived experience. So, what we provide is in-depth insight into what is it like to have HD in a family and be a patient or their loved one, and then what the HD community, at large, is thinking.

It’s very often the case that industry sponsors would like to have people from specific countries where they are planning or running a trial, and we may try to broaden their perspective. Honestly, our experience is that geography isn’t necessarily hugely important in relation to most of the discussions we have, so generally, this becomes a non-issue. Coping with HD implies the same sense of being isolated, the same uncertainty, the same search for knowledge and support, no matter whether you live — in Ohio, Norway or Abu Dhabi.

 There is a strong argument for a company to bring you in early instead of just asking for your feedback later. We hope trial sponsors choose to invest in the patient community in this way in the early phases, to generate interest, motivation and knowledge in support of their recruitment.

Mechanics of the CAB Member Role

The easiest way to describe the mechanics of the HD-CAB and my role is to give a recent example of our part in a trial HSG was doing with a biosciences company on a new drug. HSG contacted us and said the client wanted to use a patient advisory board, and wanted this to be face-to-face, to the degree possible. 

There were about 14 of us in the CAB group for this study, and we met on a hybrid basis because some lived quite far away. We were asked to react to the protocol, the drug, and how it works. The company probed how well we thought potential participants would understand the goal and process, and whether there were some common misunderstandings out there they should address.

They asked practical questions involving the protocol. Can the people get off work to do what is required, perhaps staying at the collection or treatment site for quite some time and regularly? What potential hurdles or needed supports did we see?

In this trial, as in many, the route of administration is often a topic for discussion. Specifically, they wanted to know how the participants would likely respond to the intervention. Would it be “easy-peasy” or too invasive for them to assent to do? Is doing a spinal tap or spinal injections acceptable? What about brain surgery to do gene therapy? What if this kind of treatment takes two hours, four hours, etc.? Is that acceptable? How can we communicate all of this properly?

So, we provide kind of a market analysis to answer the larger question: Are there people out there willing to do what I am intending to offer them in a trial?

Then we have had discussions about what participation would look like. Who would really want this treatment and what kind of procedural evidence would they require us to demonstrate to them? Where would these treatments take place? The duration of the trial is also something we very often discuss, looking at the feasibility of retention.

The questions asked by the sponsor go to to each CAB member individually, but then we can discuss them, often asking one another what they think people in their families would like. Ultimately, CAB gives an answer on behalf of a whole patient population.

Interested in Serving on a CAB?

Most CAB members generally have full-time jobs, if they can work, but they are compensated for their time when they serve on a CAB assignment. We always require that from the company because we want to value people’s time. However, CAB membership and training and the social things we do together are just as volunteers.

Required training involves ongoing readiness and digital training, to the tune of four or five trainings a year, and these happen virtually all over the world. You will learn things that are not public knowledge, so you commit to confidentiality.

The job is demanding because you have to focus and work on finding, defining and supporting your message and how to best communicate it among CAB members and in the group communications to the sponsor. It’s not something you just drop into. It involves preparation, but it’s also really rewarding. It provides really a unique opportunity to potentially influence how things are done to help ensure high quality and successful trial completion.