This section highlights the importance of collaborative interactions between patient groups and medical professionals, to work together to achieve a common goal. Some examples of how this can be done, through advisory panels and committees, scientific articles/specialist meetings, and family days/patient events, will be shared.

Identifying and Connecting with Medical Professionals

In order to begin a collaborative relationship, patient groups may first need to connect with relevant medical professionals, if these relationships don’t already exist. There are several ways to reach out to and network with medical professionals:

• Attendance at specialist conferences or meetings for your rare disease area can help you identify and network with professionals who work in the field.
• Reaching out to genetic councillors and consultants, especially those who work with patients in your community, is a great way to connect with professionals who have an interest in your rare disease.
• Reaching out to specialist rare disease centres or clinics, such as those located at Birmingham Women’s and Children’s Hospital, Guy’s and St Thomas’ Hospital London and the Manchester Centre for Genomic Medicine, can help you identify rare disease specialists who may be interested in collaboration. When reaching out to a potential collaborator, it is recommended that you have a clear plan for the activities you will take on together, so you can ensure your priorities and interests match, to be open about what you are trying to achieve.

Collaborating on Advisory Panels and Committess 

An advisory board is a group of experts that provides advice, important insights and recommendations to a charity or company. Medical and scientific advisory boards can be very valuable to a patient group; patients and patient groups may be experts on the day-to-day impact of a condition but may not be so knowledgeable about the latest scientific advances and treatments for a condition. Therefore, gathering expert insight into these topics through a medical or scientific advisory board can be very valuable to a patient group and feed into plans for future projects.

Ataxia UK is a great example of a charity which has built both a Scientific Advisory Committee (SAC) and a Medical Advisory Panel (MAP); these perform different roles for the charity, allowing them to get expert advice on their research strategy, and to gather scientific and medical knowledge.

Scientific Advisory Committee

The main purpose of a SAC is to gather expert advice on a charity’s research strategy and projects. This is useful for:

• Ensuring funds raised by the charity for research will be spent as effectively as possible, e.g. through recommendations as to how money is allocated.
• Generating new ideas and expanding the charity’s network of researchers helping to raise the profile of the charity as a legitimate partner for research.
• Monitoring ongoing projects and reviewing applications and proposals for research funding

When setting up a SAC, it may be useful to discuss how the committee will function, including:

• How many times the committee will meet throughout the year.
• What the roles of each scientific member will be.
• The review processes for each member.

You may want to document this information in a terms of reference document, outlining a full list of the members of the committee, details on the roles and expectations, and any confidentiality and conflict of interest policies. More information on terms of reference documents can be found in the ‘Patient Group and Charity Collaborations’ guide.

As for many rare diseases, there are often only a small number of researchers in a country who focus on the condition. If only these researchers are invited to a SAC this could lead to conflicts of interest, e.g. when applying for funding. It is therefore recommended that you try to include other researchers, who have a general interest in the disease area you work in (e.g. broader knowledge of metabolic disorders, neurological conditions etc.) but aren’t specialists in your condition of interest. You might also want to consider inviting people with more general expertise, e.g. in statistics or clinical trial design, to ensure a broad range of viewpoints are shared.

Medical Advisory Panel

The main purpose of a MAP is to support the work of a charity or patient group, by giving scientific and clinical advice. This may involve:

• Responding to medical questions from patients.
• Producing medical guidelines.
• Advising the patient group on medical issues.
• Helping with policy statements and information leaflets.

A MAP is usually made up of medical professionals and specialists on a specific rare disease. Different types of medical professionals should be included to make sure the range of clinicians and specialists that patients will meet during their care and treatment are represented. If the condition is extremely rare and there are few specialist medical professionals, you might want to consider inviting anyone with an interest or expertise in a similar field or in one of the symptoms of the condition.

As medical professionals have very limited free time, the workload must be spread evenly between those involved by keeping track of tasks and how often they are participating in a meeting or project. When setting up a MAP, it may therefore be useful to discuss how the panel will function and set this out in a terms of reference document. More information on terms of reference documents can be found in the ‘Patient Group and Charity Collaborations’ guide.