Rare Disease Watch recognises that people living with rare diseases, their families and carers have knowledge and experience that can contribute to research, healthcare, policy and organisational decision-making.
Their involvement can help identify problems that may not be apparent to researchers, clinicians or organisations working outside the day-to-day experience of a rare condition.
Patient involvement should be meaningful, respectful and appropriate to the activity. People should understand why they are being invited to contribute, how their views may be used and whether compensation or reimbursement is available.
These principles explain how Rare Disease Watch approaches patient and carer involvement, including voluntary participation, paid contributions, accessibility, independence, confidentiality and the responsibilities of organisations seeking to engage with the community.
They form part of the Rare Disease Watch Governance Framework.
Patient involvement refers to activities in which patients, carers or advocates contribute their knowledge, experience or perspectives to inform decisions, research, services, resources or organisational activities.
It is different from simply receiving information or accessing a service.
Patient involvement may include:
The nature and extent of involvement should be clear before an individual agrees to participate.
Patient involvement is also distinct from participation as a research subject. Different ethical, regulatory and consent requirements may apply to research participation.
Rare Disease Watch approaches patient involvement according to the following principles.
Respect
Patients and carers should be treated as individuals with relevant knowledge and experience, rather than simply as sources of information.
Voluntary Participation
Individuals should be free to decide whether to participate and should not be pressured to accept invitations.
Clarity
The purpose, expectations, time commitment and intended use of contributions should be explained in advance.
Fairness
Compensation and reimbursement arrangements should reflect the nature of the activity and should not create unreasonable barriers to participation.
Accessibility
Activities should take account of disability, fatigue, communication needs, caring responsibilities and other circumstances affecting participation.
Independence
Patients and carers should be able to express their views without pressure to support a particular organisational or commercial position.
Confidentiality
Personal and health information must be handled appropriately.
Accountability
Organisations seeking patient involvement should be clear about how contributions will be considered and, where appropriate, what influence they have had.
Rare Disease Watch distinguishes between several forms of participation.
Individuals may join the Rare Disease Watch community to access information, discover relevant organisations and participate in permitted community activities.
Community membership is not an employment or paid advisory arrangement.
Members are not expected to provide professional services simply because they have joined.
Individuals may choose to volunteer their time to support Rare Disease Watch's activities.
Volunteering is unpaid and governed by the Volunteer Statement.
A volunteer should not automatically be expected to undertake substantial specialist advisory work outside their agreed responsibilities without discussing the nature of that work.
Individuals may contribute personal experiences to articles, interviews, discussions or educational resources.
These contributions may be voluntary or compensated, depending on the nature, purpose and arrangements of the activity.
Patients and carers may be invited to provide expertise through formal advisory meetings, workshops, research consultations, service-development projects or other defined assignments.
Such activities may warrant compensation, particularly where they require preparation, specialist knowledge, substantial time or ongoing responsibilities.
Participation in clinical trials, observational studies or other research is governed by the applicable research protocol, ethical approvals, consent arrangements and regulatory requirements.
Rare Disease Watch's patient involvement principles do not replace those obligations.
Rare Disease Watch recognises that patient and carer expertise has value.
Compensation should be considered where an individual is asked to provide a defined contribution that goes beyond ordinary community participation or informal volunteering.
Relevant considerations include:
Payment should not depend on an individual expressing a particular opinion or supporting a predetermined outcome.
The purpose of compensation is to recognise the contribution, not to purchase agreement.
Where compensation is offered, the arrangements should be explained before participation begins.
Information should include:
Compensation may take different forms depending on the activity, country and applicable legal requirements.
Rare Disease Watch does not prescribe a universal payment rate for every form of patient involvement.
Where external organisations commission patient involvement activities, compensation should be agreed in advance and should reflect the work being requested.
Individuals should not be required to negotiate payment after completing an activity where compensation was expected.
Responsibility for Compensation and Expenses
The organisation commissioning a patient or carer involvement activity is responsible for paying all agreed compensation and reimbursable expenses, unless Rare Disease Watch has expressly accepted that responsibility in writing.
Payment amounts, eligible expenses, payment arrangements and timescales must be agreed before participation begins.
Where Rare Disease Watch facilitates an introduction or coordinates an activity, this does not, in itself, make Rare Disease Watch responsible for payment.
Participants must be informed which organisation is responsible for payment before agreeing to take part.
Reimbursement is distinct from compensation.
Compensation recognises an individual's time, expertise or contribution. Reimbursement covers agreed costs incurred because of participation.
Eligible expenses may include:
Not every activity will involve reimbursement.
The responsible organisation should explain which expenses are covered, whether prior approval is required and how claims should be submitted.
Where possible, arrangements should avoid requiring participants to incur substantial costs before receiving reimbursement.
Rare diseases can affect individuals in different ways.
Some participants may experience fatigue, pain, mobility restrictions, sensory difficulties, cognitive effects or fluctuating health.
Others may have significant caring responsibilities or limited access to technology.
Organisers should consider appropriate adjustments, including:
Adjustments should be discussed with participants rather than assumed.
Where accessibility requirements involve additional costs, responsibility for those costs should be clarified before participation.
Before agreeing to a structured involvement activity, individuals should receive sufficient information to make an informed decision.
This should include:
Participation should be voluntary.
Individuals should be able to decline an invitation without losing access to ordinary Rare Disease Watch community services.
The consequences of withdrawing after an activity has begun, including any effect on agreed payment or the use of contributions already provided, should be explained where relevant.
Patients and carers must be able to express their experiences and views without being required to support a particular conclusion.
Organisations should not select, edit or present contributions in ways that materially misrepresent what participants have said.
Where an activity involves discussion of medicines, medical devices or healthcare services, participants must not be pressured to endorse products or treatments.
Compensation must not be conditional on providing favourable feedback.
Participants should be informed where an activity is funded or organised by a commercial organisation.
Relevant conflicts of interest should be disclosed and managed.
Related documents:
Rare Disease Watch may facilitate appropriate opportunities for patients and carers to contribute to activities organised or funded by pharmaceutical, biotechnology, healthcare or other commercial organisations.
Commercial involvement must be transparent.
The purpose of the activity, the identity of the commissioning organisation and the proposed use of contributions should be explained before participation.
Commercial organisations must not use Rare Disease Watch's platform to obtain unrestricted access to patient information.
Members must not be treated as a commercial contact database.
Any introduction should follow the organisation's membership, consent and data protection arrangements.
Payment for access to a Rare Disease Watch service does not entitle a commercial organisation to patient participation, personal contact information or favourable opinions.
Where a commercial organisation commissions an activity, responsibility for compensation, contracts, regulatory compliance and other obligations must be clearly allocated.
Rare Disease Watch may share information about relevant opportunities or facilitate introductions between interested individuals and organisations.
Participation should be based on individual choice.
Members should not be contacted for involvement activities outside the applicable platform permissions and agreed communication arrangements.
Where Rare Disease Watch facilitates an introduction, the member should have an opportunity to accept or decline it.
Personal contact information should not be disclosed without appropriate authority and a lawful basis.
The organisation must not guarantee that an individual will be selected for a project or research activity.
Related document:
Membership & Introduction Policy
No individual patient or carer can be expected to represent every person living with a particular condition.
Experiences may differ according to diagnosis, disease severity, age, location, healthcare access, socioeconomic circumstances and other factors.
Rare Disease Watch encourages organisers to consider whose perspectives are being included and whose may be absent.
Where an activity requires a range of experiences, recruitment should be designed accordingly.
Organisations should avoid assuming that one participant's experience reflects the views of an entire patient population.
Participants should not be described as official representatives of a patient community unless they have an appropriate representative role or mandate.
Patient organisations and advocates can contribute important knowledge about community needs, healthcare systems and research priorities.
Their involvement may differ from that of individual patients or carers.
An organisation may participate through a designated representative, while an individual advocate may contribute personal experience, professional expertise or both.
The capacity in which a person is participating should be clear.
Where compensation or funding is provided to an organisation, the arrangement should be distinguished from payment to an individual participant.
Any relevant financial or organisational interests should be disclosed.
Patient involvement may involve sensitive personal information, including medical histories and experiences of healthcare.
Participants should be informed about what information is collected, why it is needed, who will have access and how it may be used.
Only information necessary for the activity should be requested.
Health information requires particular care under applicable data protection legislation.
Where identifiable contributions are to be published, appropriate publication permission and a lawful basis for processing must be established.
Participation in an activity does not automatically authorise public disclosure of a person's diagnosis, identity or personal circumstances.
Information must not be reused for unrelated commercial, research or promotional purposes without appropriate legal justification and safeguards.
Related document:
Some involvement activities may result in reports, publications, recordings, presentations or other publicly accessible material.
Participants should be told in advance whether their contributions may be quoted, recorded, attributed or published.
Where appropriate, participants should be given an opportunity to review how their personal experiences are represented.
Permission to participate in a meeting does not automatically constitute permission to publish identifiable information.
Any limitations on withdrawal or removal of material after publication should be explained clearly.
Where patient involvement produces written material, presentations, recorded content or other original work, the intended ownership and permitted use should be explained.
Participants should understand whether their contributions may be reproduced, edited, shared or incorporated into reports and other materials.
Copyright and other intellectual property rights should be addressed through appropriate agreements where necessary.
Payment does not automatically transfer copyright or establish unrestricted rights to reuse a person's work.
Organisations must not assume that participation permits unlimited commercial use of contributions.
Patient involvement activities must not be presented as a substitute for healthcare.
Participation must not be used to provide individual medical diagnoses or treatment recommendations through Rare Disease Watch.
Research and commercial partners remain responsible for their own professional, ethical and regulatory obligations.
Activities should be designed to minimise unnecessary distress, pressure or risk to participants.
Where sensitive subjects are discussed, organisers should consider appropriate support and clear arrangements for responding to concerns.
Any immediate safeguarding concern should be handled through appropriate reporting procedures.
Related document:
Safeguarding & Code of Conduct
Compensation may have different implications depending on an individual's country of residence and personal circumstances.
Payments may affect taxation, welfare benefits or other financial arrangements.
Rare Disease Watch cannot provide individual tax, benefits or legal advice.
Participants should be informed that they may need to consider the implications of receiving compensation.
Where relevant, organisations commissioning activities should provide clear payment documentation and comply with applicable legal requirements.
Compensation arrangements must also take account of any applicable restrictions concerning healthcare professionals, research participants or other regulated activities.
Patient involvement should not be assessed solely by the number of people who attend meetings or participate in consultations.
Where appropriate, organisations should consider whether contributions have influenced decisions, changed plans, identified previously overlooked issues or improved the relevance of an activity.
This may involve asking:
Not every suggestion can be implemented.
Meaningful involvement requires an honest account of what participation could influence and what happened afterwards.
Rare Disease Watch encourages organisations to provide appropriate feedback to contributors rather than allowing participation to end without explanation.
Organisations seeking patient involvement through Rare Disease Watch are responsible for conducting their activities lawfully and ethically.
Depending on the arrangement, responsibilities may include:
Rare Disease Watch may decline or discontinue involvement in an activity where adequate safeguards are not established.
Facilitating an introduction does not automatically make Rare Disease Watch responsible for every aspect of an external organisation's project.
Its responsibilities will depend on its actual role, applicable law and the relevant agreements.
Participants should have access to an appropriate route for raising concerns about involvement activities.
Concerns may relate to payment, consent, confidentiality, conduct, accessibility or the way contributions have been used.
Where an external organisation is responsible for an activity, its complaint arrangements should be explained to participants.
Concerns involving Rare Disease Watch may be raised through the organisation's designated reporting arrangements.
Complaints should be assessed fairly and with appropriate regard to confidentiality and conflicts of interest.
Related document:
Rare Disease Watch's executive leadership is responsible for implementing these principles within its delegated responsibilities.
Activities involving significant commercial, legal, financial or safeguarding implications may require additional organisational approval.
The operating company's director or directors retain ultimate corporate accountability.
The Advisory Board may provide advice on patient involvement practices but does not exercise automatic authority over individual activities or compensation decisions.
These principles form part of the wider Rare Disease Watch governance arrangements.
They should be reviewed when significant changes occur in the organisation's activities, relevant legislation, commercial arrangements or patient involvement practices.
Material amendments require approval by an appropriately authorised representative of the organisation.
Organisational Governance
Partnerships and Participation
Editorial and Information Standards
Conduct and Accountability
