Rare Disease Research Partners
Not-for-profit research and medical communications organisation supporting patients, families, patient organisations and pharmaceutical sponsors in rare and complex diseases. Services include patient-centred clinical trial support and logistics, managed-access programme implementation, medical communications and evidence generation (real-world data and patient-reported outcomes), stakeholder engagement, and advisory support for health-technology assessment and reimbursement.
Industries
Nr. of Employees
small (1-50)
Rare Disease Research Partners
Services
Coordinated patient logistics and case management for participation in local and international clinical trials, including travel, accommodation, relocation, reimbursement and 24/7 support.
Design and operational delivery of managed access agreements including PRO collection, data management, analysis, and preparation of reports for health authorities.
Design and delivery of primary research and evidence synthesis in rare diseases, including global patient surveys, PRO instrument deployment, qualitative studies (interviews, focus groups), literature reviews, audits and consensus statement development. Services include multilingual materials, ethics-reviewed protocols and participant-administration logistics.
Development of patient-facing materials, scientific manuscripts, conference content, educational resources and symposia to communicate research and clinical findings.
Advisory services including patient engagement strategies, HTA evaluation support, advisory board planning, stakeholder contact and communication strategies.
Counselling services delivered in collaboration with specialist mental-health partners to reduce participant and family stress related to trial participation and to support retention; includes accredited counsellors and lead-counsellor contact model. Available for English-speaking participants in UK and Europe (regional exemptions may apply).
Coordinated patient logistics and case management for participation in local and international clinical trials, including travel, accommodation, relocation, reimbursement and 24/7 support.
Design and operational delivery of managed access agreements including PRO collection, data management, analysis, and preparation of reports for health authorities.
Design and delivery of primary research and evidence synthesis in rare diseases, including global patient surveys, PRO instrument deployment, qualitative studies (interviews, focus groups), literature reviews, audits and consensus statement development. Services include multilingual materials, ethics-reviewed protocols and participant-administration logistics.
Development of patient-facing materials, scientific manuscripts, conference content, educational resources and symposia to communicate research and clinical findings.
Advisory services including patient engagement strategies, HTA evaluation support, advisory board planning, stakeholder contact and communication strategies.
Counselling services delivered in collaboration with specialist mental-health partners to reduce participant and family stress related to trial participation and to support retention; includes accredited counsellors and lead-counsellor contact model. Available for English-speaking participants in UK and Europe (regional exemptions may apply).
Expertise Areas
- Clinical trial management and patient logistics
- Patient survey design and administration
- Qualitative research (interviews and focus groups)
- Managed access programme implementation
Key Technologies
- Patient-reported outcome measures (PROs)
- Online survey platforms
- Multilingual survey deployment
- Qualitative research methods (interviews, focus groups)
News & Updates
Announcement of NICE recommendation for routine NHS use of an enzyme-replacement therapy for MPS IVA following data collected through a managed access programme.
Publication of a consensus statement and literature review addressing psychological support needs of families and caregivers at the time of rare-disease diagnosis.
Webinar presenting considerations for HTA and managed access agreements aimed at improving access to rare-disease treatments.
Description and participant invitation for a global survey (ATP study) capturing patient and caregiver experience of TK2d, including multilingual survey deployment, ethics review, and participant information sheets. The study provides participant support contacts and describes participant eligibility and incentives.
Announcement of NICE recommendation for routine NHS use of an enzyme-replacement therapy for MPS IVA following data collected through a managed access programme.
Publication of a consensus statement and literature review addressing psychological support needs of families and caregivers at the time of rare-disease diagnosis.
Webinar presenting considerations for HTA and managed access agreements aimed at improving access to rare-disease treatments.
Description and participant invitation for a global survey (ATP study) capturing patient and caregiver experience of TK2d, including multilingual survey deployment, ethics review, and participant information sheets. The study provides participant support contacts and describes participant eligibility and incentives.