Experience matters in rare disease research. Take advantage of our agile team’s extensive experience in delivering clinical trials for rare conditions across multiple therapeutic areas. Our full-service solutions are tailored, adaptable, and designed to integrate closely with the specific needs of your patient, patient community, and study stakeholders.
Delivering a clinical trial for a rare condition requires a nuanced, practical and integrated approach.
Our established partnerships with specialist NHS hospitals, UK Biomedical Research Centres and Clinical Research Facilities (CRF) enable us to quickly identify sites with the right experience in recruiting and retaining patients for your trial.
Accelerate your path to approval with our bespoke services:
OPTHALMOLOGY
Phase I Novel Treatment
One of the largest clinical studies of patients with Birdshot Uveitis (eye disease) in the UK running at Moorfields Eye Hospital and University Hospital Birmingham.
ONCOLOGY
Phase I Advanced Therapy Trial
A novel new treatment therapy INKmune, for patients diagnosed with Myelodysplastic Syndrome or Acute Myeloid Leukaemia.
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GENE TRANSFER
Phase I ATMP study
An open label dose-escalation study of a self complementary adeno-associated viral vector for gene transfer in patients with haemophilia B
IMMUNOLOGY
Phase I Rare Disease
Trialling Secukinumab, a monoclonal antibody that selectively targets interleukin-17A (IL-17A), a cytokine involved in the inflammatory processes underlying Behcet’s disease – a rare autoimmune disease
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ONCOLOGY
Phase I/II Interventional
A new transplant pre-conditioning technique TRALA (Targeted Radiotherapy for AL-Amyloidosis) that may result in an improvement in the response rate posttransplant for S-ALA patients
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FABRY DISEASE
Phase I-IV
Our in-house team has experience in running Fabry disease clinical trials in the UK and ROW. From our collaboration with the Rare Disease Research Partners and the MPS Society we can provide enhanced trial design for the sponsor, patients and sites.
"The most collaborative CRO I've worked with. PHARMExcel bothered to know us as a client and understands our studies inside out, making it an efficient and friendly partnership."
Prof Mark Lowdell, CSO INmune Bio Inc
"PHARMExcel’s passion for their projects, the flexibility shown by the team, combined with direct access to the CEO, sets PHARMExcel apart from other CROs."
Daniel Green, Imperial College London
"It has been a pleasure working with Yvanne and the team. The personal approach and attention to detail have given us confidence to ensure patient safety and manage the risks of the Trust acting as Sponsor in particular for the early phase trials. "
Karen Underwood & Prof Saul Faust-Sponsor (OBE), Southampton Hospital
"The support from PHARMExcel has been extremely high quality. This includes all aspects trial development, management and monitoring, that was also evidenced by a recent sponsor audit. We look forward to working with PHARMExcel going forwards. "
Tom Fletcher, Liverpool School of Tropical Medicine
We are dedicated to advancing research and improving access to innovative therapies for individuals with rare conditions. We are excited to announce the expansion of our rare disease initiatives through new collaborations with UK-based research charities and non-profit organisations, including the MPS Society and Rare Disease Research Partners.
Our passion for supporting rare disease research is exemplified by our proud sponsorship of the Fabry Matters Research Grant. By leveraging our strengths as a CRO and partnering with research experts, local communities, healthcare professionals, patients, and their families, we aim to enhance trial experiences and accelerate the delivery of novel therapies to those in need.
