Rare and ultra-rare disease trials

Specialists in rare disease studies

The complexities of managing rare disease studies require determination and adaptability from your Contract Research Organisation partner.

Rare disease clinical trials present exceptional challenges. Since treatments for rare or orphan diseases are often not available, even small studies can have a big impact. As a specialist rare disease CRO, we have a deep understanding of orphan and rare disease clinical trial designs. By applying an adaptive and resilient approach, we strive to deliver positive outcomes and help you to get the most out of every study.

Therapeutic acumen and dedication from your CRO are crucial for successful rare disease clinical research. Committed to patient-centric and novel rare disease trial designs, Simbec-Orion has been delivering complex clinical trials in rare and ultra-rare patient populations for over 27 years. Our expertise spans a range of indications including Duchenne muscular dystrophy (DMD), Amyotrophic lateral sclerosis (ALS), Huntington’s disease, Systemic Lupus Erythematosus (SLE) as well as rare oncology indications, and our studies.

All our rare disease trials are supported by our independent panel of world-leading rare disease Scientific Advisory Board

About our rare disease trials

A collaborative research approach

As rare disease specialists, we are proactive in understanding the specific needs of your orphan drug development program. Our dedicated rare disease Project Managers and specialists work as an extension of your team, enabling efficient communication and strong collaboration

Working in partnership from the outset, we will devise an optimal rare disease clinical trial design, focusing on study delivery that meets your specific objectives.

Rare disease trials are unique, so they need a bespoke approach. We apply the most effective methodology to achieve your target outcomes based on a wide range of tried-and-tested approaches. As a rare disease CRO, we have the experience, technology and expertise to provide a custom service based on your data.

Full rare disease clinical trial delivery

Simbec-Orion’s more than 27 years’ of experience in driving complex orphan and rare disease studies also gives us an in-depth understanding of the science, potential regulatory limitations, and operational challenges involved in orphan disease research. It means we will always seek to mitigate obstacles before they arise.

From conception to completion, we will design, manage and deliver your rare disease clinical trial programme, across early to final phases; devised for the specific challenges of rare disease clinical research, and always with an appropriate mitigation plan.

If you’re looking to collaborate with a specialist rare disease CRO, we’d love to learn more about your specific program requirements.

Contact us today.

Patient recruitment for orphan disease studies

With low and dispersed patient populations, recruitment for orphan and rare disease clinical research is specific and challenging. We stay committed to helping improve patient lives throughout, developing a tailored patient recruitment strategy to maximise the reach and potential of our rare disease clinical trials.

Our team includes dedicated recruitment specialists who collaborate closely with patient advocacy groups and clients, considering geographical, practical and patient care elements to ensure patient-centric rare disease trials that deliver the best possible results. We can also conduct decentralised trials, where appropriate, to make recruiting patients with travel or other limitations easier.

Through our established networks we are able to offer home visits to those for whom travel can be particularly challenging and stressful. Being able to participate in clinical trials without the additional stress of travel to clinical sites can support increased patient recruitment and retention by reducing this burden on patients and their carers who are already busy and time-limited

rare disease studies

Over 50% of rare diseases affect paediatric patients. Our rare disease clinical trial team are highly experienced with paediatric patients, and create tailored trial designs that work collaboratively within the demands placed on families. Our trial processes are designed to consider practical and geographical limitations, ensuring a fully patient-centric approach.

Handling rare disease clinical trials
with expertise

We have over 25 years experience in rare and orphan diseases and can handle complex clinical trials. We conduct phase 1-3 trials, including tailored patient recruitment strategy.

Orphan Drug Types

As a specialist CRO, we have managed rare disease clinical trials with all product types including vaccines, new chemical entities (NCEs), antisense therapy, small molecules, peptides, antibodies, genetically modified organisms (GMOs) and biologics, and gene therapy. No matter the focus of your rare disease clinical trial, we get the job done.

If you’re ready to collaborate with a specialist rare disease CRO, get in touch today.

Got a question?
Get in Touch or Submit an RFI/RFP

Our team is on hand to answer any questions you might have relating to the work we do at Simbec-Orion. Just fill in the form below, making sure to tick the boxes that apply, and we’ll respond as quickly as we can.

Looking to volunteer for a clinical trial?

Visit our dedicated volunteer website here, or email enrolmentservices@simbecorion.com

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