Rare disease CRO

Experts in navigating complex orphan and rare disease clinical trials across multiple global sites

Specialists in rare disease trials

We understand the complexities of managing rare disease studies, and the determination and adaptability required from your CRO. Simbec-Orion has been delivering complex clinical trials in rare patient populations for over 27 years, with expertise across a range of indications including Duchenne muscular dystrophy (DMD), Amyotrophic lateral sclerosis (ALS), Huntington’s disease, Systemic Lupus Erythematosus (SLE) and rare oncology indications.

Rare disease clinical trials present unique challenges, since treatments for rare or orphan diseases are often not available. However, it means that 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. Together, we can improve patients’ lives.

Therapeutic acumen and dedication from your CRO is crucial for successful rare disease clinical research. It’s why we are committed to patient-centric and novel rare disease trial designs. We work with you to improve patients’ quality of life with the support of a world-leading rare disease advisory board.

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A collaborative research approach

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

By working together, 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 for what you want to achieve 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

We have over 25 years’ experience in driving complex orphan and rare disease studies. Our in-depth understanding of the science, potential regulatory limitations and operational challenges involved in orphan disease research means we will always seek to mitigate obstacles before they arise.

From conception to completion, we design, manage and assess 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 speak with you.

Patient recruitment for orphan disease studies

Due to low patient populations, recruitment for orphan and rare disease clinical research is specific and challenging. We stay committed to helping improve patient lives, 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 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.

Paediatric 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.

Whitepaper - Practical Steps to Achieve Orphan Drug Designation Status

Accelerating the needs of rare disease clinical research

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Case Study 1

Phase II/III rare paediatric metabolic disease, Nieman Pick

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Case Study 2

Phase I/IIa rare respiratory disease, Idiopathic Pulmonary Fibrosis (IPf)

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Webinar – Orphan Drug Designation (ODD): Meeting The Unmet Need

If you missed our live Orphan Drug Designation (ODD) webinar you can view on demand now. Achieving ODD status is a significant step during the rare disease clinical trial process. This enables your product to be licensed faster, cost-efficiently, and achieve market exclusivity for 10 years.

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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.

Let’s collaborate

If you think we could be right for you, get in touch.

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