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Rare Disease Consortium

Because patients and families are waiting

Meanwhile, patients (more than half of whom are children), their families and caregivers anxiously await advances in treating these serious and often life-threatening conditions.

We share their sense of urgency

At Syneos Health®, helping companies develop rare disease therapies is something we take personally. It takes novel and integrated approaches, including early engagement of key stakeholders and patient perspectives to accelerate products to market and de-risk the development of critical patient therapies.

That’s why we created the Syneos Health Rare Disease Consortium

The Consortium facilitates streamlined access to the full range of our experience in rare disease clinical development and commercialization.

We established the Consortium to integrate our strong medical, operational, regulatory science and commercial expertise—all of which is enriched by our collaborative relationships with investigative sites, treating physicians, patient advocacy groups, academic thought leaders and sponsor companies.

Better together: a collaborative team powered by rare disease drug development and commercialization experts

Our regulatory experts recommend and support the best regulatory pathway to obtain regulatory agency support through the clinical development plan and commercialization

Our operational team has the experience to support patient localization and access to clinical trials and registries, creating customized strategies at the level of each patient

Our partnerships with niche vendors support customized rare disease strategy logistics and ensure the patient remains at the center of everything we do

Our medical expert teams provide medical and scientific input on partnering to ensure a strong development plan

Our commercial teams bring insights to bear early in the development process and through to commercialization to optimize prioritization strategy, pricing and reimbursement, public policy and engagement with patient advocacy groups and other stakeholders

This collaborative group harnesses the power of our physicians, scientists, commercial strategists and key professionals to ensure a holistic view — from clinical trial planning and execution through successful commercialization.

Extensive hands-on experience and specialized knowledge

In the past 5 years, we have conducted more than 350 projects in rare disease indications, including:

>30 Real World Evidence projects, including registries, noninterventional studies and expanded access programs

~25% of our rare disease research experience is in the pediatric population, including first-in-patient studies in ultra-rare indications

~20 projects with ATMPs (Advanced Therapy Medicinal Products)

>55 cell therapy studies and >60 gene therapy studies

Our Rare Disease Consortium can help make a difference.

Approximately 50 percent of rare diseases affect children, 30 percent of whom will not live to see their fifth birthday

Our experts in rare disease and pediatrics often collaborate to meet the uniquely complex regulatory and ethical challenges involved in pediatric drug development. Read about how our Pediatrics Consortium helps shorten the distance from lab to life for pediatric therapies.

Navigating the challenges unique to cell and gene therapies

Cell and gene therapy (CGT) represents a new frontier in the fight against many devastating diseases, including rare genetic disorders and certain cancers. Our Cell and Gene Therapy Consortium provides the cross-functional capabilities needed to navigate the challenges unique to CGT clinical trials, accelerate their development and deliver on their promise.

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