Rare Disease & Orphan
Indication CRO

Overcome challenges and drive efficient rare disease clinical trials by partnering with Medpace cross-functional experts

Overcome challenges and drive efficient rare disease clinical trials by partnering with Medpace cross-functional experts

Innovative Patient Enrollment and Retention Approaches for Rare Disease Clinical Trials

Rare disease clinical trials present a unique set of challenges in terms of clinical trial design, regulatory strategy, site selection, patient enrollment, and project management. These trials demand a highly experienced,  full-service CRO with innovative strategies to identify and engage sites and scarce patient populations, with expertise in site and project management to achieve study aims.

Drive patient recruitment and site selection for difficult-to-recruit studies with guidance from our experienced team of cross-functional rare disease experts, innovative study approaches, and strong site relationships.

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Making the Complex Seamless® in Global Rare Disease Clinical Trials

 Development of innovative rare disease therapies is often complex given the cutting edge nature of the product (i.e., cellular and gene therapies) or uncommon administrative routes (such as intracisternal or intrathecal). Sponsors from emerging biotechs to global pharmaceutical companies have trusted Medpace to lead their rare disease development programs across indications, spanning all ages, and in a spectrum of therapeutic areas, including but not limited to neuroscience, metabolic, cardiology, and ophthalmology. 

Cultivate efficiencies by leveraging our extensive experience in the design and conduct of Phase I-IV  global rare disease clinical trials.

Medpace rare disease experience over the past 5 years:


The Medpace Advantage

Partner with Medpace to successfully overcome the complexities of clinical trials in rare diseases through our expertise in enrolling rare disease patients, maximizing site selection, and implementing regulations and ethical standards into study conduct.

Rare Disease CRO Capabilities

As a global CRO for rare diseases with a cross-functional collaborative model and extensive experience, our team can support your clinical development through:

  • All phases of global clinical trials, in a variety of therapeutic areas, including, but not limited to, metabolism, neuroscience, hematology/oncology, cardiology, nephrology, immunology, and ophthalmology. These trials span the age continuum including pediatric populations and the most complex trial designs using traditional small molecule to advanced therapies.
  • Collaboration with Sponsors and regulatory bodies to identify and navigate the best pathways for approval in key regions including the US, Europe, Latin America, and Asia-Pac.
  • Strong site relationships to drive patient enrollment, with connections to many rare disease communities with access to patient registries to expedite site identification and patient recruitment in competitive landscapes.
  • ClinTrak®, Medpace’s proprietary Study Management system, provides aggregate data and reporting to support all aspects of a project to enhance and streamline study management.
  • Global regulatory understanding and consultation regarding the unique environment of rare disease studies.

Proactive Patient Recruitment & Retention

Patient recruitment and retention remain one of the most challenging hurdles in clinical development, especially for rare diseases. This is not only because of the rarity of patients but also because of the increasingly competitive landscape and research opportunities available to patients. The success of clinical trials is wholly dependent on the success of sites and the engagement of patients.

Medpace’s IntelliPACE® model synthesizes data from internal and external data sources to guide the selection of the best countries and sites for rare disease study participation. This data is further refined through Medpace expert analysis and input from sites and Key Opinion Leaders to align the target patient population with high-performing investigative sites.

These insights allow Medpace to provide Sponsors with a highly customized recruitment strategy tailored to the unique needs of each rare disease trial. Once the optimal strategy is defined, our in-house patient recruitment and retention team steps in to execute the customized recruitment and retention plan seamlessly and efficiently through our ExcelliPACE™ process, leading to faster enrollment and fewer dropouts.

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Patient-Centered Approach

Patients with rare diseases face untold obstacles in their daily lives and minimizing burden is a critical component in recruiting a committed group of patients into studies. Medpace places patients at the center of a trial to increase awareness and reach enrollment goals while helping yield successful outcomes in terms of study design, participation, adherence, satisfaction, and data collection.

Medpace Patient Concierge Services (PCS) provides global travel support to minimize patient burden, facilitate compliance with study visits and help keep the study on track. Additionally, Medpace offers a suite of decentralized clinical trial (DCT) capabilities that provide greater flexibility and support to the patients to help keep studies on track and on budget.

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Thoughtful Trial Design & Regulatory Strategies

Design of rare disease clinical trials can present challenges due to limited patient numbers and while randomized clinical trials remain the gold standard, designs that include a single-arm, non-randomized, open-label approach can be successfully conducted, along with natural history or registry data as a control. Extensive experience with these study designs allows Medpace to advise and support innovative approaches specifically for rare diseases from a medical, operational, and regulatory perspective.

Achieving Orphan Drug Designation (ODD) from the FDA or EMA is key to accelerating clinical development of rare disease therapies and provides various benefits including regulatory assistance, financial incentives, and market exclusivity. Early and frequent interactions with regulators are important to ensure agreement on proposed trial designs and overall development strategy, especially when adaptive trial designs may be considered and expedited pathways to reach approval can be utilized. Medpace supports clients through these interactions with an experienced regulatory affairs team that can provide extensive expertise in clinical and statistical aspects of rare disease studies to meet regulatory expectations.

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Rare Disease Clinical Trial Experts

Identify and address issues before the study is impacted through our team’s broad therapeutic experience and expertise in rare disease and pediatric studies. Medpace’s committed cross-functional team of rare disease experts is comprised of doctors, project managers, regulatory consultants, and late-phase specialists to orchestrate these complex studies and produce truly seamless drug development.

Designing protocols and executing studies in limited patient populations require specialized expertise. Our integrated board-certified physicians, including pediatricians, have backgrounds in treating patients with rare diseases/conditions and understand the complexity involved.

Key Rare Disease Indications

Medpace understands the unique challenges rare disease clinical trials face because we have conducted global trials covering Phase I-IV across a broad range of rare disease indications. Explore a sampling of our experience in these therapeutic areas:

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Patient and Caregiver-Centered Approaches in Rare Disease Clinical Trials