Rare Disease and Pediatric Center

Rare Disease

A dedicated cross-functional team of experts made up of strategic leadership and tailored support for rare clinical trials

We turn patient voices into better data.

We are focused on urgently accelerating the clinical development of new treatments in rare diseases.

Combining our industry knowledge, alongside personal experience within rare disease clinical trials, makes us a more valuable research partner for Sponsors looking for a truly tailored service experience. We can build a study around the individual patients’ needs, due to a nimble team that are never too far removed from the patient.

By providing a tailored approach to trial design and delivery, we can expedite the clinical development process and ultimately improve outcomes for patients and their families.

Schedule a Meeting

To speak with one of our rare disease experts about your trial or plans, please contact us.

We welcome your inquiries and will respond promptly.

As well as having a track record of supporting global research in cell and gene therapies and rare diseases in many therapeutic areas, we’re also experts in biostatistics, bioinformatics and adaptive/alternative study designs.

Orphan Strategy
Orphan Strategy

We can support you with the optimized orphan strategy, in-depth insight and experience that helps you to effectively navigate the orphan drug regulatory landscape.

Clinical Development Delivery
Clinical Development Delivery

Our rare disease experts provide the strategic vision and roadmap to facilitate full service clinical research delivery. There are clear advantages in small patient populations when using this type of offering coupled with our agile approach to both patient and customer needs.
Our service delivery approach enables us to provide:

  • Global access to local resources in excess of 100 countries
  • Operational hubs across Canada, US, Europe and India that are the backbone to driving project delivery
  • Expert insight from a highly experienced, focused leadership team, who can tailor the approach to project delivery on an individualised basis.
Patient Recruitment and Retention
Patient Recruitment and Retention

With fewer patients in rare disease clinical trials, the integrity and completeness of data from each patient assumes even greater importance. We employ many methods, including the following to retain patients and maintain compliance.

We foster close relationships with patient advocacy groups, Key Opinion Leaders and individual patients themselves to ascertain real life experiences, to ensure the patient and their experience is always contributing to retention and compliance.

Our established site networks enable us to provide more efficient start up timelines and the ability to pre-empt site requirements from the outset.

We support investigators and their clinical teams by providing resources to decrease the burden within their roles, to increase efficiencies with the trial itself.

Our deep understanding of the inclusion/exclusion criteria save time for patients and financial losses for sponsors by de-risking the number of screen fails.

We harness our established strategic partnerships with grass root patient organisations, as well as global patient organisations, to gain access to their registries, in order to meet recruitment timelines effectively.

Patient Perspective

Acquired in 2022, Casimir (now Emmes Endpoint Solutions) takes a new approach to clinical studies from the patient’s perspective, enabling us to build a better understanding of rare disease progression and treatment. 

Whether you are early clinical or post-marketing, we give you access to full-service protocol design, strategy and trial execution services. 

Together, we design and run rigorous decentralized and hybrid studies designed to address the needs of regulators, payers and patients. We focus on getting to the truth of a disorder and adapting or developing the tools necessary to truly understand disease progression and treatment benefits.

Visit Endpoint Solutions

Patient Perspective

A Sample of Emmes’ Experience in Rare Diseases

Ophthalmologic Rare diseases including 
inherited retinal diseases
ABCA4 Retinopathy, Stargardt’s disease, Usher syndrome, Xlinked Juvenile Retinoschisis, Macular Telangiectasia Type 2, Retinitis Pigmentosa, Leber Congenital Amaurosis (LCA), Albinism, Best’s disease, Uveal Coloboma, Spinocerebellar Ataxia Type 7 (SCA7), Von Hippel Lindau, Behcet’s syndrome
Blood Disorders including some blood cancersSickle Cell disease, Aplastic Anemia, Acute Myeloid Leukemia, Multiple Myeloma, Myelodysplastic syndromes, Bone Marrow Failure syndromes
Infectious DiseasesYellow Fever, Smallpox, Anthrax, Tularemia, Severe Acute Respiratory syndrome (SARS), Cytomegalovirus Infection, Neonatal Herpes (HSV)
Immune System/Immune DeficiencyHemophagocytic Lymphohistiocytosis (HLH), Primary Immunodeficiency (PID), acute Graft-versus-Host Disease (aGVHD), Chronic GVHD, Steroid Refractory aGVHD
OncologyAIDS-related lymphoma (e.g., Hodgkin and non-Hodgkin, Plasmablastic lymphoma (PBL), Burkitt’s lymphoma, Primary effusion lymphoma), Kaposi sarcoma, Anal cancer, Diffuse large B-cell lymphoma (DLBCL), T-Cell Lymphoma, other various rare leukemias/lymphomas
AutoimmuneUlcerative Colitis, Primary Sclerosing Cholangitis
NeurologyStatus epilepticus, Spasticity associated w/Cerebral Palsy
Lysosomal Storage DisordersFabry disease, Mucopolysaccharidosis II (MPS II or Hunter Syndrome), Niemann-Pick Type C, Cystinosis
Other Rare diseases/disordersBronchopulmonary Dysplasia (BPD), Limb-Girdle Muscular Dystrophies, Apnea of Prematurity