Rare and Orphan Diseases
We leverage our experience working closely with patients, advocacy groups, and key opinion leaders to successfully recruit, retain and empower patients.
In the realm of rare diseases, traditional large-scale study approaches can fall short, necessitating a blend of deep scientific expertise and innovative thinking. Our team excels in designing unique programs that maximise the impact of even the smallest patient cohorts.
Common areas in which we have expertise include:
- Huntington's Disease
- Usher Syndrome
- Ehlers-Danlos Syndrome (EDS)
- Sickle Cell
- Cystic Fibrosis
- Duchenne Muscular Dystrophy (DMD)
- Haemophilia
- Gaucher Disease
- Cutaneous T-Cell Lymphoma
- Glioblastoma Multiforme
How we work with rare and orphan disease drug developers:
- Pre-IND and IND FDA Regulatory work
- Development of Bioanalytical Assays and Identifying Biomarkers
- FDA/EMA Orphan Drug Designation applications
- Liaising with patient support groups all over the world
- Identifying countries with the highest prevalence of the rare diseases
- Liaising with orphan disease funding bodies
Disciplined,
Cost-Efficient Execution
while maintaining the Highest Quality
Local Trials,
Global Approvals
Access FDA Regulatory Expertise
Our Clinical Team has over
120
years
of Combined Clinical Trial Experience
How iNGENū CRO Addresses Key Pain Points Associated with Rare and Orphan Disease Clinical Trials
Genetic and Molecular Understanding
iNGENū leverages advanced genomics and molecular analysis techniques to uncover the genetic and molecular mechanisms underlying rare diseases. Our team collaborates with leading researchers, employs cutting-edge technologies, and participates in international research networks to accelerate discoveries in this field.
Drug Development and Therapeutics
We are committed to facilitating collaboration between academia, industry, and patient advocacy groups. Through our integrated approach, we streamline the drug development process, providing regulatory expertise and leveraging incentives for orphan drug designation to expedite the development of novel therapies for rare diseases.
Patient Registries and Natural History Studies
iNGENū supports the establishment and maintenance of patient registries and natural history studies. Our data management and analytics capabilities enhance the quality and accessibility of these resources, contributing to a deeper understanding of rare diseases and facilitating more effective research strategies.
Access to Diagnosis and Treatment
We promote initiatives to raise awareness among healthcare professionals and implement educational programs for early recognition of rare diseases.
Patient Empowerment and Advocacy
Through partnerships with patient advocacy groups, iNGENū empowers patients and their families by providing resources, support, and guidance to navigate complex healthcare systems, ensuring their voices are integral to the research process.
Understanding the Complexities of Clinical Research in Rare and Orphan Diseases
- Rare and orphan disease research presents distinctive challenges. Patient recruitment is a critical aspect, and iNGENū leverages our experience working closely with patients, families, advocacy groups, and key opinion leaders to successfully recruit and retain vulnerable patient populations.
- We understand the importance of identifying the right regulatory strategy from the outset, offering support in developing optimal strategies, applying for orphan drug designations, and facilitating engagements with the FDA and EMA.
- Developing innovative rare disease therapies, particularly those involving cutting-edge products like cellular and gene therapies or uncommon administrative routes is often complex. Whether you are an emerging biotech or a global pharmaceutical company, iNGENū is committed to guiding you through the complexities of rare disease research, ensuring the success of your groundbreaking initiatives.
Rare and Orphan Disease Clinical Trial Resources
Phelan-McDermid Syndrome
Navigating Diagnostic Criteria, Trial Endpoints, and Drug Development Challenges.
Glioblastoma
Navigating the Complexities of Glioblastoma Clinical Trials: From Diagnosis to Enhanced Protocols.
The iNGENū CRO Difference
Local Trials, Global Approvals
We uniquely combine Australian expertise with direct FDA submission capabilities, offering a streamlined path for sponsors to achieve global approval. Our seamless integration allows trial data from Australia to be submitted directly to the FDA via our E-Gateway, eliminating the need for third-party intermediaries.
Disciplined, Cost-Efficient Execution
iNGENū CRO takes a highly strategic and disciplined approach to clinical research, ensuring that every dollar is directed toward generating meaningful outcomes. Our trials are designed with efficiency in mind, eliminating unnecessary costs while maintaining the highest scientific and regulatory standards.
Australian-Headquartered, Asia-Pacific Reach
We provide sponsors with comprehensive trial capabilities across the Asia-Pacific region, offering a seamless combination of cost-efficiency and high-quality clinical trials. We leverage the region's competitive pricing while maintaining rigorous Australian-based project management and clinical oversight.
"We have been using iNGENū for a Phase 1, first-in-human trial of our investigational drug. We have used iNGENū for: Medical writing, project management, clinical data management and full end-to-end, clinical trial execution and monitoring.
We have been very happy with all aspects of the management of the clinical trial and have been particularly impressed by the out-of-the-box creative solutions to speed up and reduce overheads for our company during this clinical trial."
Giles Moss
Chief Executive Officer
Ready to discuss your Rare or Orphan Disease Clinical Trial?