Ilze Balode, MD
Senior Medical Director,
Rare Diseases
Rare Diseases
Research in rare disease & orphan drugs requires incisive thinking, experience, and a gentle touch
Rare diseases are a diverse and vast group of disorders that affect not only patients but their entire families. With over 8,000 diseases identified and 95% of these lacking treatments, they impact approximately 300 million people worldwide, of which 75% affect children.
As a rare disease CRO our Rare Disease & Orphan Drug Center of Expertise encompasses the entire range of clinical trials, from natural history studies to pivotal trials, addressing complex considerations including pediatric and vulnerable populations, novel biomarkers, clinical and surrogate endpoints, and innovative trial designs like basket and umbrella trials. We emphasize the integration of patient perspectives and specialize in a wide array of innovative medicines and advanced therapeutics for rare and ultra-rare diseases clinical trials.
Your Challenges in Rare Disease & Orphan Drug Development
Regulatory Hurdles
Navigating the complex regulatory landscape, including expedited pathways and orphan drug applications, and compliance with evolving guidelines, can be challenging. Additionally, clinical design aspects such as single arm pivotal studies and historical controls can present unique challenges.
Complex Study Designs
Designing clinical trials in rare diseases requires careful consideration of the patient burden, clinical endpoints, biomarkers, overcoming placebo challenges, continuous monitoring of data, and adaptive trial designs to optimize efficacy and safety assessments.
Patient Identification & Recruitment
Rare disease trial patient populations are limited and dispersed requiring innovative strategies for identifying, recruiting, and retaining patients.
Pediatric Patient Populations
Conducting pediatric clinical trials is a complex but necessary and urgent need, affecting not only young patients but entire families. These challenges are often magnified by the small patient populations, the severity and complexity of the diseases, and the ethical considerations involved.
We share your passion for finding treatments for intractable disease states and understand the intricacies of rare disease research require highly specialized knowledge and steadfast commitment to patient-centricity in trial designs.
The exceptional challenges faced when conducting rare disease and orphan drug clinical research require patient-centric clinical trials that address the needs of patients and their families, reduce the burden of participation, whilst ensuring trials are both robust and ethical.
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In rare diseases, every patient’s journey is different, but no one should go through it alone. By focusing on their experiences and acknowledging the high unmet needs, we can advance clinical trials that bring us closer to easing their struggle. Together, we’re not just searching for new treatments, we are creating hope, fostering resilience, and building a future where rare diseases are no longer overlooked.
MARIA-CRUZ MORILLO
GLOBAL THERAPEUTIC OPERATIONS LEAD, RARE DISEASES
Depth of Rare Disease & Orphan Drug Experience
As a rare disease CRO Allucent has more than 30 years of expertise successfully navigating the unique challenges of rare and orphan disease clinical trials including ethical considerations in protocol design, small sample sizes, pharmacokinetic sampling limitations, the need for standardized subjective endpoints, vulnerability of the patient population, and constantly evolving regulatory requirements.
120+
Studies
300+
Consulting
Projects
3,400+
Sites
40
Orphan Drug
Applications
All-time years of experience
With global breadth and depth across a wide range of rare diseases, Allucent partners with biotech companies to bring innovative therapies to light.
Cross-Therapeutic Expertise
Allucent’s rare and ultra-rare disease clinical trials expertise spans multiple therapeutic areas including neuroscience, autoimmune, pulmonary, genetic, metabolic, and oncology. It includes a variety of novel drug classes and precision medicine including gene therapies, stem cell therapies, biologics, GMOs, mRNA and antisense therapies.
RARE & ULTRA-RARE DISEASE EXPERIENCE
- Endocrine (e.g. acromegaly, Cushing’s disease)
- Epilepsies (e.g. pediatric, adult; progressive, genetic,
refractory) - Hemolytic anemias (e.g. CAD, WAD, PNH, SCD)
- Blood clotting disorders (e.g. hemophilia, ITP, TTP)
- Spasticity and sialorrhea (e.g. CP, MS, stroke, trauma)
- Neuromuscular disorders (e.g. ALS, muscular dystrophy, CP, ataxia, Huntington’s disease)
- Lysosomal storage disorders (e.g. various subtypes)
- Gastrointestinal disorders (e.g. short bowel)
- Pulmonary disorders (e.g. CF, PAH, IPF)
- Cardiac disorders (e.g. ATTR-CM amyloidosis)
- Skin disorders (e.g. RDEB, DDEB, EBS)
- Lupus nephritis
Meet Some of Our Rare Disease & Orphan Drug Experts
- Pediatrician and neonatologist with 20+ years of experience in clinical research and all aspects of clinical trials, including gene therapies
- Experience in many rare pediatric indications and treatment modalities
Milan Marinkov, MD
Executive Medical Director,
Therapeutic Area Medical Lead,
Rare Diseases
- Leads Allucent’s Rare Disease Medical Affairs Group; providing scientific and medical oversight in rare indications
- 15+ years of medical expertise encompassing all aspects of clinical trials from set up to study close-out
Joyce Moore, PhD
Global Head,
Patient Engagement
- Over 20 years of experience in the clinical trial sphere
- Deep expertise in patient engagement including conducting DCTs and collaborating with patient advocacy groups
Maria-Cruz Morillo
Global Operations Head,
Rare Diseases
- Head of Allucent’s Rare Disease Center of Expertise integrating cross functional expertise to provide strategic solutions for rare disease trials
- 25+ years of experience in Project Management and Strategy and IMP Supply in both biotech and CRO
