Rare Diseases

The design and delivery of preclinical and clinical trials in the rare disease arena brings specific considerations and potential pitfalls for researchers, patients, pharmaceutical companies, and regulators. Because these trials can’t risk anything going wrong, sponsors need experienced partners they can rely on to get it right the first time, every time.

Perceptive combines operational excellence with extensive scientific, medical, and technological expertise to support rare disease researchers through every step of the clinical development lifecycle.

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De-Risking Rare Disease Trials

Perceptive can help you minimize risks and optimize rare disease development outcomes by:

  • Tackling the complexities of lengthy rare disease trials, including those involving stem cell and gene therapies
  • Minimizing central-site reader discordance in therapeutic areas where imaging is critical to accurate diagnosis
  • Determining optimal randomization strategies to minimize patient numbers while achieving your development goals
  • Delivering high-quality imaging data from multiple modalities to support subject enrollment and determine treatment efficacy
  • Optimizing delivery platform biodistribution and establishing robust PD biomarkers that can stratify and/or assess efficacy in early genetic disease and cancer studies.

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How Can We Help?

Reach out today and one of our solution experts
will be in touch to learn about your specific needs.

Therapeutic Area Expertise

Oncology

Hematology

Neuroscience

Radiopharmaceutical Therapy & Theranostics

Musculoskeletal

Additional Therapeutic Areas