Rare Disease Clinical Research Services

Rare disease clinical trials bring a vast array of challenges and complexities to developing therapies and getting access to patient populations that are small and geographically diverse. Our team of experts is here to help you conquer these challenges and bring life-changing therapies to these patients.

Because many rare disease clinical trials patients are located across the US, Europe, Asia-Pacific, and Australia, ProPharma Group’s global presence allows us to help you reach the patients you need to complete your study. Our experts understand the regulatory requirements for conducting clinical trials in these areas and can leverage our experienced in-house decentralized clinical trials team to get your therapy to patients in an efficient manner.

Rare Disease Expertise by Indication

At ProPharma Group, our team of rare disease experts has experience supporting a variety of indications for both adult and pediatric populations.

  • Palmoplantar Pustulosis
  • Mucopolysaccharidosis
  • Sickle Cell Anemia
  • Graft Versus Host Disease (GVHD)
  • Pancreatic Cancer
  • Solid Tumors
  • Hepatocellular Carcinoma
  • Friedreich’s Ataxia
  • Multiple Myeloma
  • Long-Chain Fatty Acid Oxidation Disorder (LC-FAOD)
  • Glycogen Storage Disorder (GSDIa)
  • Non-Small Cell Lung Cancer (NSCLC)
  • Prader Willi Syndrome
  • Placenta Accreta
  • Tay Sachs, Sandhoff
  • Alport Syndrome
  • Coronary Artery Disease

Rare Disease Resources

Decentralized Visits in Rare Disease Study

Decentralized Visits in Rare Disease Studies

The COVID-19 pandemic caused major disruption to many clinical trials and the participants receiving treatment. The risk of exposure for pediatric rare disease patients and their families was particularly challenging and stressful. Reduced travel options compounded by the risk for exposure during travel or at the Site, affected the entire family.

Read more about how ProPharma Group worked with the sponsor to implement and execute decentralized visits so that patients could continue their study as planned.

Download the Case Study

Get Expert Guidance for Your Rare Disease Clinical Trials

If you’re planning to outsource your rare disease studies, you deserve to work with an experienced team that partners with you and is invested in the success of your study. You shouldn’t have to worry about whether your studies are being done the right way.

Contact us to connect with a member of our team and tell us how we can help.

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Tell Us About Your Rare Disease Trial

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