Rare Disease Research: Conducting Clinical Trials Where the Patients Are

Life Sciences, Clinical Trials, Patient Recruitment & Retention,
  • Tuesday, December 15, 2015

young-boy_small-1In many rare and ultra-rare disease studies, sponsors do not have the luxury of choosing the countries where the study is conducted. They must use the sites where those patients are being treated, regardless of geography. Additional protocol criteria, such as treatment-naïve patients, or general prevalence of a disease by region may limit site selection even further, requiring the sponsor to open sites in countries that normally would not be considered for clinical trials.

Opening sites in countries where clinical trials are uncommon requires detailed knowledge of the host countries and the complex regulations they may impose, along with thorough site training in protocol and good clinical practices. In addition, monitoring and trial oversight must focus on getting every patient through the study with minimal protocol deviations and other issues to ensure data integrity.

It’s highly preferable to identify patients before opening a site and to ensure the patients will qualify for the study. This can avoid wasting effort and expense in opening a site where no patient will be enrolled. Also, extra measures may be required to ensure that patients are able to participate in the trial — for example, arranging for extended patient travel, supporting the patient’s family, and arranging home visits by a study nurse.

Other important site considerations:

  • Some countries have complicated import/export requirements, especially regarding shipment of samples.
  • There are various travel restrictions — for example, FDA inspectors cannot travel to sites in Iran. Therefore, if you use an Iranian site, the quality of data and documentation must be top notch or the FDA might not accept it.
  • Countries have widely different regulations and processes for site and patient reimbursement that need to comply with GCP and the sponsor’s local regulations.
  • Investigator meetings must be conducted in a country where the sponsor and all participating sites can travel freely.


Alison Sampson, Senior Project Director, Premier Research

Alison Sampson has over 20 years Clinical Research Experience including roles as CRA, Project Manager, Senior Project Manager and Project Director covering phase I – IV global studies. As an experienced Project Manager, she has led both clinical and cross-functional teams. She has worked in all phases of clinical research (I-IV) including global phase III studies as the Global Project Manager. She has experience of a wide variety of therapeutic areas with particular expertise in oncology, medical devices and rare diseases in pediatrics and neonates. Her work experience includes roles in Biotechs, Blue Chip Pharma and CROs.

Prior to entering clinical research, Dr. Sampson trained as a chemist and she is a Chartered Member of the Royal Society of Chemistry. She achieved Chartered Scientist Status in February 2009 through the Institute of Clinical Research.

Message Presenter

Hanna Wide, MSc, Project Manager, Premier Research

Hanna has over 8 years’ experience of project management of clinical research and a large portion of her professional career has been dedicated to rare indications. She has worked as Program and Project Manager for clinical trials in rare indication, including both adult and paediatric populations. She has worked on global studies with significant experience of working in the MENA (Middle East North Africa) region.

Hanna has been involved in rare disease feasibility, protocol review, study and program management, CRF review, management of the clinical team, vendor management and a range of other activities.

Hanna has a Master of Science in Medical Biology from Linkoping University, Sweden, and prior to joining Premier research, Hanna has worked in Clinical Research Project Management at QED Clinical Services (a niche CRO specialised in rare indications) and Quintiles.

Message Presenter

Who Should Attend?

VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:

  • Clinical Affairs
  • Clinical R&D
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Operations
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

for pharmaceutical, biotechnology and medical device companies.

Xtalks Partner

Premier Research

Premier Research is a leading CRO serving highly innovative biotech, Pharmaceutical and medical device companies. The company has a wealth of experience in rare disease and pediatric research having managed about 100 projects in each area in the last five years alone. Its services include clinical research and regulatory outsourcing in the areas of analgesia; neurology; infectious, cardiovascular, and respiratory disease; dermatology; oncology; and medical devices.

Premier Research and operates in 84 countries. It employs 1,000+ clinical professionals dedicated first and foremost to fulfilling each customer’s requirements in a timely, accurate, and cost-effective manner. This includes a strong international network of monitors and project management professionals combined with regulatory, data management, statistical, scientific, and medical experts, and staff at its well-established network of dedicated clinical sites.

Media Partner

You Must Login To Register for this Free Webinar

Already have an account? LOGIN HERE. If you don’t have an account you need to create a free account.

Create Account