How to Manage Multiple Site Trials while Ensuring Consistency of Study Conduct

March 7, 2013 - PRLog -- Due to the increasing pressure from global regulatory authorities to successfully implement regulations and ethical standards into pediatric study processes, companies must ensure that their clinical programs are up to par with global standards. Therefore, attending this conference will enable attendees to close the gap between regulatory requirements, study development and execution of trials.

Sophie Biguenet, Medical Director, Global Clinical Research Virology at Bristol-Myers Squibb will be covering multiple site trials during the marcus evans 9th Annual Pediatric Clinical Trials Conference, April 24-26, 2013 in Philadelphia, PA.

marcus evans spoke to Sophie before the upcoming conference. In her presentation, Sophie will touch upon how industry professionals can ensure an adequate amount of participants are enrolled in their study and how to select sites with sound infrastructure. She will also cover how to effectively collate study data when dealing with multiple site trials.

We asked Sophie a series of questions about her role in both her day-to-day job, as well as at the conference.

Question 1: What is the top challenge faced when designing clinical trials for pediatrics?
Sophie Biguenet:

•   Integrating/respecting the pediatric specificities/particularities of the studied disease
•   Age subgroups might also have their own disease particularities
•   The trend is doing a copy/paste of the adult protocols, but this strategy is a failing approach
•   There is a crucial need for Pharmaceutical companies to share and build solid pediatric clinical plans and studies designs, which are respectful of the disease specificities in Pediatrics and even age sub-groups.

Question 2: When managing multiple site studies, how do you ensure an adequate amount of participants are enrolled in your study?

SB: Ensuring that an adequate amount of participants are enrolled in a pediatric clinical study is planned before starting the study and monitored during the study:

•   BEFORE starting the Study:
o   Agreement with Regulatory Agencies (EMEA + FDA) on study design:
   Randomized? Single arm? Adaptive trial?
o   Agreement with Regulatory Agencies (EMEA + FDA) on study size:
   This is not definitive! And it can change during the course of the pediatric plan
   Consider the term “evaluable” in the following sentence: “number of subjects evaluable for the primary endpoint” in the pediatric plan
o   Agreement with Regulatory Agencies (EMEA + FDA) on the Study population/Eligibility criteria:
   Do we need a minimum percentage of subjects from a specific region, race, genetic family, disease/clinical/biological characteristics?
o   Consider dropout rate:
   Can change with different populations/ages/baseline disease characteristics
   May also vary from one study to another: since highly dependent upon study design (example: if DC criteria are modified)
o   Sites selection:
   Countries selection: What is the epidemiology of your disease?
   Site selection: Consider Pediatric research networks; Sites experience in clinical research; Local facilities. Sites visits are key to establish strong connections between teams
   Sites training: Global and local training sessions; GCP

•   DURING the study:
o   Regulatory agencies (EMEA, FDA, local)  correspondence
   Check duties
   Inform of any question/issues: right away
o   Monitor Enrollment and Dropout rate
   Can differ from age/weight band groups to another
   An amendment impacting the study design can also impact the enrollment/dropout rate
o   Interim study analysis can lead to study design changes (amendment) impacting enrollment
   Example: add a new weight band or a new group + different dose

•   Communication is key:
   Internally: Operation teams, Medical, Regulatory, stats, Clinical pharmacology
   Externally: with local teams, sites, FDA, EMEA and local agencies
   Before during and after the study

Question 3: Due to the strong need of having a good working relationship between site coordinators and local health authorities, what have you found useful in managing these collaborations?

SB: The key messages would be:
•   In order to have a good working relationship between the site coordinators and the local authorities, it is the global team’s duty to assist and support the local team as well as possible.
o   Respond quickly to any question/issue
o   The collaboration is not only about local health authorities
o   Advice is not only one way (We need advice from the investigators as the also need from us)
o   The goal remains the child’s well being

•   Keep connected, establish a constant communication. Get to know each other.
o   Face to face meetings work always betters than teleconferences: At least, the investigator meetings can be an opportunity. Also consider sites visits
o   Not only when starting the studies but during the course of the study; Newsletters are not the only way to communicate.
o   The distance should not affect our communication

Question 4: How has the renewal of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) affected your programs at Bristol-Myers Squibb?

SB: The BMS Pediatric center of Excellence consists of team members representing key functions at BMS. Its goals are to:

•   Efficiently utilize BMS assets to comply with current legislation and produce high quality pediatric programs resulting in approved indications of BMS compounds for use by children of all ages delivered in age appropriate formulations. Obtain reward for pediatric program whenever possible.
•   Lead and support BMS asset teams in complying with current pediatric legislation
•   Consultation for development teams:
o   Guidance for BMS pediatric drug development
o   Review of pediatric programs
o   Optimize pediatric clinical trials
•   Education of the company in pediatric drug development
•   Maintain knowledge of the pediatric field and influence policy
•   Establish functional structure and process for accomplishing pediatric book of work

Sophie is a graduate of the Nancy University School of Medicine in France. She entered the industry in 2002 when she joined Roche in a medical affairs role, working in the transplantation and nephrology medical departments. From 2004 to 2006, she worked in the medical department of Biogen-Idec in Neuroscience & Immunology. She joined BMS in 2006 as an associate European Medical Lead in Virology-HIV, based in Paris, France. She move to the US in 2009, relocated to the BMS Global Clinical Research, Virology Department, where she is responsible for the BMS pediatric Clinical development programs for HIV compounds. Sophie is also a member of the BMS Pediatric center of excellence.