Pediatric trial education in focus

November 14, 2011 – 4:27 pm by Michael Christel

With new legislation and regulatory polices calling for more pediatric research as part of a drug-development program, the need for increased education around pediatric clinical trials is paramount, experts in this space stress. Unlike the case under earlier regulations, companies developing a drug intended for an adult market can no longer defer the planning of the pediatric component of the program until after initial approval.

Therefore, earlier planning and negotiation with regulators on trials for children has become essential. To that end, contract research company INC Research recently launched a complimentary education Webinar series called “The Classroom – Sharing the Lessons of Sucessful Pediatric Strategies.�

The first session, held last week, focused on the regulatory climate for pediatric research in the United States and Europe. A second session is planned for Dec. 13, which Kathryn Bohannon, INC Research’s principal strategist for pediatrics, tells me will explore approaches in pediatric early-phase pharmacology trials.

“It’s a natural evolution,� Ms. Bohannon says. “First, we wanted to set to stage and the framework for, ‘Why are we here? What’s driving the increase in pediatric research?’ A big reason for this push is we don’t have appropriate dosing information for our children. Historically, it’s been we give the drug to adults at X milligrams per kilogram, so let’s give it to kids at that same dose. That’s just not appropriate. A key piece in many pediatric development plans is to conduct those pharmacology studies and understand the [pharmacokinetics] of the drug across the whole pediatric population.�

INC is planning quarterly Webinars in 2012 as part of the series. Topics will include global trial design, medical challenges, dosing and safety issues, ethics, logistics, parental permission, and pediatric assent. The CRO may continue the series into 2013 based on audience interest.

I asked Ms. Bohannon to share her thoughts on pediatric research legislation and the growing responsibility of sponsors to plan and negotiate trials for children more effectively. Below is our exchange:

R&D Directions: What key factors have driven the need for drugmakers to better educate themselves on pediatric clinical trials?

Ms. Bohannon: Legislation within in the U.S. (since 1997) and EU (since 2007) require that pharmaceutical companies must address the pediatric population during the drug-development process; and when applicable, design and conduct pediatric trials in the appropriate pediatric age groups, consider development of more pediatric friendly formulations, and disclose study results.

Both the FDA and EMA encourage incorporation of pediatric research early in the drug-development process. The EU’s Paediatric Regulation requires that pediatric research be addressed earlier, specifically following conclusion of pharmacokinetic studies in adults, and involves often lengthy negotiation of a paediatric investigation plan with the EMA.

As the pediatric population must be addressed within the drug-development process, an increasing number of pharmaceutical companies must conduct pediatric research studies. It is therefore important that companies increase their pediatric competency and understand the unique considerations associated with conducting studies in children.

R&D Directions: Have the evolving pediatric regulations generally been embraced by pharmaceutical companies?

Ms. Bohannon: The requirements within the U.S. and European regulations do place an additional burden on companies when it comes to product-development planning and costs. Developing and negotiating the appropriate pediatric program that will satisfy the FDA and EMA can prove to be challenging and time-consuming.

In order to more effectively and efficiently satisfy the regulations, sponsors must consider the pediatric population early in drug-development planning and proactively engage in discussions with the FDA and EMA.

R&D Directions: What resources do pharma companies need to manage pediatric trials, and do most sponsors have them in-house?

Ms. Bohannon: Depending upon the size of the company and number of products, many customers do not have internal resources with pediatric expertise and rely upon consultants and partners for support.

Pediatric trials require management team members similar to those for adult trials, but with pediatric experience. Pediatric trials are often smaller in scope than adult trials, but the amount of effort in planning and execution may be the same as for larger adult trials.

Given the many factors that complicate pediatric clinical trials, sponsors should commit sufficient time and resources to formulating a strategy before beginning pediatric studies. Such a strategy must incorporate the unique pediatric considerations with respect to key factors inlcuding regulatory/legislative requirements, study design, drug formulation, consent and assent, and age groups.

R&D Directions: Beyond perhaps some of the obvious differences, what are the unique subtleties developers need to consider in pediatric trial design and execution?

Ms. Bohannon: Importantly, the design of pediatric trials needs to be customized for children, and each aspect must be thoughtfully considered and adjusted as necessary based upon the pediatric participants. Even a clinical trial’s basic safety and efficacy endpoints might need to be adjusted for pediatric patients.

The design needs to be appropriate for the stated objectives and take into account the specific physiology, pharmacology, and normal daily activities for each age group. In designing a pediatric protocol, the company must consider the balance of risks and benefits to the pediatric participants, and design a study that minimizes risk and offers the potential for information that will improve the care of children and of the individual study patient whenever possible.

The pediatric study’s schedule of events and required procedures must be customized in order to minimize risk, discomfort, inconvenience, and overall impact on the pediatric patients and families.

Given the special needs and considerations associated with the vulnerable patient population, the inclusion of patients and parents/guardians in the research study process, and the critical focus on ensuring safety and well�being of the pediatric participants, conducting a pediatric research study undeniably involves unique challenges.

R&D Directions: What does INC Research hope to accomplish through this Webinar series?

Ms. Bohannon: Pediatric data is more than just a requirement. We have a moral obligation to provide data for safe and appropriate pediatric dosing information and formulations for the products we develop.

As a CRO with a demonstrated commitment to pediatric research, we wanted to provide a forum for sharing information and indeed raising the awareness of the importance of pediatric research. Certainly we want to establish INC Research as the “go-to� resource for pediatric clinical development.

Tags: clinical trial regulations, INC Research, pediatric clinical trials, pediatric research