Kayda Norman

A new study by Premier Research shows that both American and European pharmaceutical companies are facing similar problems in regards to pediatric trial regulations. The number one concern for both? Patient recruitment.  

The study, which surveyed 55 companies, revealed that patient recruitment may be suffering because of the lack of children available who meet study guidelines. In fact, over 70% of US respondents indicated that finding enough patients for Pediatric Research Equity Act (PREA) studies is difficult. 

 asked Charli Sanders, MD, Premier Research Vice President, Regulatory Affairs and Pediatric Consulting, how a company can go about improving patient recruitment. 

"One thing that companies embarking on pediatric studies ought to do is to evaluate the customs, cultures, and healthcare access patterns of the pediatric populations they intend to enroll," Sanders said. "Alternatively, they should select a partner—such as a qualified CRO—which can do such an evaluation for them." 

Sanders also noted that companies should remember that matching patient profiles to the clinics and countries where the trials are conducted is essential to the success of a study. 

Another option for trials that have only a few available subjects or with a long enrollment period is "pooled population study." 

"The value of such studies is that they are able to rationalize scant data derived from seemingly unrelated sets of pediatric patients exposed to the same product. This methodology pools the data, which are analyzed together to construct a pediatric profile for a specific product," Sanders said. 

Although studies with larger subject numbers would be preferable, these computer-based results can also deliver informative data. 

In addition, the Premier Research survey showed that companies faced other challenges besides patient recruitment such as general confusion about PREA rules in the United States, as well as the Pediatric Regulations' Pediatric Investigation Plan (PIP) requirements in Europe. 

Seventeen percent of US respondents do not understand PREA, about 60% said they "somewhat understood" PREA, while an alarming 10% never even heard of PREA, which has been around since 2003. 

And results were little better across the pond. According to those surveyed from Europe, two in 10 said they do not understand PIP, only 55% said they "somewhat understand" PIP, and 6% claim they never heard of PIP. 

To counteract this problem, Sanders recommends that companies begin looking at PREA and PIP guidelines earlier rather than later. 

"Because many companies don't fully understand the process, delays occur," Sanders said. "Obstacles frequently have less to do with the regulations themselves than how companies are set up to handle the global demands that these pediatric regulations make, both financially and in terms of time management." 

For those who are still baffled by the pediatric guidelines, Premier Research has conducted a series of webinars about pediatric clinical research which are available on their website at 

. The company has also issued the first of two white papers regarding pediatric trials for further background. 

But just how important are these regulations to companies anyway? In the United States, 36% said complying with PREA is a moderately high priority, 12% relatively low priority, and 52% noted it as a high priority. Results were similar in Europe, where 26% placed pediatric regulations as a moderately high priority, 4% admitted it was a relatively low priority, and 54% said they made it a high priority. 

Other key takeaways to note from the survey are that about one-third of users did not realize they could revise their PREA obligation by working with FDA. 

"For that expressed reason, we recommend an initial consultation," Sanders said. "When appropriate, it also may be helpful to invest in pediatric pharmacometric work up front that could potentially eliminate the need for more expensive and elaborate clinical trials down the line." 

About half of respondents in the United States and Europe said that PREA and PIP guidelines have forced them to increase R&D budgets. And almost all surveyed in both the United States (92%) and Europe (90%) agreed that having an independent analysis of the company's' PREA or PIP obligation would save time and money. One major difference between Europe and the United States? Europeans (68%) believe securing PIP waivers or deferrals should be easier, compared to 39% of Americans surveyed. 

To see more results from the survey, go to 

Benefit from industry updates and case studies related to this article at CBI’s Workshop on Pediatric Investigation Plans, September 20-21, 2012, Boston, MA 

Articles

A new study by Premier Research shows that both American and European pharmaceutical companies are facing similar problems in regards to pediatric trial regulations.

Survey Reveals Patient Recruitment is the Biggest Challenge in Pediatric Trials

will be leading the company's advertising segment as the new President of inVentiv Health Communications. 

has also joined the company as Senior Vice President of European Operations and Development for inVentiv Health Commercial, which provides sales, marketing, communications, and patient outcome services to a wide variety of pharmaceutical, biotechnology, medical device, and life sciences companies. 

as Senior Vice President of Sales and Marketing and has promoted 

, PhD, to Senior Vice President, Global Project Management, to support growth in the company and development of international opportunities. Both executives join CEO Wendel Barr, COO Stewart Bieler, and CFO Patrik Jeanmonod on the executive council for the company. 

 (Research Triangle Park, NC) has appointd a new Chief Executive Officer, 

. Dennis Gillings, who founded the company, continues as Executive Chairman of its Board of Directors. 

, MD,  as Vice President of Medical and Scientific Affairs, Medical Device and Diagnostics. 

has also joined the company as Vice President and General Manager for the Asia-Pacific region in the Singapore      office.

 as Executive Vice President, Corporate Operations. Within this post, Barker will be responsible for maintaining high-quality deliverables from POI's range of global trial services, including government contracts, data management, biostatistics, and other functional services. 

, PhD, to the position of Executive Director, Clinical Development Services, Pediatrics. In this position, Koskelo will play a principal role in the strategic planning and execution of pediatric studies at Premier Research.  

to its global resourcing solutions team. Stephenson brings her expertise in the area of functional service provider and staffing models to serve clients' needs. 

 (Research Triangle Park, NC), a division of Novella Clinical, has hired 

• Bringing with her 18 years of clinical and regulatory drug development expertise in biopharmaceutical and clinical research industries, 

CTI Clinical Trial and Consulting Services

, PhD, as Vice President, Clinical Trial Operations. CTI has made several other promotions and appointments including the promotion of 

 to Assistant Director, Global Safety and Pharmacovigilance and 

 promotion to Accounting Clerk II. Three more additions, 

 as Senior Clinical Research Associate at CTI, Spain, 

as Clinical Project Manager Europe at CTI Europe, and 

, JD, as Corporate Counsel, have recently joined the team. 

 (Newtown, PA) announced that Amarin Corporation has chosen the eClinical suite from BioClinica to capture and manage clinical data for their Phase IIIb cardiology clinical trial, including: electronic data capture and data management, interactive response technology, and clinical trial management solutions.  

(Concord, OH) announced the formation of "The Biosimilars Alliance." The Biosimilars Alliance is focused on preclinical and early clinical assessment of biologics manufactured by a new supplier. 

(Dublin, Ireland) announced that it has been selected by Roche as its technology partner for storing and managing medical images collected during the company's clinical research programs. 

(Research Triangle Park, NC) has accepted 

annual Training Top 125 award, which highlights companies' excellence in employer-sponsored training and development programs. 

 (Fort Lauderdale, FL) announced that all seven sites making up the original clinicalRSVP user network, including Clinical Pharmacology of Miami (one site), Comprehensive Clinical Development (three sites), Elite Research Institute (one site), and Seaview Research Inc. (two sites), have agreed to extend their commitment to the clinicalRSVP network through 2012 and beyond. 

(Annapolis, MD) has enhanced its mission by recently expanding its on-site audit program designed to assess how imaging personnel are implementing their quality practices and maintaining their imaging equipment.  

 (Research Triangle Park, NC) marks the 30th Anniversary of the company's incorporation, transforming from a small statistical consulting firm housed in a university campus trailer to one of the world's largest biopharmaceutical services company with 25,000 employees and offices in 60-plus countries. 

 (Lenexa, KS) has announced the full integration of its long-standing laboratory partner Phoenix Pharma Central Services in Singapore. 

The Association for the Accreditation of Human Research Protection Programs 

(Washington, DC) has accredited two more organizations: Boise VA Medical Center in Boise, ID, and VA Puget Sound Health Care System in Seattle, WA. 

• In an effort to educate the public, media and policymakers about clinical trials and the growing role of clinical research organizations in the drug development process, the 

Association of Clinical Research Organizations

 (Washington, DC) has launched a YouTube channel @ACROHealthChannel.  

The International Academy of Clinical Research 

(Maidenhead, UK) has launched a campaign calling for the introduction of accredited clinical research training with a recognized industry standard and demonstrable, measurable levels of competence. 

 (Dublin, Ireland) announced the acquisition of PriceSpective. The acquisition strengthens ICON's exertise in value based pricing and market access. 

(Burlington, MA) announced that it has signed a definitive agreement to acquire Kforce Clinical Research from Kforce Inc. for $50 million in cash.  

(London, UK) announced expansion of its capabilities in India with the opening of a new office in Bangalore, India.    

(Paris, France) continues their expansion of services in India, by augmenting their existing Mumbai facility with four additional branches to support the growing demand in clinical trial logistics within India. The expansion in New Delhi, Hydrabad, Pune, and Bangalore will support the growing need for temperature controlled logistics of sensitive drugs and cold chain specimens.  

(Plymouth Meeting, PA) new web-based tool, TrialMax Web™, offers a solution with patient usability benefits including enhanced patient focus to promote better engagement, data capture, and compliance to generate higher-quality data. 

(Research Triangle Park, NC) has released CGIRB Connexus®  2.0., an award-winning eDocument management system, utilized by sponsors, CROs, and sites to manage the IRB review process.  

 (Freiburg, Germany)launched its "Google for Pharma & Biotech," a Web2.0 tool for finding the right partners in the pharmaceutical industry worldwide. 

Industry news focusing on the people and organizations who work in the clinical trials profession.

Business and News Update June 2012

Quintiles held a webinar a few weeks ago titled   “The New Health Report 2012: Rethinking the Risk Equation in Biopharmaceutical Medicine," reviewing its recently released 2012 New Health Report. Overall, the report showed companies need to better analyze and communicate risk with stakeholders. In regards to clinical trials, the survey also showed that payers would like to be more involved across the drug development cycle. 

Now, for instance, only about one quarter (or less) of UK payers are involved in preclinical and Phase I testing. However, as testing gets farther along, payer involvement increases.

 Fifty two percent of UK payers are involved in Phase III testing, increasing to 59% for post-marketing surveillance. Perhaps payers will become more involved in the future across the board, as 43% have indicated they would like to be involved in preclinical testing (currently only 18% are involved).

Up to 75% of executives and payers, as well as 61% of investors believe that pre-competitive alliances could help increase new drug innovation. Nonetheless, currently only 37% of executives indicated their company participates in such an alliance. And although there is a belief that pre-competitive alliances could help increase drug innovation, the survey also concludes that biopharma executives believe alliances would increase the time it takes to bring a new drug to market.

For a more detailed look at the report, click 

Quintiles 2012 New Health Report

regarding pediatric trials and reglations earlier this month. 

 was able to speak with  Charli Sanders, MD, Premier Research Vice President, Regulatory Affairs and Pediatric Consulting about what she hopes the future of pediatric trials brings and how these trials have already started to change.

What has changed in pediatric trials over the last few years? 

For one thing, pediatric clinical trials have gotten more costly. It's not that the regulations have changed so much as enforcement of pediatric regulation compliance is becoming more rigorous. Additionally, under PREA (Pediatric Research Equity Act of 2003), the FDA has leverage to require more rigorous and comprehensive pediatric programs than previously have been requested. Consequently, procrastination can prove costly.

How do you think the results of this study will differ in the next five years?

In terms of how this survey's results will be different in five years, hopefully the biggest change will be that companies will understand and be more comfortable with the process, and will have developed a systematic method for complying.

Look for a more detailed overview about the Premier Research survey in our June issue.

Pediatric Trials: Past and Future

New research released has indicated that the majority of clinical trials are small and missing significant data. This has come to light after analyzing over 95,000 trials registered on 

 despite the 2004 mandate that all published clinical studies must be registered on the online database.
 
 Some studies (7%) even failed to mention the trial’s purpose, and 4% didn’t state how man volunteers were taking part in the study. Those that did give volunteer numbers showed that 62% of trials from 2007 to 2010 had 100 or fewer participants. Nevertheless, the number of registered studies has increased (due to the new law in 2004) with 40,970 studies registered from October 2007 to September 2010 vs. 28,881 from 2004-2007. 
 
 Of the trials analyzed, three specific areas were focused on including oncology, cardiology, and mental illness (totaling 79,413 studies). Cancer trials in particular were shown to be the least stringent in following medical guidelines. For example, 65% of oncology trials failed to randomize patients.

"We think expert groups need to start scrutinizing the database more and really examine the quality of the trials one by one," report author Robert Califf, MD, Vice Chancellor for Clinical and Translational Research at Duke University, told 

. "For the first time, we have a chance to look at the entire universe [of clinical research] and we can see that we can do better."

Do you agree with the results of this study? How can companies improve clinical research?

Research Shows Most Clinical Trials are Too Small and Missing Data

• Profil Institute for Clinical Research

, MD, a Pharmaceutical Research Executive, and Jerrold Olefsky, MD, Professor of Medicine at the University of California, San Diego, to its Board of Directors. 

• With more than 20 years of experience in the pharma industry, 

 (Södertälje, Sweden) quality assurance and regulatory team where he will oversee GMP projects. 

, MD, have joined the company, bringing their expertise in the areas of gastroenterology, oncology, and pediatrics to serve clients' needs.

, MD, to Chief Medical Officer of European Operations.

(Glen Burnie, MD) has made several new additions including 

 (Houston, TX) has named three new members to its advisory board including 

, MD, Clinical Associate Professor, Stanford University, Director of Cardiology, HealthHelp, Medical Director for the CVCTA Education Center, and Director of Cardiovascular Imaging at Nevada Imaging Centers; 

 MD, former Chair of Radiology at Thomas Jefferson University Hospital, faculty appointments and leadership roles at the New York Hospital-Cornell Medical Center, Downstate Medical Center, Harvard Medical School, and Brigham and Women's Hospital; and 

, MD, Diagnostic Radiologist at Reno Diagnostic Center; former Chairman of the ACR Coding & Nomenclature Committee and its Regulatory Compliance Task Force, former President of the CRS, and current Executive Committee Member. 

• CTI Clinical Trial and Consulting Services

 (Cincinnati, OH) has made several new hires and promotions including 

both as Research Associates. Recently promoted are 

to Senior Project Manager and Medical Writer,

 to the position of Vice President, Marketing, where he will have global responsibility for leading and developing global marketing. 

• Following a career with increasing levels of responsibility in finance for several successful, global public companies, 

(Toronto, CA) Executive Vice President, Chief Financial Officer.  

(Malvern, PA), a provider of clinical trial management consulting services and software solutions, has named 

•Bringing with him over 28 years of leadership experience in oncology R&D, 

 (Montreal, Canada) announced the successful implementation of an enterprise resource planning system for managing all pre-sales, operations, and finance related processes for ethica Clinical Research Inc.

(Pittsburgh, PA) announced that DiaryPRO®, its field-based electronic clinical outcome assessment solution, captured the primary efficacy data in the pivotal studies that led to INSYS Therapeutics' successful new drug application for SUBSYS® fentanyl sublingual spray. 

To improve the assessment of how cancer patients are responding to investigational therapies in clinical trials, 

 (Sophia Antipolis, France) announced a strategic agreement with 

 (Durham, NC) to offer advanced integrated image services to biopharmaceutical customers worldwide.

, PhD, has been recognized by the Carnegie Science Center as an honorable mention in the Entrepreneur Category of its 2012 Carnegie Science Awards.

(Mumbai, India) has begun clinical research of medical devices. It has commenced working on a CE (European Conformity) mark registration trial for a coronary stent device. 

 has doubled its analytical capacity at its 240,000 sq. ft. North American headquarters in Souderton, PA.

 (Buckinhamshire, UK), a clinical trial support organization that offers home trial, site nurse, recruitment, and retention support, has launched a new web-based training program for nursing teams.

(Boston, MA) has introduced a new service offering that supports expert readers in a timely manner to complete cardiovascular endpoint committee reviews quickly and cost-effectively. The new service offering includes upload of narratives and clinical data by sponsor/CRO via WorldPRO; the ability for sites to upload imaging and other supportive information for review and analysis, if applicable; review of consolidated subject data by external MD to confirm need for assessment and complete QC; and organization of data into an easy-to-access format for FDA audit.

 (Philadelphia, PA) has launched a new corporate website to further elevate its market and thought leadership position available at 

Ellen Morgan, former Co-Founder, President, and CEO of Synteract, has launched a non-profit contract research organization, 

Public Health Alliance for Clinical Trials (

PHACT) (Carlsbad, CA) with two co-founders Christian Sacher and Yaw Asare-Aboagye. PHACT is conducting clinical trials for biotech and pharmaceutical products to treat diseases disproportionately affecting the poorest people in developing countries. 

 (Fort Lauderdale, FL), the participant registry that prevents research volunteers from enrolling in overlapping and concurrent research studies, announced that all seven sites making up the original clinicalRSVP user network, have agreed to extend their commitment to the clinicalRSVP network through 2012 and beyond.

 (Cary, NC), a global full-service contract research organization with a focus in oncology, has completed the acquisition of Nexus Oncology. The transaction also provides Ockham with an institutional investment partner. 

(Verona, Italy), an international full service contract research organization, has completed a private-public deal with Centro Ricerche Cliniche di Verona (CRC), a modern early phase clinical research unit, founded in 2005. The CRC is located in Verona and is now under state and CROMSOURCE ownership.

(Greenwich, CT) announced the completion of the sale of the Clinical Trials Supply business to Catalent Pharma Solutions.

(Philadelphia, PA), a specialty service provider focused on the business and financial management activities for clinical trials, has expanded its global presence by opening a new office in Edinburgh, Scotland. 

 (Sacramento, CA) has completed its new corporate headquarters suite in Sacramento, where the company plans to move staff and operations, which adjoins the offices of Radiological Associates of Sacramento. The move is expected to enable a high degree of strategic collaboration between the two firms.