Clinical News Roundup: FDA Awards Funding To Pediatric Clinical Trials

By Ed Miseta, Chief Editor, Clinical Leader

Despite many efforts over the past two decades to increase the number of FDA-approved therapies for children, 60 percent of drugs that are used in children are not approved for pediatric use. The percentage of FDA-approved options is even smaller for neonates, with 90 percent of drugs used in this population unapproved for neonatal use.

The FDA is now taking steps to address this problem. According to Susan McCune, FDA’s director of the Office of Pediatric Therapeutics, notes these drugs have not been approved for use in children because they have not been successfully tested in pediatric clinical trials. In fact, a recent study found a failure rate of up to 42 percent for trials that were done under the 2002 Best Pharmaceuticals for Children Act (BPCA). The act grants an additional 6 months of marketing exclusivity for companies that study the drug in children.

It is not clear whether these failures mean the drugs do not work in children or whether investigators did not select the right drug, identify the right patient population, choose the right dose, use the right trial design, or identify the right endpoints for their trials. Many sponsors also said they were unable to complete their studies because they couldn’t recruit subjects for the trials.

FDA believes it is critical to support the development and maintenance of a scientific and organizational infrastructure that can plan, start up, conduct, and close out pediatric clinical investigations. For that reason, the agency will issue awards to facilitate pediatric clinical trials and pediatric trial-related research to the Institute for Advanced Clinical Trials for Children (IACT for Children) and Duke University.  Each awardee will receive $1 million for this year under the Global Pediatric Clinical Trials Network Cooperative Agreement.

Clinical Trials Offer Hope Of Treating Heroin Addiction

Long-term methadone and buprenorphine maintenance are mainstays of heroin addiction treatment. These medications bind to the same opioid receptors in the brain as does heroin, reducing cravings for and use of the deadly drug. But a new study shows that an alternative to medications that substitute for heroin in the brain can be highly effective by blocking the drug's effect on opioid receptors.

The research team, led by Lars Tanum of the Norwegian Center for Addiction Research, randomly assigned 159 opioid-addicted individuals to drug counseling, combined with either daily buprenorphine maintenance or monthly injections of extended-release naltrexone. Because naltrexone stops heroin and other opioids from binding at the opioid receptor, it makes drug use less rewarding and therefore easier to quit.

Three months after the start of treatment, a majority of patients receiving either medication had used no heroin or other illicit opioids in the past month. However, the rate of abstinence was statistically higher for patients on extended-release naltrexone.

Drug-addicted individuals are deeply stigmatized and often their perceptions of the treatment they receive are not assessed or valued under the faulty logic of “who cares what a drug addict thinks?” But Tanum and colleagues wisely made such an assessment and found that extended-release naltrexone patients were more satisfied with their care and were more likely to recommend it to others than were patients on buprenorphine maintenance.

Marken Launches New App For Clinical Patients

Marken has introduced a new online tool which will allow patients to track their home deliveries of clinical trial materials and the pickup of their biological specimens via their mobile device or personal computer.  

With a simple click, Marken Viseo allows patients to view real-time updates on the driver's name, arrival time window, shipment number, and address at which the driver will stop. Patients can follow the driver's progress right up to their front door, with a clear view of exactly where the driver is and the projected arrival time, even taking traffic conditions into account. A photo of the driver shows exactly who will ring the doorbell with the delivery or pickup. The on-screen rating option after delivery provides direct and immediate feedback on the service and experience of the patient.

Marken believes real-time driver traceability can translate into improved patient expectations and confidence with Direct-to-Patient or Direct-from-Patient services. More deliveries can be performed on the first attempt with fewer reschedules or delays. Marken introduced the product and its features at the CPhI Worldwide conference in Frankfurt last week.

Can Seeker Portal Revolutionize Trial Enrollment?

Seeker Health, a digital health company focusing on patient enrollment, has announced the launch of the Seeker Portal, a patient management software solution that helps evaluate, assign, manage, and track participants to enrollment. The company hopes to accelerate drug development by deploying compliant social media campaigns to improve patient education and using the Seeker Portal to accelerate the enrollment of participants into clinical studies for serious or rare diseases.

Sandra Shpilberg, CEO and founder of Seeker Health, notes the time to complete a clinical trial averages two years, and much of that time is spent on patient enrollment. “Finding novel ways to accelerate this effort is paramount," she says. "We are using social media and technology to solve this thorny problem and accelerate drug development so medicines get developed faster and reach those who need them earlier."

Patient enrollment is often cited by biopharmaceutical companies as one of the most significant barriers to conducting clinical trials and failure to enroll sufficient numbers of patients can result in costly delays or even cancellation of the entire trial. These barriers intensify in oncology and in rare diseases, where there is increased competition for participants.

Key features of the Seeker Portal include:

• Secure and immediate delivery of qualified patient referrals to active clinical trial sites.
• Evaluation of pre-screen patient submissions against set inclusion/exclusion criteria.
• Immediate assignment of qualified patients to an active clinical site based on geographic analysis.
• Immediate communication with qualified participants via email and text messaging.
• Multi-language messaging capability for global clinical trials.
• Real time tracking of referral lifecycle and progress toward enrollment.
• Scalability of recruitment and enrollment efforts.
• API integration with medical call centers.

Improving Minority Access To Clinical Trials

Today, fewer than 10 percent of patients enrolled in clinical trials for breakthrough medicines are racial or ethnic minorities, despite the fact that this population makes up nearly 40 percent of the U.S. population.

According to Dr. Janice Greene, president of the Snohomish County (WA) NAACP, the cost and time commitment of clinical participation, plus inadequate outreach efforts to diverse communities, puts up a barrier to participation. In turn, people of color are immediately at a medical disadvantage as they sometimes respond differently to treatments. This varied response means that a potential new cure applies only to those able to participate in its trials.

Greene believes it is crucial for hospitals, health care staff, and researchers to understand these barriers and work to address the inequality that exists in clinical trials. Last month was Breast Cancer Awareness Month, and we are especially reminded that deadly disparities still exist. Today, breast cancer mortality rates are higher in African American than Caucasian women — and women of color have not benefitted as greatly from improved detection and treatment methods.

“It is also important that we acknowledge and address one of the underlying reasons that people of color do not participate,” says Greene. “People of color have a long and difficult history with the medical community. An example is the notorious Tuskegee syphilis trials of the 1930s. As a result, there is skepticism with participation. Now, the safety and ethics of clinical trials are heavily regulated and federal rules are enforced to ensure compliance. Building those relationships — and investing in our own health — is the only way for us to benefit from ongoing research and development of potentially life-saving treatments.”