Non-Profit Organization Seeks to Enhance Care and Introduce New Market Opportunities Through Pediatric Drug Reformulation
According to a recent survey of pediatric hospital pharmacies, one-third of children admitted to pediatric hospitals receive drugs that have been reformulated by the hospital pharmacies using extemporaneous recipes.1 This is because many of the needed drugs are available only in pill form. So the hospital pharmacies reformulate them into liquids so that they can be administered to pediatric patients via oral syringe. These extemporaneously formulated drugs are not subjected to the rigorous analysis of purity, consistency, stability, and bioavailability that manufactured products must undergo in order to secure FDA approval. The Joint Commission has noted that this practice significantly increases the risk for adverse drug events among children – a risk that is approximately three times higher than among hospitalized adults.2
“If you’re an adult receiving medication in a hospital, you know that the medication you receive has been manufactured under extremely rigorous, FDA-approved, quality control practices and the effectiveness and safety has been studied in other adults,” said Don Lombardi, president, CEO and founder of the Institute for Pediatric Innovation, Inc.
“The key issue is to make sure that children have access to the same kinds of high quality, drug products that adults have, in a delivery form suitable for administration to children to enable safe, accurate, adherent use of the medicines.”
In 2002, the Best Pharmaceuticals for Children Act (BPCA) was signed into law to address this issue. It established a process for studying both on-patent and off-patent drugs for use in pediatric populations and offered pharmaceutical manufacturers six months of additional marketing and patent protection for drugs that were studied in accordance with the act. This was followed by the Pediatric Research Equity Act (PREA) of 2003, which authorized the U.S. Food and Drug Administration (FDA) to require pediatric studies of drugs when deemed necessary.
Don Lombardi, president, CEO and founder of the Institute for Pediatric Innovation, Inc
According to Lombardi, while this legislation spurred the industry into action, manufacturers did not take it far enough to benefit the majority of patients.
“Many manufacturers conducted studies for pediatric formulations but never developed and marketed the actual drugs,” said Lombardi.
“Now some of these drugs are off-patent so there’s no real incentive for the manufacturer to develop the proper formulations.”
Meeting the Need
That’s where Lombardi’s group, the Institute for Pediatric Innovation, comes in. It is a non-profit organization working to improve pediatric care by stimulating the development of medical products designed specifically for children. Under its Pediatric Pharmaceutical Reformulation Program, the organization is collaborating with leading hospitals, foundations, universities and investors to tailor existing drugs so that they are safe and effective for children. The organization also has a parallel program to address related issues in the medical device industry.
“We are trying to do a very simple thing – to identify drugs that are already in use but have never been made into products suitable for treating young children, and to take the necessary steps to develop and deliver pediatric formulations,” said Lombardi. “Our goal is to provide hospitals with the benefit of properly manufactured drugs in a delivery form appropriate for children to help them improve the state of care.”
Working closely with a consortium of six U.S. hospitals, led by Children’s Mercy Hospital and Clinics in Kansas City, MO, IPI has developed a needs analysis to determine which drugs are most commonly reformulated for pediatric patients and how often these extemporaneously prepared formulations are used. Based on this analysis, they’ve prioritized which drugs are most needed in pediatric forms.
IPI has contracted with the University of Kansas to carry out pre-GMP formulation research to identify the correct way to reformulate the indicated drugs in liquid or other delivery methods. To date, they’ve completed this process for one drug product and they expect to complete the process for a second product in the first quarter of 2010. From there, they are targeting one additional product opportunity every six months.
IPI is initially focused on products for which the pharmaceutical industry obtained patent life extensions and conducted pediatric clinical trials but never developed the actual drug products and have since lost patent protection. IPI is collaborating with Children’s Mercy Hospital and Clinics, the University of Kansas, and Beckloff Associates, Inc. to create a novel model for financing and managing commercialization of these products.
“Big pharma doesn’t want to put the resources into developing these products so we are essentially taking the problem off their hands,” said Lombardi. “We’re targeting drugs that are low-risk, inexpensive to develop and are able to provide an adequate return to investors. Through preliminary conversations there is a strong indication that the numbers work and there are investors willing to fund this effort.”
Uncovering Opportunities
Lombardi points out that this presents a valuable opportunity to the pharmaceutical industry, particularly smaller manufacturers.
“Everyone knows there’s a need. What they don’t realize is that there’s an opportunity, but you have to be able to think small,” said Lombardi. “There are a lot of specialty pharmaceutical companies that could make a nice business out of portfolios of four to eight products that each sell in the low double digit millions. As a non-profit, we minimize the expense and risk by kicking the tires to determine if the product is feasible and needed in the market and by providing the infrastructure and collaborations to provide the companies with finished products ready to manufacture and sell.”
In addition, IPI hopes to collaborate with the developers of novel drug delivery technologies to establish a portfolio of pediatric drug products that can be delivered in safe and effective ways beyond traditional oral syringe delivery.
For more information on IPI’s initiatives, please contact Elisabeth D’Angelo, [tel: 1.617.401.2377; email: elisabeth.deangelo@pediatricinnovation.org ]
About the author:
Kara Nadeau Della Vecchia, founder of KDV Communications, Inc., USA, is a healthcare communications consultant and freelance writer. Visit http://www.kdvcommunications.com or contact her directly at kara@kdvcommunications.com .