February 15, 2008 (Vol. 28, No. 4)

Paucity of Therapeutics for Children

Long underserved and undervalued, the pediatric population is beginning to emerge as an opportunity area for prescription pharmaceuticals. As a new, uncertain era opens for the pharmaceutical industry—one with fewer obvious blockbuster products and more emphasis on portfolio optimization—companies are beginning to see that “children are the future” is not just a cliché.

Clinicians in most specialties have complained for years about the lack of options and information available to them in the treatment of the pediatric population. Having to rely on off-label prescribing in many cases because of a lack of formally approved products, pediatricians and GPs have raised concerns about safety and efficacy of the adult therapeutics available.

In addition, the emergence of a host of cardiovascular, CNS, and metabolic conditions in the pediatric population—itself an extremely broad spectrum from neonates to young adults—has created a gulf of unmet need and highlighted the paucity of pediatric pharmacotherapeutics.

In response to the problem, which was apparent over a decade ago, the federal government launched the Pediatric Exclusivity Program to encourage developers to study their products in the pediatric population, offering the incentive of expanded market exclusivity as a reward for undertaking the additional expense of research. Several studies have suggested that this program has yielded both patient-care and economic benefits. The exact fate of the program is still not settled by law, but some form of incentive will undoubtedly be extended.

Until recently, this offer of an additional six months of protection was one of the only driving forces for pediatric approvals. This is changing, however. Indeed, the additional protection will remain an important reason for companies seeking as much incremental improvement to their margins as possible, but an additional factor of healthy revenue opportunity from the pediatric products themselves is also now a large factor.

The world market for prescription pediatric drugs is approximately $38 billion, and will grow to nearly $43 billion by 2011, according to Kalorama Information research. Although some traditionally well-served and mature segments of the pediatric market such as anti-infectives will display lackluster growth, other areas show much more potential.

Pediatric Rx Drug Market

In the cardiovascular segment, the prevalence and rate of diagnosis of hypertension in children and adolescents is increasing, due in part to the increasing prevalence of childhood obesity, as well as growing awareness of the disease. Obesity in children, among other factors, contributes to childhood hypertension, which research is now showing can lead to adult hypertension. Products that have documented safety and effectiveness in children include diuretics and beta blockers.

Preferential use of specific classes of medications for certain underlying or coexisting pathology has led to the prescribing of ACE inhibitors in children with diabetes or proteinuria and beta-adrenergic or calcium channel blockers for children with migraines. Many medications have growing research to support their use. There are currently more than 15 medications in later development for pediatric hypertension, cholesterolemia, and other cardiovascular issues.

The recognition of chronic conditions such as diabetes and hypertension, certain behavioral health conditions, and the epidemic of obesity in the pediatric population is pushing those segments ahead at a brisk rate. Revenues for hormone drugs including diabetic medication and growth factors for the pediatric population, for example, have displayed 10% annual growth over the past few years; other drugs such as dermatologic products have also shown 8% to 9% annual growth.

While the focus of most healthcare watchers has been on the world’s aging population, one must not forget that children number nearly 2.5 billion, or 40% of people on the planet. The demographics, large unmet need, regulatory incentives, and opportunity for product portfolio extension are making developers wake up and take a second look at this market.

Bruce Carlson is the publisher of Kalorama Information. Kalorama’s
The Worldwide Market for Prescription Pediatric Drugs, 3rd edition was
published in December 2007.
For more information visit

Previous articleAlexion Purchases Patents Related to Approved Drug from OMRF for $10M
Next articleResearchers Uncover Role of Mitochondrial DNA in Degenerative Diseases