Pharma R&D Today
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Developing Child-Friendly Drugs
Posted on July 12th, 2017 by Betsy Davis in Pharma R&D
In a recent article titled A Spoonful of Kids’ Medicine Makes the Profits Go Up, Kaiser Health News reporter Shefali Luthra wrote about how some pharma industry players may view the need for child-friendly versions of drugs as a “lucrative opportunity”. Luthra specifically cited the opportunities presented by laws like the Best Pharmaceuticals for Children Act(which allows for delaying the approval of competing generics if companies test their drugs in children) and the Pediatric Research Equity Act (which requires more pediatric-focused drugs to be clinically assessed in children). “These laws have spurred companies to do more in terms of testing and developing pediatric medicines,” Luthra explained. “The companies can market the drugs without facing competition for a longer period of time.”
Previously, it was often hard to make a business case for investing R&D in medicines for children, given that kids are healthier and don’t suffer from the wide array of maladies that their elders do. But these laws help create real incentives for pharma firms to develop medicines for a market that is on the rise. In addition to incentives provided by the aforementioned laws, BCC Research has attributed significant growth in the multi-billion dollar pediatric medicines market to the industry’s increasing familiarity with designing and implementing trials for children, as well as the focus on commercially viable targets of high unmet clinical needs like the diseases designated under the Orphan Drug Act. BCC has also suggested that the rise in conditions such as obesity, diabetes, and hypertension in children is contributing to the surge, along with behavior conditions like ADHD, depression, and anxiety.
While that shows that there is a real opportunity in this area for pharma, there is also competition from compounding pharmacies, which can often provide specialized formulations for kids at cheaper costs. Pharmaceutica North America, for example, says on its website that children’s prescriptions can be made more “palatable” by compounding pharmaceutical companies like itself. In order to make medicines suitable for children, they can add flavorings, remove allergens or put pediatric medications in more “kid-friendly formulations” ranging from liquids to tablets to gummies.
However, some compounding pharmacies have come under fire. In 2013, Andrew Pollack reported in the New York Times that a federal inspection program had uncovered unsafe practices at 30 compounding pharmacies, including improper airflow, rust and mold found in clean rooms, and even unidentified black particles discovered in supposedly sterile medicines. “In recent years, the number of compounders has multiplied, and some have become essentially mass manufacturers, distributing huge quantities of medicines all over the country,” wrote Pollack. Much of the problem seemed to be a lack of regulatory oversight (an issue that heavily regulated drug makers don’t share).
Pharma companies interested in pediatric medicines shouldn’t consider them as an afterthought. Discussing Pediatric Formulations in the American Pharmaceutical Review, Samuel Maldonado and Daniel Schaufelberger argued that formulations suitable for children should be explored early in the drug development process. “Initial clinical testing of a pediatric formulation may need to be done in adults to demonstrate acceptable bioavailability and palatability,” they wrote. “Once a formulation is chosen based on preliminary testing in adults, initial clinical trials in children may occur once the safety and efficacy of the drug product in adults has been demonstrated.”
In a feature in The Medicine Maker, Jenny Walsh highlighted the importance of remembering that the difference between developing drugs for kids versus adults isn’t just about scale. “During infancy and childhood there is rapid growth and changes in various organs, body composition, and metabolic pathways,” she explained. “There are also differences in gastric pH and gastrointestinal motility between adults and children. This means that babies, infants, and children may handle excipients and active pharmaceutical ingredients (APIs) differently, leading to potential toxicity and changes in the required dose of API.”
Mansoor A. Khan and William Rodriguez, in an article published in Infectious Diseases in Children, further emphasized how important it is to consider both dosage and taste. While bad taste may seem like a mild inconvenience to adults, it is a far bigger obstacle for kids, so it is important that a medicine is inoffensive enough that a child will be willing to take it. Calculating the correct dose is also a major challenge, and the authors suggested that “A lack of serious availability of novel dosage forms and platforms call for the emergence of more champions. These champions must adopt a broad-based approach to develop novel platform technologies of the 21st century. Through the cooperation of toxicologists, pharmaceutical scientists, pharmacokinetics and clinicians, the field can be advanced.”
The global pediatric market has been projected to hit $100 billion by 2019, according to BCC Research. Whether focusing in-house R&D efforts on advancements in pediatric medicine or working in collaborative relationships with nonprofits like American MedChem (which targets rare diseases), pharmaceutical companies should see this area as a promising opportunity for innovation and growth.
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All opinions shared in this post are the author’s own.
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