Patient centric dosage form opportunities for pediatric and orphan drug products

Creating patient centric dosage forms to enter specialty markets such as orphan drug designation programs and to treat pediatric patients will help to address an emerging industry need for enhancing adherence in patients while providing manufacturers with a successful financial outcome. 

Pediatric patients often present a serious challenge for adherence, given the need for easy administration, pleasant taste and mouthfeel to increase compliance. The pediatric population is also hard to treat because drug development tends to be slower, riskier, and more intensive, resulting in fewer drugs being developed for this group. However, regulators are embracing incentives to push drug companies to seriously address commercially viable solutions for small patient populations like pediatrics. If you develop doses for pediatrics, and it is done correctly, then the regulator will offer incentives to help accelerate the drug development process and extend the patent exclusivity on your drug product. In Europe and the US, a pediatric investigation plan, or PIP, is required to benefit from these incentives.

PIPs are a special addendum to the NDA or ANDA which provide a detailed plan to conduct clinical trials of the drug in children. The regulator wants to ensure you choose a path that includes pediatric development. If the plan does not include children, it will be slowed down. The regulator will question why pediatric needs have not been addressed. There is a significant population under the age of 18 and they need medication in the proper dose and form just as adults do.

Traditionally, formulation teams have not developed easy to administer dosage forms with pediatrics in mind, so many drug companies are not prepared for this. Children will be reluctant to take a large tablet or something that tastes terrible. Administering medication through inhalation or through a needle can also be challenging with a child. These factors all contribute to the importance of formulating a product with the patient in mind in order to successfully reach the goals set forth in the PIP.

Pediatric medicine is also helping the industry shift focus to treating diseases in small populations.  Developing drug products for populations affected by rare diseases can be hard to justify for some manufacturers. As such, drug developers will request an Orphan Drug Designation for a rare disease they are hoping to treat. Orphan Drug diseases overlap strongly with patients under the age of 18 because many diseases affecting children are rare in the general population. An Orphan Drug is a pharmaceutical product developed to treat medical conditions which, because they are so rare, would not be profitable. In addition to current government subsidies, regulators also provide extra incentives for drug companies to invest such as accelerated approvals and priority review, for example.

Pediatric and Orphan Drug opportunities represent approximately half of all new drug applications worldwide because of the high-value and high-growth potential of this market. Additionally, about 50% of Orphan Drug Designations contain a pediatric investigation plan. Research shows significant market value increase for Orphan Drug products historically and the expected growth over the next few years. 

Patient centric drug products offer effective ways to treat these unique populations. Patient friendly dosage forms such as orally disintegrating tablets (ODTs), chewable tablets and orally dispersible powders (sachets and stick packs) have been successfully utilized across the Pharma, OTC and nutritional markets for many years. These patient centric dosage forms would also be effective to support growth in specialty markets and smaller populations. New and innovative excipients and taste masking technologies will be needed as these markets grow, become more competitive, and active ingredients become more challenging to develop into a final drug product.

SPI Pharma’s flash ODT platform UltraBurst™ can provide super-fast disintegration of less than ten seconds. Products formulated with the platform can be administered easily and can potentially enhance the bioavailability of the drug. This platform can also replace older delivery systems such as oral suspensions and solutions. The FDA released guidance recently questioning the effectiveness, stability and safety of oral suspensions and solutions, stating dose accuracy as a primary issue. Orally dispersible dosage forms like flash ODTs mitigate dose accuracy issues and can provide a safe and efficacious option for these patient populations. 

Drug formulators need to rediscover that drug efficacy and safety are driven by patient behavior. Patient centric dosages can drive the types of behavior that result in easy administration, good adherence and, ultimately, positive medical outcomes- something currently adopted in OTC and nutraceutical formulations.  SPI Pharma sees a world in which prescription drugs are managed in a similar way. Enabled by patient centric formulations, it is emerging through orphan medications and pediatric indications. By focusing on smaller patient populations with unique needs, the Pharma Industry is posed for a significant shift in the way medication is delivered to meet patient experience requirements.