Children are not small adults

Interview with Dr. Annette Grave and Philippe Tschopp of Glatt Pharmaceutical Services on child-appropriate dosage forms, a recent project and the future of patient-centred formulations 

From the initial concept to use by patients, pharmaceutical development is often a long journey. Alongside efficacy and safety, one question has become increasingly important: how can a medicine be developed to meet the specific needs of the patient group that will use it? 

A recent project for a specialty pharmaceutical company illustrates both the challenges and the opportunities associated with paediatric medicines. In this interview, Dr. Annette Grave and Philippe Tschopp of Glatt Pharmaceutical Services discuss the importance of patient-centred formulations, the lasting lessons they have learned on recent projects and the developments likely to shape the field in the years ahead. 

 

Denis Bozbag: Dr. Grave, why are paediatric formulations receiving greater attention today? 

Dr. Annette Grave: The key insight is simple: children are not small adults. Their metabolism works differently and their medical needs can be very different from those of adult patients. 

It is really not as simple as taking an existing medicine and lowering the dose. Since the introduction of the EU Paediatric Regulation in 2007, pharmaceutical companies have had to consider, early in the development cycle, whether and how new medicines can also be made suitable for children. As a result, paediatric development is now addressed much more systematically than it was a few decades ago. 

 

Denis Bozbag: What does a well-designed paediatric formulation need to achieve? 

Dr. Annette Grave: A paediatric medicine must be effective, safe and, above all, acceptable to the child. Even the most sophisticated formulation will not work if a child cannot or will not take it. 

That is why taste, size and dosing flexibility are so important when developing medicines for children. The selection of appropriate excipients is also important: ingredients suitable for adults are not necessarily appropriate for paediatric patients. 

 

Denis Bozbag: What was the original objective of the most recent client project, and what was Glatt’s role? 

Dr. Annette Grave: A well-known, existing API is used to treat children with adrenal insufficiency who need lifelong hormone replacement therapy. When we started this project, no approved formulation was available for children at the low doses required. Parents and pharmacies often had to adapt adult medicines by crushing tablets and preparing smaller doses, an approach that was neither ideal nor particularly precise. 

There was a European-funded project with research and industry partners. Within the consortium, Glatt Pharmaceutical Services was responsible for the formulation development. In collaboration with universities and clinical partners, we developed a dosage form specifically designed for children that was subsequently approved. 

 

Denis Bozbag: Why is this still relevant today? 

Dr. Annette Grave: It highlights two important points. First, it demonstrates that small patient populations also deserve medicines specifically designed for their needs. Second, it shows how patient-centred development can work in practice: the product was consistently developed with the needs of young patients in mind. 

For children, the correct dose is essential, but taste, ease of administration and dosing flexibility also matter. The product uses a multiparticulate system: small, taste-masked granules supplied in capsules. The capsules can be opened, and the pellets can either be administered directly or sprinkled onto soft food. The formulation enables precise dosing and can make everyday administration easier for families. 

 

Denis Bozbag: You mentioned multiparticulate systems. What advantages do these dosage forms offer? 

Dr. Annette Grave: Multiparticulate systems consist of many small individual units, such as pellets or mini-tablets. They provide several advantages: different strengths can be produced flexibly by filling different quantities of the smaller units. In addition, individual particles can be taste-masked and engineered to provide the desired release profiles. They can also be sprinkled onto food, which can substantially simplify administration. 

These characteristics are particularly valuable for children, but they are not limited to paediatric patients. Older people and patients with swallowing difficulties can also benefit from dosage forms that are easy to administer. 

Despite their potential, pellets are still used less widely in the pharmaceutical industry than one might expect. Many companies have been working with established technologies for years and are understandably hesitant to introduce new platforms. There is still a need to raise awareness of the possibilities multiparticulate systems can offer. 

 

Denis Bozbag: Which lessons continue to inform new projects? 

Dr. Annette Grave: Every development project generates valuable experience. In our most recent project, we demonstrated that very low doses of an active pharmaceutical ingredient can be manufactured with a high degree of accuracy. We also gained extensive experience in drug layering, coating technologies and taste masking. 

Because the project received public funding, many findings could be presented at conferences and published in scientific literature. In this way, the project contributed to the advancement of the field beyond the product itself. 

 

A CDMO Perspective

Denis Bozbag: Mr. Tschopp, what is the importance of projects like this for Glatt Pharmaceutical Services today? 

Philippe Tschopp: Specialty medicines and niche products are a core part of our business. Complex, high-value products are especially well suited to our business model. Projects of this kind enable close collaboration with customers throughout the product lifecycle — from development through to commercial manufacturing. 

As volumes are often comparatively small, it is possible to establish long-term and economically sustainable production solutions. This is an important consideration for many customers developing specialised medicines. 

 

Denis Bozbag: Are you seeing rising demand for paediatric or more broadly patient-centred formulations? 

Philippe Tschopp: Paediatric medicines and patient-centred specialty formulations are fundamentally important topics. Whether they develop into market-ready products, however, often depends on the commercial framework. 

We see relevant activity particularly in rare diseases and among specialised orphan-drug companies. For larger standard products, additional development costs can be more difficult to justify, even where the medical benefit is clear. 

 

Denis Bozbag: Why is this project still used as a reference point? 

Philippe Tschopp: It is an excellent example of how a complex development challenge can be translated into a successful commercial product. Such references are highly valuable to prospective customers because they demonstrate that a technology not only works in the laboratory but can also be successfully scaled up and established in the market. 

Furthermore, the project illustrates the contribution patient-centred formulations can make. When medicines are better tailored to the needs of specific patient groups, both patients and healthcare providers ultimately benefit from simpler and more reliable administration.