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从分子到患者影响:患者依从性与辅料创新

From Molecule to Patient Impact

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文章指出美国每年约12.5万例可预防死亡与用药不依从相关,呼吁从制剂设计早期就将患者需求纳入药物开发。文章从适口性与给药形式、便利性、个性化给药三方面展开,提及羟丙基-β-环糊精(HPβCD)掩味、缓释制剂减少给药频率,以及3D打印可实现剂量、形状与释放曲线的定制。Roquette的研究显示,改性淀粉基配方结合山梨醇等辅料具有应用潜力。

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 From Molecule to Patient Impact

The pharma industry has made remarkable progress in developing new treatments over the past decade, from the rapid rollout of COVID vaccines to breakthroughs in cell and gene therapies for rare diseases. But even the most innovative treatments only work if patients are willing to take them.

Medical non-adherence is a silent epidemic. In the U.S. alone, it causes approximately 125,000 preventable deaths annually.1 Unpleasant sensory profiles, inconvenient dosing schedules and other delivery challenges can keep patients from following treatment regimens, with serious health implications. Therapeutic success, therefore, depends not only on active ingredients, but also on patient experience.

To close the adherence gap, patient needs and experiences should be embedded into drug development from the earliest stages of formulation design. This article will explore three areas where more patient-centric thinking can improve outcomes — from palatability and delivery formats to increasingly personalized therapies — and the critical role excipients play in making this possible.

Better-Tasting Medicines, Better Outcomes

Palatability is a clear example of where patient-centric formulation often falls short. Taste plays a critical role in medication adherence, particularly among children and older adults, who tend to be more sensitive to unpleasant flavors. Effective taste-masking is therefore essential for supporting adherence. For example, some antibiotics have a bitter flavor and can cause nausea upon administration,2-3 leading to poor compliance and reluctance to continue treatment. To overcome challenges like this, formulators are increasingly turning to functional excipients that improve palatability. Some molecules can encapsulate bitter compounds and reduce interactions with taste receptors in the mouth.4 Cyclodextrins like hydroxypropyl-β-cyclodextrin (HPβCD), for example, have been shown to successfully mask the bitter taste of active ingredients, thereby alleviating immediate side effects such as gagging and nausea.

Combined with other taste-masking approaches such as using sweeteners, coatings, or other masking technologies, these solutions are supporting the development of more pleasant tasting dosage forms across a range of formats, from chewables to orally disintegrating tablets to liquid formats.

Convenience Drives Adherence

Patient experience extends beyond taste. How medicines fit into patients’ daily lives — including how often meds need to be taken and how easy they are to administer — also plays a major role in long-term adherence. The vast majority of medications prescribed to patients around the world fall into the immediate-release category, where the objective is to deliver the active pharmaceutical ingredient (API) promptly after administration. This can result in people having to take medicine more regularly which can become a burden, particularly in chronic disease management.

Modified release formulations can help to address this challenge by reducing dosing frequency and improving convenience. As an example, some diabetes drugs have historically been associated with gastrointestinal side effects and frequent dosing requirements. Extended-release formulations help reduce pill burden while providing a more consistent release profile, improving both patient convenience and comfort.

At the same time, the industry is seeing growing demand for alternative oral dosage forms that better accommodate diverse patient needs. Not all patients can or want to take traditional tablets. Chewables, lozenges, orally dissolving films, mini tablets, effervescent tablets, liquid formulations, or medicated confectionery are increasingly being used to improve patient and consumer compliance, particularly among pediatric and geriatric populations. These dosage forms require carefully selected excipients that can support stability, controlled release and manufacturability, while maintaining a positive sensory experience.

The rise of biologics is also reshaping drug delivery. GLP-1 receptor agonists, for example, represent a major advance in healthcare, but their injectable format can create barriers to long-term adherence.5-6 This highlights the need for more patient-friendly delivery approaches, including oral formats, to better support the treatment experience.

Personalized Drug Delivery Nears Reality

The era of ‘one-size-fits-all’ medicine is giving way to a more personalized approach to drug design. In this environment, 3D printing, also known as additive manufacturing, offers advantages that traditional mass production cannot easily match. With greater customization and smaller batches, 3D-printed drugs can potentially be tailored in size, shape, texture, flavor, dose and release profile to suit individual patient needs. Dose flexibility is particularly compelling, allowing strength to be adjusted precisely for each patient — a valuable capability in pediatric, geriatric and hospital pharmacy settings, where standard dosing may not always fit.

The intricate geometries created through additive manufacturing also create opportunities for spatially controlled release profiles. Layered tablet structures could even allow multiple APIs to be combined into a single polypill with staggered release patterns, significantly reducing pill burden for patients managing chronic conditions.

However, much of this promise depends on excipient innovation. Excipients influence key formulation parameters including rheology, flow, melt behavior, mechanical properties, printability and drug release. Selecting the right excipient is therefore critical to 3D-printed drug product success. Plant-based excipients are emerging as particularly promising materials. Polymers such as starches and polyols like mannitol and maltitol can offer relevant functional properties depending on the printing technology and formulation design. Our research at Roquette7 shows that modified starch-based formulations using sorbitol or mannitol as plasticizers could generate printable filaments for 3D printing, supporting immediate and controlled release of a model API in tablet form. Together, these advances position excipients as key enablers of the next generation of personalized therapies.

Looking Ahead

The future of pharma will be shaped by patient-centric drug design. Closing the non-adherence gap will require closer collaboration across the healthcare ecosystem, bringing together formulation scientists, excipient specialists, drug developers, regulators, clinicians and technology partners earlier in development.

Ultimately, putting patient needs at the heart of development gives us an opportunity to unlock the full potential of the therapies we work so hard to create — making them not only more effective, but also more convenient, palatable and easier to integrate into everyday life.

References

1. Khan, R, and Socha-Ditrich, K. (2018). Investing in medication adherence improves health outcomes and health system efficiency: Adherence to medicines for diabetes, hypertension, and hyperlipidaemia. OECD Health Working Papers. 105.
2. Uchida, T., et. al. (2003). Evaluation of the bitterness of antibiotics using a taste sensor. Journal of Pharmacy and Pharmacology. 55.
3. Wright, J. and Paauw, D. (2013). Complications of antibiotic therapy. The Medical clinics of North America. 97(4). 667-679.
4. Jansook, P., et. al. (2018). Cyclodextrins: structure, physicochemical properties and pharmaceutical applications. International journal of pharmaceutics. 535(1-2). 272-284.
5. GLP-1 Market Industry Trends and Global Forecasts 2024-2035: Over 150 GLP-1 Drug Candidates in Development for Multiple Indications, Type 2 Diabetes and Obesity Drive GLP-1 Drug Development Boom. (2025, Mar). Research and Markets.
6. Catanese, L. (2024, Feb). GLP-1 diabetes and weight-loss drug side effects: "Ozempic face" and more. Harvard Health Publishing.
7. Chow, K.T., et. al. (2026, Sept). Exploring Applications of Plant-Derived Polymers in Fused Deposition Modeling of Oral Pharmaceutical Tablets. Roquette.

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