Recent Publications

The development of modified-release (MR) drug products aims to address a clinical need such as improving patient compliance. There are multiple pathways and development strategies for the registration and approval of MR products. The development strategy of an MR product is usually dependent on the availability and pharmacokinetic/pharmacodynamics (PK/PD) characteristics of the reference drug product, that is, an immediate-release (IR) product or a reference MR. Compared with a reference IR product, an MR product is likely to have a different PK profile over the least common dosing time due to unequal dosing intervals. In case of differences in PK profiles between the MR product and the reference product, confirmatory efficacy and safety studies may be needed to support registration. 

Poor adherence to oral antiretroviral therapy (ART) remains an important challenge in the treatment of HIV. Microneedles (MN) potentially could offer a non-invasive long-acting (LA) delivery approach, avoiding the need for daily dosing of ART. However, this claim has yet to be explored amongst its potential end-users. The aim of this mixed methods study was to investigate the perspectives from various end-users surrounding the translation of MN technology to general clinical practice, with a particular focus on delivery of ART. Quantitative postal questionnaires were distributed amongst healthcare professionals (HCPs) and the lay public (LP). A total of 208 responses were obtained (HCP, 69; LP, 139), with a completion rate of 34.7%. The consensus on MN technology was positive from both demographics (HCP, 97.1%; LP, 98.6%), with further strong support of postulated MN use within HIV (HCP, 97.1%; LP, 98.6%). 

Cell and gene therapies have the potential to be curative for severe disease states such as cancer or incurable orphan genetic diseases. Despite the promise, there are only few such therapies available, although more are appearing in pharmaceutical pipelines. A major culprit limiting a fast translation from preclinical research to the clinic and the market is chemistry, manufacturing and control. The root cause is that most cell and gene therapies currently are personalized in form of ex vivo manipulated cells. This approach stands in sharp contrast to the population-based approach seen for small molecules and protein therapeutics. Therefore, it warrants a different approach to product manufacturing, testing, release, regulatory submissions, and product distribution.

Decision-making in drug development benefits from an integrated systems approach, where the stakeholders identify and address the critical questions for the system through carefully designed and performed studies. Biopharmaceutics Risk Assessment Roadmap (BioRAM) is such a systems approach for application of systems thinking to patient focused and timely decision-making, suitable for all stages of drug discovery and development. We described the BioRAM therapy-driven drug delivery framework, strategic roadmap, and integrated risk assessment instrument (BioRAM Scoring Grid) in previous publications (J Pharm Sci 103:3377-97, 2014; J Pharm Sci 105:3243-55, 2016).