Protocol Development #10: QbD, CIDs, Patient Engagement
In the first double-digit newsletter edition, there's fewer articles than normal - whether this is suggestive of the summer slowdown or a competing end-of-academic-year marking schedule is unclear. In this edition, there's some important articles on QbD and master protocols, however, patient engagement provides the most numerous take-home messages - with important contributions from Pfizer (that may be more relevant to protocol developers in industry). In the education corner there's an interesting article of drug delivery that's worth a read regardless of your role within clinical research. Lastly, if you've got a spare 30 mins on Tuesday there's a webinar that’s looking to address how DCTs will enhance diversity and adherence.
QbD
Stegemann et al published a review article on Rational and practical considerations to guide a target product profile for patient-centric drug product development with measurable patient outcomes – A proposed roadmap. Target product profiles (TPPs) are an important source of information for clinical study design and can substantially influence a protocol's development. Patient centricity is often considered at the individual study level, however, in this article the authors propose an even earlier engagement with the intended patient population in order to enhance acceptability and usability of the intervention. By doing so, the patient centric approach to the TPP feeds into an overall QbD strategy whereby the interventions development and subsequent clinical studies are inclusive of the target population's needs rather than uniquely based on efficacy and safety.
CIDs/Master Protocols
Williams et al published an article on the Approach for reporting master protocol study designs on ClinicalTrials.gov: qualitative analysis. In the US-preferred 'Master Protocol Research Programs' (MPRPs) terminology, the authors provide a significant "must read" insight into CT.gov registration and reporting of master protocols - a common and problematic issue when designing master protocols. The outcome of the work is that master protocol substudies and any common screening protocols should be independently registered using multiple study records. By doing so, valuable study details would not be obfuscated by bundling all substudy data in a single record.
Patient Engagement
Guerra et al published an article on Strategies to Advance Equity in Cancer Clinical Trials. The authors touch on several protocol sections including eligibility (broadening eligibility criteria per ASCO and FDA guidance) as well as screening (expanding screening capabilities from both study and patient perspectives), and general recruitment strategies (a relatively new requirement for industry protocols related to EU CTR 536/2014).
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Rottas et al published an analysis on the Demographic diversity of participants in Pfizer sponsored clinical trials in the United States. In their analysis of 213 interventional studies between 2011-2020 the authors sought to provide a baseline for study diversity in the US. Aside from an underrepresentation of Hispanic or Latino participants, the representation of other ethnic or racial groups and gender was in line with the US census. The work provides an important reference for understanding diversity in industry-led studies.
In another Pfizer publication, Masters et al published an article on Ensuring diversity in clinical trials: The role of clinical pharmacology. The authors point to several program-level activities to enhance diversity in clinical studies - including the development of a model-informed drug development (MIDD) plan that guides overall strategy and diversity goals. The spotlight on a clinical pharmacologists' role in improving racial and ethnic representation is significant as it assigns some direct responsibility to a specific role rather than more generally to study teams.
Education Corner
Mike May's article on Why drug delivery is the key to new medicines is an interesting read and draws attention to the contents of the protocol section Study Intervention (and concomitant therapy). The efficacy and safety of an intervention are frequently discussed characteristics, however, outside of CMC, the delivery of interventions is not something given much consideration (unless it's still under discussion that is). In the article, Mike puts a spotlight on the successes of lipid nanoparticles in the delivery of the mRNA vaccines as well as discussing other advancements in methods of intervention delivery.
Webinars
Almac, THREAD, and AARDEX Group are hosting a webinar on 28 June 2022 16:00-16:30 CEST: A Prescription for Pharma: How DCT Technology Partnerships Deliver Better Patient Journeys, Diversity, and Adherence. The discussion points are: