Pharma data collaboration speeds R&D

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Historic clinical trial data represent a valuable and, until now, relatively untapped resource that could accelerate research and development of new drugs. Ed Bowen explains how the Placebo & Standard of Care initiative is helping to overcome the challenges.

Increased expectations, demand for speed, skyrocketing costs and design complexities; these are just a few of the challenges that impact the success of clinical trials today.

As anyone in the biopharmaceutical industry knows, developing a drug and bringing it to market has always been an arduous and time-consuming endeavour. But the industry is reaching a point of critical mass – a point that demands major change from the globe's leading biopharmaceutical and clinical development forces.

Fortunately, there have been some major efforts to identify clinical trial pain points and develop solutions to address them. Among these is TransCelerate BioPharma, a consortium of biopharmaceutical companies working together to simplify and enhance R&D processes in order to minimise inefficiencies through innovative, new solutions.

One major hurdle for sponsor companies has been lack of access to, and utilisation of, historical clinical data that could help enhance clinical trial designs, develop disease models, provide context for safety observations, improve patient recruitment and, importantly, bring better medications to market faster.

Innovative solution

Traditionally, patient data in a clinical trial is collected, examined for a specific trial and then never re-used. Further, until recently, few methods existed to maximise its value.

TransCelerate established the Placebo and Standard of Care (PSoC) Initiative to collect and share anonymised clinical data historically gathered in the placebo and standard-of-care arms of clinical trials among participating member companies.

The use of historical data in clinical development may be familiar to developers of rare disease drugs, but researchers and sponsor companies are realising the power of its use in trials that impact larger patient populations as well.

Randomised Controlled Trials (RCTs) are the current gold standard of drug development – and for good reason. But RCTs are resource-intensive and time-consuming; integrating historical data could help mitigate these concerns. With proper understanding and the matching of study design and demographic parameters, historical data can be used as a supplementary tool to reduce the number of control subjects needed in a trial, meaning that those patients will have access to the new therapy. Taking it one step further, historical data could be used as replacements for subjects, avoiding the need for a control arm altogether.

To simplify and guide the use of placebo and standard-of-care data, TransCelerate is developing a repository that collects and organises anonymised patient data from previous clinical trials to be used as input and applied to new trials.

The database

Members of the PSoC team at TransCelerate released a white paper last autumn on the potential applications of a large historical PSoC database and examples of implementation for the industry at large.

A collaborative platform, it will contain anonymised GCP-compliant demographic, safety and medical data on tens of thousands of subjects from both placebo and active arms of previous studies. The database will collect approximately 150 completed clinical studies within the first three years of the building period, with initial interest in studies on Alzheimer's disease, diabetes, schizophrenia and cardiovascular disease, to name a few.

Current and future applications

The PSoC database has the potential for additional possible applications that can advance drug development:

1. Information exchange: Biopharmaceutical companies are increasingly recognising the power of exchanging information to develop a better understanding of diseases and, in turn, better drugs. If one company specialises in a certain disease, for example, having access to its data may enable others to create better disease progression models.

2. Increase drug safety: The database may also contribute to drug safety. Safety events of interest typically require an adequate understanding of the type and frequency of expected and unexpected adverse events specific to the populations being studied. Patient safety is a paramount concern of sponsors. In addition, the process of actually investigating safety/adverse events can result in months of delay to product development, impacting resources and costs. Indeed, clinical development delay or failure in phase 3 can often be caused by safety-related questions.

3. Ease of recruitment: As mentioned, another potential application of the database could be its ability to reduce the number of prospective subjects required during clinical development. This benefits patients by requiring fewer of them to participate in clinical trials to bring medications to market. Of those patients still required to participate, fewer patients would be randomised to the standard-of-care arms, reducing potential patient exposure.

It may also boost the speed of study start-up, since less time is dedicated to recruitment. Fewer patients in a trial means overall costs and resources could be reduced through lower numbers of clinical visits, costly tests and investigator sites. Freed resources can be allocated to underfunded projects; freed time means critical medicines are reaching patients faster.

4. Biomarker exploration: The PSoC database also has the potential to contribute to biomarker development – a revolutionary path towards innovative medicines. With a large-scale PSoC clinical database, drug developers will have the opportunity to explore elements of biomarker research more accurately than is presently possible. Current clinical trials are conducted globally, leading to inherited variables between regions or countries in terms of subject characteristics, disease progression and other factors that require examination. Access to a PSoC database could lead to more precise understanding of the relationship between biomarkers and clinical outcome endpoints, and with less bias. Ultimately, development of biomarkers benefits patients directly by not only speeding drug development, but also by potentially identifying disease faster.

Measures like PSoC data sharing, enabled by TransCelerate, are challenging the traditional clinical trial model to bring about real change. It's an exciting time for research and development; true revolution and innovation are right around the corner.

About the author:

Ed Bowen is the Senior Director of Translational and Bioinformatics at Pfizer and leads the TransCelerate Placebo and Standard-of-Care Data Sharing Initiative.

In his role at Pfizer, he is responsible for strategy and execution of technology efforts in translational research and bioinformatics for the Research and Clinical Research teams executing target selection/validation, biomarker identification and patient stratification using technologies for analysing gene expression, whole genome analysis, next-generation DNA sequencing, integration of clinical and molecular data, and pathway analysis. He has 14 years of experience at Pfizer developing large-scale data management and analytics solutions for electronic medical record data, 100 million+ patient claims datasets, clinical trial data, and primary pharmacology data.

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22 February, 2016