Pragmatic, real-world trials will bring treatments to patients

Related tags Pharmacology

Adoption of a pragmatic, real-world research culture would bridge the gap between discovery and treating the intended patients.

Translational research has succeeded in moving molecules from the laboratory into the clinic and further improvements would benefit patients and biopharm. Conducting studies in real-life settings to get real-life results, known as pragmatic research, is proposed to optimise the process.

It is about bringing the right intervention to the right patient at the right time, and that is the essence of “pragmatic research”​”, says a piece published in the journal of Pragmatic and Observational Research​.

To achieve this “long overdue​” goal​new, innovative approaches must be adopted alongside expansion and improvements of current methods, says Abdul Rahman Jazieh, of King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia.

For instance, use of expanded-access programmes, which make investigational new drugs available to patients outside of clinical trials​, should be increased. Using expanded-access could tap into a larger patient pool and provide data from a more real-world setting than trials.

Widespread use of expanded-access should be complemented by innovative study designs. These should produce results that are relevant to real-world patients and be conducted as early as possible in the regulatory process to support systemic collection of safety and efficacy data.

Particular efforts should be made to test medicines in different ethnic groups to assess the impact of pharmacogenomic variability prior to widespread use of a medicine. Globalisation of the pharmaceutical and clinical trial sectors has increased focus on pharmacogenomic variability.

Finally, Jazieh says research infrastructure should be revamped through increased use of practice-based trials. Using a network of doctors’ practices will increase the accessible patient population and “may help to identify the gap between recommended care and actual care​”.

Implementing these changes is intended to increase the validity of extrapolating clinical trial findings across the broad heterogeneous patient populations that receive marketed therapeutics.

Related news

Related products

show more

Automated metadata management in clinical trials

Automated metadata management in clinical trials

Content provided by Formedix | 01-Aug-2023 | White Paper

When it comes to efficient clinical study build, content is king. Most importantly: metadata content. In this blog, we explore the role of metadata in...

Small Molecule development – getting it right

Small Molecule development – getting it right

Content provided by Lonza Small Molecules | 20-Jun-2023 | Insight Guide

Small Molecule drug development is something more and more ambitious emerging pharmaceutical companies are taking on from end to end. But this path can...

Validate clinical study data with Formedix CORE

Validate clinical study data with Formedix CORE

Content provided by Formedix | 19-Jun-2023 | White Paper

In April 2023 at the CDISC Europe Interchange, we launched Formedix CORE, the first free-to-use, downloadable application encompassing the CDISC Open Rules...

The Right CDMO Can Unlock Faster Drug Development

The Right CDMO Can Unlock Faster Drug Development

Content provided by Lonza Small Molecules | 15-May-2023 | Interview

Drug development presents biotech companies with ever-changing challenges. Enabling a faster end-to-end process requires seamless flexibility and extensive...

Related suppliers

Follow us

Products

View more

Webinars