While clinical trials are still the mainstay for generating efficacy and safety data, the industry is facing a revolution in the way it designs, plans, conducts and analyses studies, Dr Patrick Delavault – senior vice-president and chief medical officer, Global Medical & Regulatory Sciences, at specialty pharma company Ipsen – told an audience of senior clinical colleagues at the recent eyeforpharma Clinical Excellence Europe conference.
“The traditional, empirical model based on population-centric models and the randomisation of populations that satisfy inclusion-exclusion criteria will need to evolve towards a more sophisticate patient-centric model with adaptive study design,” he says, pointing to the advances seen in precision oncology.
Many new challenges face pharma, he says. “The need to produce real-world evidence beyond the constraints of clinical trials, crowdsourced research protocol initiatives, digital patient communities as a launchpad for patient-centric trial design and the big data environment that means clinical trials are no longer the only source of data to drive medical learning.” For example, the use of electronic medical records to inform optimal prescribing decisions.
However, for Delavault, the most important transformation is the central role that patients are playing in the clinical research arena. “[Patient involvement] cannot be disconnected from the evolution of science, medicine and technologies; now we have wearables and biosensors, social media, big data analysis and the shift in our patients behaviour as they take a more proactive role. We are no longer in the same era.”
Patient insight is a powerful driver of future clinical trial success, he says. “We are seeing the emergence of new paradigms such as patient preference research throughout the R&D value chain. In the clinical phase, it can help identify the endpoints that are important to patients or an aspect of trial design that may hamper patient participation or adherence.”
Yet, questions remain unanswered. “How do we address these evolutions? How do we leverage input from patients in our clinical research programmes? How do we better communicate with patients about the research objectives?” he says.
“Improving patient-centric clinical research means two things; firstly, making sure industry and other stakeholders are seeking patient insights and incorporating them in the design of trials, and, secondly making sure patients understand pharma processes and the constraints of designing, planning, running and reporting trials.”
An holistic approach is needed if pharma is to realise the vision of delivering better, patient-centric clinical trials, he says, pointing to both the EMA pilot programme aimed to include patient views in assessments and the FDA Voice of the Patients initiative.
Collaboration and innovative ways of working are essential, says Delavault, citing the Cystic Fibrosis Foundation and its early-stage funding to biotechnology companies for breakthrough therapies. He quotes former FDA Commissioner Dr Margaret Hamburg: “The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development.”
