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Clinical Trials Innovation

How patients are transforming pharma research and development

scrip-logo-smallPatient-centric R&D is imperative for pharma in delivering effective medicines and better outcomes. The push to implement it is challenging long-held biopharma business practices, including how clinical trials are designed, recruited and run – and what they measure.

Download the full seven-page report that originally appeared on Scrip here.

  • Commercial, scientific, technological and regulatory forces are urging the pharma industry toward greater patient-centricity.
  • This is impacting all stages of R&D, including clinical trial design, efficiency and endpoints.
  • New technologies are enabling pharma’s patient-centricity, offering tools to better understand disease and patients’ experience thereof, and to improve outcomes.
  • Several challenges remain, including which patient voices to incorporate into R&D decision-making, and how to do so systematically.
  • Yet patient-centric R&D is a business imperative for pharma in helping deliver the outcomes the industry needs to survive.

The pharmaceutical industry is in the grip of “patient-centricity” – a vigorous, vocal effort to put patients at the center of what it does and the drugs it develops. New positions have been created, divisions re-named, patient declarations written and published. Efforts are underway to change cultures and mind-sets within pharma to focus first and foremost on patients’ needs and priorities, rather than those of the health care professional, as has traditionally been the case.

The movement has a pleasingly ethical, feel-good aspect to it. But it’s driven by commercial, scientific, technological and regulatory/legislative forces in the health care industry that leave pharmaceutical firms with little choice but to embrace patient-centricity. Budget constraints and the drive for cost-effective care, as decreed within the Affordable Care Act, are forcing payers and providers to focus on the outcomes that medicines deliver to patients in the everyday setting. Those outcomes depend on whether patients are given appropriate therapy that they perceive as beneficial – and with which they’re motivated to remain compliant. Avoiding wasteful spending on ineffective treatment is a priority.

Science is evolving to focus on the individual, too. Advances in genomic tools and other “-omics” are driving the development of more personalized medicines, often tailored to individuals’ genetic mutations or susceptibilities. These advances are also allowing scientists to sub-segment diseases into ever-narrower categories. Half of trials now collect DNA from patients to help develop biomarkers, according to the Personalized Medicines Coalition. Meanwhile, industry’s focus on developing treatments for specialist, rare conditions continues.

If costs and science are compelling patient-centricity, digital technologies are helping enable it. They’re offering new, richer, more convenient and perhaps more accurate sources of patient data, helping to understand patients’ experiences but also the course of their disease. Improved data analytics and “big data” expertise are allowing scientists to extract new kinds of insights from huge numbers of patients – far more than a traditional clinical trial would allow. These novel information sources – behavioral, social and environmental, for instance – are starting to inform the broader directions that R&D should take. Meanwhile, increasingly sophisticated wearable devices and sensors allow clinicians to track heart rate, blood pressure, movement, sleep and much more, over long periods. Technology has also empowered consumers, who are driving the growth and development of advocacy groups, and a louder and more organized patient “voice.”

Regulators, too, are encouraging more patient involvement in the approval process. FDA is committed, under the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their condition and on available therapies. It has led the way in engaging with patients and seeking to incorporate their views into the regulatory process, including by encouraging greater use of patient-reported outcomes (PROs). (See sidebar, “Patient-Focused Regulators.”) PROs are measures captured directly by patients via a questionnaire or similar tool, and often pertain to aspects of treatment that matter to patients, such as how they feel and how a treatment is impacting their everyday lives and quality of life, rather than purely clinical endpoints used by physicians.

Download the full 7-page report that originally appeared on Scrip here.

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