Trends from the 2016 PDA Annual Meeting

The event highlights the importance of engaged patients, advocacy groups and planning for personalized medicine.

Putting the patient first

The opening session’s message was quite clear, and indicative of the welcome shift in the pharma industry’s focus. “We’re all working for one person, that’s the patient,” began Maik Jornitz, President at G-Con Manufacturing, Inc. As experts have said, serving the patient is a privilege, but it comes with responsibility.

Dave deBronkart, also known as “e-Patient Dave” delivered a moving presentation about the rise of educated patients and the potential for improved outcomes. The spokesman for patient engagement knows first-hand about the journey from diagnosis to education and treatment. After being diagnosed with stage IV kidney cancer with multiple tumors throughout his body, he turned to an online patient community for advice. What he learned from his peers about treatments, doctors and side effects helped to save his life.

Because of the Internet, patients can connect to information and to each other (along with other healthcare providers). This becomes extremely beneficial when considering the gap between reality and awareness (i.e. what is known through patients and studies, and what has been published in literature). In his case, motivated, savvy patients knew certain published information was out of date, and that the risk of a treatment was lower than stated in literature. Additionally, he explained that there was nothing in the literature about getting through the side effects, but the online community prepared him with 17 first-hand stories of survival. He was clear not to dismiss literature, but said that some of the most useful knowledge remains outside of it.

Patient advocacy groups accelerating innovation

Non-profit organizations are playing an increasingly big role in drug development. Marina Kozak, Ph.D., is the science policy analyst at Friends of Cancer Research, an advocacy organization based in Washington, DC. “There’s a greater appreciation for non-profits in last few years,” she explained. “Patients know issues are often overlooked by providers and regulators.” Patient advocacy groups are helping bring those issues to light.

Non-profits have traditionally provided financial support to drive research, particularly when federal funding is harder to come by. But now they are impacting patients in new ways, promoting research through infrastructure support, legislation, and public-private partnerships by:

  • Developing specialized tools, including patient registries, genetic profiling, tissue banks and biomarkers

  • Prompting legislation that benefits new drug research

  • Fostering relationships and collaboration between industry, regulators, patients and academia that helps to identify gaps and accelerate innovation (including breakthrough therapy designation)

Kozak noted that there are ongoing efforts to modernize clinical trial design to increase patient enrollment, streamline operational efficiency, and ultimately benefit patients by bringing efficacious therapies to market sooner. Non-profits that speak for the patient are no longer simply providing funding, but are active partners in research and critical to the drug development ecosystem.

Supply chain and cold shipping in the era of personalized medicines

Cold chain logistics often involves considerable planning and customized testing and technology to ensure that products arrive safely. In the case of autologous cell therapies—products developed from the patient’s own cells—timing and tracking become even more crucial. Challenges include:

  • Distance - With apheresis collection sites often a flight away from production facilities, cells must be transported to production and the resulting drug is moved back to patient for infusion.

  • Supply – The drug is personalized, and therefore there is no backup supply. A damaged or lost shipment could mean the difference between life and death for a patient in need.

  • Temperature – Extra caution must be taken to ensure that finished drug products remain at cryogenic temperature (at or below –150 °C) during shipment.

  • Tracking – The labeling and tracking of shipments must be robust to ensure that a patient receives the correct medicine. Companies will have to determine what constitutes secondary packaging as well as best practices for labeling individual, personalized batches.

Experts agreed that the traditional linear supply chain is developing into a circular one, starting and ending with the patient. Additionally, the effort must include smooth data flow between the manufacturer, sponsor, clinician and payer to make the process secure and ensure product integrity.