What if there was a cancer trial that was a perfect fit for you, but your doctor did not even know it existed? That, unfortunately, is the sad reality today for many of the new immunotherapy drugs. “These medicines are not working for all advanced cancer patients but they are proving effective for some – even if sometimes only briefly,” says Renata Khoshroo Louwers, a writer and bladder cancer patient advocate with the Bladder Cancer Advocacy Network and the Research Advocacy Network. “Researchers are racing to figure out for whom they work and why.”
Louwers notes nowhere are these changes more evident than in advanced bladder cancer. Prior to May 2016, the FDA had not approved any new treatments for advanced bladder cancer since platinum-based chemotherapy in 1978, despite the fact that the median lifespan for patients with advanced bladder cancer was only 14 months.
The situation began to change in May 2016 when FDA approved an immunotherapy drug as a second-line treatment after platinum-based chemo. FDA has since approved four more immunotherapy drugs for advanced bladder cancer, including one as a first-line treatment for patients unable to tolerate chemotherapy. Three of these approvals have occurred within the last month. Unfortunately, the landscape is changing so fast, doctors are having difficulty keeping up.
Louwers has written about how hard it can be for patients to find and participate in clinical trials. She even participated, as a patient advocate, on a panel in Washington D.C. sponsored by the Coalition for Clinical Trial Awareness. She discussed the challenges patients encounter in finding and participating in clinical trials.
“There is little that is more emotionally challenging than seeing someone you love dying of cancer and knowing that there may be an answer – but you can’t find it,” notes Louwers. “There may be a drug that isn’t typically prescribed for this particular cancer or that has just been invented. There may be an actionable mutation sitting in a dataset of thousands of mutations but you just don’t know how to unlock it.”
Is there a solution? Patients talking to other patients may be a good start. As a cancer patient advocate for the last three years, Louwers has been impressed at the sophisticated understanding some patients and caregivers develop. She states anyone who says patients shouldn’t talk to each other because they might get bad information simply don’t know what it’s like to want to save the life of someone you care about. They also do not recognize that patients and families may be a lot sharper than they realize.
Marken Launches Hybrid Clinical Trials Service
Marken announced it has launched a hybrid logistics service which leverages the UPS global transportation network. The hybrid service enables Marken to utilize the UPS network, including its expansive airline, for a portion of the route. The hybrid offering reduces reliance on commercial aircraft for many routes. Hybrid solutions involving a mix of dedicated and common carrier fleets have been offered in the past. But, this is the first time the specialty and integrator services are owned by the same parent company, ensuring seamless integration and enhanced visibility.
One client already using the service has Marken collecting shipments of biologic material from sites in Russia, consolidating them in Moscow, and transporting them to their final destination using the UPS airline. UPS flies from Moscow to its hub in Cologne, Germany, and onward to all European destinations.
"We want customers to think of Marken as the clinical subsidiary of UPS," said Wes Wheeler, Marken's CEO. "We intend to continue executing on our strategy to be the supply chain service provider of choice for the clinical trials community, and to expand our broad range of services supporting the movement of clinical drug product, clinical drug substance, medical devices, biologic samples and other regulated materials."
Blow-Ups Spark Call For Clinical Trial Overhaul
Large international trials are under fire. In recent weeks, serious questions have been raised about three major heart failure trials, and there is no reason to believe these are the only trials about which questions will be raised. Further, there is no reason to believe the concerns are limited to heart failure trials or even cardiology trials in general. Instead, these questions almost certainly apply to many large international trials in many different clinical areas.
“In response to these concerns, clinical trialists are calling for a major overhaul of the way clinical trials are performed and overseen,” notes Larry Husten, writer at CardioExchange.org. “Safeguards and controls that existed in the past are no longer present, having been removed in the rush to streamline the process and reduce the expense of performing trials, they say. The current system has degenerated, and faith in results cannot be taken for granted as in the past.”
“There has been a great deal of internal discussion about reform, and interestingly, an unusually high degree of consensus,” said Milton Packer (Baylor University). “Amazingly, this has not been an issue that generates division among clinical investigators; it has actually generated an incredible amount of unity.” Packer promised: “Rely on us to meet together and fix this. We will.”
The immediate concerns about clinical trials were sparked by the investigators in a heart failure trial who uncovered compelling evidence that a large number of patients enrolled in Russia never took the study drug, thereby, undermining the reliability of the trial. Then a new analysis of another heart failure trial found strong evidence that large numbers of patients from Eastern Europe were ineligible for the trial and should not have been enrolled. Finally, a recent presentation of the main results of another acute heart failure trial (titled RELAX-AHF-2) raised the possibility that yet another trial may have been compromised.
Academic investigators appear to be united in their support for greater oversight and involvement. Clinical trial experts believe a basic underlying reason for the problems is the movement of clinical trial sites from the U.S. and Western Europe to countries and cultures less familiar with the principles of scientific research, including some but by no means all sites in Russia, Eastern Europe, and Asia.
Califf Makes A Fresh Start With Verily
After concluding a short stint as head of the FDA, former Commissioner Bob Califf is off on a new mission: One with Verily and his old employer, Duke University. He notes we are in the midst of an explosion of capability in computing and information, yet are still learning to translate that capacity into better health and healthcare.
“Bridging this gap has been a recurring theme of my career, and it’s at the heart of what I hope to accomplish at both institutions,” states Califf. “I’ll readily admit that I’ve chosen an ambitious problem to tackle, but I hope I can do my part.”
Califf first met Andy Conrad in 2004. The two found common ground in a vision to translate transformative technology and communication capabilities to healthcare. Those discussions led to Califf’s participation in Project Baseline, a program designed to create a data-driven understanding of human health. When Califf stopped work on the project to join FDA, Conrad launched Verily to achieve those goals.
“Now, as part of the Verily team, I’m hoping to offer insights that will allow the company to better tailor its technologies to meet the needs of doctors, other providers, health systems and the patients they serve, and to drive evidence-based approaches that will enable continuous learning and improvement,” says Califf. “Given my longstanding interest in applying technological solutions to improve healthcare, Verily’s ongoing work was naturally intensely interesting. But because I believe that the primary problem facing the healthcare enterprise is cultural, it also seemed appropriate that I keep one foot planted in a university-based integrated health system.”