Key Clinical Trial Themes from DIA 2014

The Drug Information Association (DIA), one of the largest non-profit organizations supporting clinical research and drug development, held its 2014 annual meeting in San Diego last week. Applied Clinical Trials’ Moe Alsumidaie looks at three of the key themes from this year’s meeting: incorporating advocacy groups and patient voices in clinical research, breakthrough research applications, and new data collection methodologies.

Incorporating advocacy groups and patients in clinical research and study design is becoming more important for sponsors and the FDA in bringing innovative products to the patient. Payers are relying more on advocacy groups and patients to determine new medical product reimbursement guidelines, and the FDA is involving these groups to help with determining whether a medical product is a viable candidate for R&D.

“Advocacy groups are the middle between where the drugs are being developed and the patients that are going to receive them, we are the voice to educate patients, and it is working,” said Bonnie Addario, Founder and Chair of the Bonnie J. Addario Cancer Foundation. “The Master Lung Protocol with the FDA has been a huge collaboration, and one that I feel [all parties] are listening.  We’ve created a trial that is going to be a game changer on how to design a clinical trial; looking for multiple drugs at one time rather than finding the drug first and then just slamming the patient with a drug that we don’t even know is going to work.  Patients love [the master lung protocol]!” added Addario.

Companies such as Organovo are starting to apply 3D bioprinting in manufacturing bioprinted human tissues, and there was discussion on the technology itself and how the FDA is addressing it in the regulatory approval process. Some bioprinted materials that were successfully implanted in humans included a 3D bioprinted titanium mandible, and a cranioplasty, and other bioprinted materials included artificial ears and printed kidneys. In the 3D printing arena, key considerations for review include imaging, digital design, and printing, according to the FDA.

“We have embraced 3-D manufacturing as a core concept, we recognize that it is coming over the years to devices and components of devices,” Said Steven Pollack, Director of FDA’s Office of Science and Engineering Laboratories at FDA’s Center for Devices and Radiological Health. According to Pollack, the FDA initiated a working group that incorporates engineering/medical device and drug development/organ tissue regulatory pathways (CDER/CBER) in order to support future organ/tissue development via 3D printing.

“Many of the simple organs are tubular (vasculature, alimentary canal, airway, etc.) and we’d like to see enabling technologies [in 3D printing] to create tubular structures for organs,” added Pollack. “While full replacement organs may be 10-15 years away, in the shorter term we can make smaller functional replacement tissues and other things such as arteries, heart valves, and patches that will all help replace, repair or augment organ function. The greatest challenge with full size organs is always going to be creating an integrated vascular network that will nourish very large tissues. But fortunately, there is a great deal of organ function that can be replaced with smaller scale tissues,” said Eric David, Cofounder and Chief Strategy Officer of Organovo. “I believe payers will be on board, but the outstanding question is how much will they be willing to cover, as generating these complex tissues will likely carry an expensive manufacturing costs relative to today’s therapeutics,” indicated Brian Strem, Director of Business Development at Allergan.

Data collection from biosensors was also a theme at DIA. Alison Greene, Sr. Scientist of Patient Reported Outcomes at Genentech, presented findings from an observational study comparing patient reported outcomes collected via eDiary format to data captured by a biosensor (placed on the patient’s arm). The study found that biosensors collect a vast amount of data that potentially builds a comprehensive picture of patient activity. The results indicated that most patients did not have a preference for either method, although those who did preferred the eDiary. At end-of-study, most patients indicated that they would prefer a different biosensor than the one used in the study.

“Patient reported outcomes are critical to understanding the patient’s perspective of treatment benefit. Biosensors will never replace direct patient report because they only give us data,” said Greene. With regards to leveraging biosensors for patient engagement and patient centric models, Greene indicated, “biosensors have incredible potential to help us understand the impact of treatment in clinical trials. We learned that subjects liked the idea of wearable technology; using a biosensor in a study is feasible and we can interpret the data. The next step is to understand how biosensor data can bring another dimension of knowledge to patient centered outcomes and ultimately help inform study endpoints.”