chromosome

trends & perspectives in clinical research

goBalto Chromosome

To you it’s a document. To me it’s my life.

by Jae Chung
19Dec2016

T.J. Sharpe is an ordinary man with an extraordinary story. Faced with the prospect of a Stage IV Melanoma diagnosis back in 2012, he vowed to never give up, determined to see his two young children grow up with a father.

The Sharpe Family

Working with his oncology team, T.J. identified the best possible treatment option, which at the time came in the form of a promising immunotherapy clinical trial nearly four hours away from his home. After packing up his family and relocating to Tampa, he learned shortly after his arrival that the trial was delayed due to a pending signature on a clinical trial agreement (CTA), a contract. Without the luxury of time on his side, T.J.'s new oncologist suggested that he may have to consider "plan B." Determined not to let a document sitting on someone's desk get in the way of a potentially life-saving treatment for him or for other patients in the study, T.J. got to work. After a long process, which involved contacting the trial sponsor, finding the right person, and telling his story, the study team resolved the startup hurdle in a timely enough manner for T.J. to receive treatment. After a long journey, which included participation in second clinical trial, today T.J. is in remission and healthier than ever. He is also a compelling patient advocate for clinical research, a constant reminder of why research is conducted and what the industry is all about – bringing life-saving therapies to patients, who like T.J., have a limited window of opportunity to fight a devastating disease.

Watch T.J. Sharpe's video

Complexity compromises patient focus

Anyone who has been involved in a clinical research study team knows how challenging the day-to-day operations can be. Protocols are increasingly more complex, and studies are more global, spanning multiple countries and cultures. Research conducted by the Tufts Center for the Study of Drug Development (Tufts CSDD) during the past 15 years has demonstrated that, compared to 10 years ago, protocols have more endpoints, procedures, eligibility criteria, CRF (case report form) pages, amendments, and investigative sites. To give you a sense of the scale, a recent report estimates that the average study protocol now includes 13 endpoints, 167 procedures, 35 inclusion and exclusion criteria, and requirements for 11 site visits per patient over a 175-day period. With this level of operational complexity, it's easy to see why we get lost in the details of execution and the associated business processes.

Often when we think about inefficiencies in the clinical trial process, we focus on the role of the sponsor or CRO, a particular aspect of the value chain, or new technologies that promote data sharing and faster decision making. While these are critical aspects that drive day-to-day operations, T.J.'s story reminds us that there is another aspect of the value chain that we may be neglecting: the patient side.

The need for patient centricity

Clinical trials simply cannot happen without patients. In study startup, we talk about patient enrollment targets, recruiting strategies, and the like, often in abstract terms. We look at the data and consider aggregate numbers that drive decision making. For example, a study may target a particular investigator or site due to the PI's research or availability of patients at that location. But are we really thinking about this from a patient-centric perspective, or more from a site-centric perspective? There is a lot of hype about patient centricity these days, but T.J.'s story challenges us to think perhaps more carefully about what this means in practice. The fact that T.J. reached out to the sponsor of the research study is an uncommon practice, but should it be? Should we make it easier for prospective participants to engage with the study sponsor before, during and after the study?

Maybe it makes sense to think about the whole process from the patient side. I am a patient with a devastating disease, let's say an advanced cancer with a particular genotype that has been characterized. I have a physician who informs me that there are a handful of clinical studies which I might consider, two for targeted therapies, another that offers an immunotherapy. There is also a FDA-approved medication, which may be available as an off-label option. He is leaning towards the immunotherapy option that is in Phase I but gives me the choice in the end. So how do I go about making that decision? Are there other clinical trials that are coming up that my physician does not know about? Where do I go next? How do I know which trials are available, where they are, the study status, eligibility criteria, etc? The reality is that the "main" clinical trial finder at ClinicalTrials.gov is difficult to navigate for an average patient. The good news is that sources are emerging that are designed to match clinical trial participants to studies and address this major issue, including trial finders on other websites that may be more patient friendly and easy-to-use. For example, ClinicalConnection connects researchers and patients. They have a trial finder, and you can join their site and be notified when trials become available. There are many others, including the Cancer Research Institute, SmartPatients, and CureClick. Some of the options even offer a helpline or specialist navigators who help patients through the process. But taking it a step further, what if I could consult with the study sponsor directly to get another data point and find out if they have done any comparative studies, even in animals?

Benefits of a patient-centric focus

How does the sponsor benefit from such a patient-centric focus? First of all, it really takes patient engagement to the next level, in which informed patients are highly engaged patients that are more likely to comply with requirements, such as completing patient reported outcome questionnaires and attending follow up visits. That leads to better data integrity. Not only that, it could improve the number and diversity of enrollment, which increasingly is seen as a major issue in clinical research. One notable example was recently published by researchers at Harvard Medical School, whose research showed that that over the last decade, genetic testing may have disproportionately misdiagnosed hypertrophic cardiomyopathy (HCM) in black Americans due to lack of diversity in genetic studies. Recognizing the importance of the issue, the FDA has become a strong advocate for increased diversity in clinical research. In fact, 2016 has been dubbed "The Year of Diversity in Clinical Trials" by the FDA, part of a coordinated campaign designed to increase the diversity of patient populations participating in clinical trials and eliminate potential bias or erroneous trial outcomes.

Are there other potential up-sides? If a patient engages with a sponsor, even if they decide to forgo participation in that particular study, they may become a potential candidate for a future study – perhaps in a later phase or for an entirely different study. Beyond study startup, you could imagine engaging the patient directly could have other benefits. What if you could invite patients to share their experiences with each other as part of the study via a closed, secure social media channel and also submit questions to the sponsor? This becomes another valuable data source and potential resource for mining trends that can be compared with endpoint information.

The end result? Turning our focus toward a true patient perspective gives us ways to think about innovative solutions for accelerating clinical research. T.J. Sharpe and patients like him may just hold the key to unlocking the next frontier in clinical research.

Follow T.J.'s story at www.philly.com/patient1/

Jae Chung
JAE CHUNG
President and Founder
goBalto, Inc
Jae Chung is the president and founding visionary of goBalto. A startup evangelist, Chung wants to change the way pharma and CRO companies initiate clinical trials. goBalto's purpose-built study startup SaaS solution allows stakeholders to better adhere to established timelines and budgets, with customers reporting reduction in cycle times by 30-plus percentage, thereby getting medicines to those in need faster.

Chung works with Rock Health to mentor healthcare technology startups, and previously co-founded Celltrion (068270:KOSDAQ), a leading biopharmaceutical manufacturing company. Prior to Celltrion, he worked as a strategy consultant with McKinsey & Company.

In 2013 Jae was recognized as a FierceBiotechIT Top-10 Techie list and in 2010 was awarded the Bio-IT World Judges Prize for Technology Innovation. Jae has experience in drug development, commercialization, and business development. He has an MBA from New York University and holds a CPA.

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