Mark Vermette

	The convergence of cancer research and clinical trials holds an insurmountable weight in the healthcare realm during the 21st century. With new technologies rapidly evolving each day, the speed of research and trials is at an all-time high. It is imperative to support the clinical trials working to bring effective and groundbreaking drug options to the hands of patients. This convergence has the ability to forge a new path in the ways innovations are encouraged and cures are secured.

	Cancer research is sharpening the focus on how the industry may succeed in solving complex health problems, but requires that the clinical trial process must be changed. Several factors are coming to light that will color the way scientists should do research. Cancer treatment is the prototype for the new research model, as oncology gets most of today’s research investment. According to the IMS Institute for Healthcare Informatics, cancer remains the biggest portion of the overall drug development pipeline in the earlier phases with four times the number of drugs in the pipeline than the next largest therapeutic class.

	While cancer gets the most investment, fewer cancer drugs are progressing to Phase II and III trials, which indicates both the high levels of early-phase activity and the difficulties in generating successful results in the clinic, according to IMS. About 80% of cancer pipeline drugs are potentially first-in-class treatments and a portion are the first of their kind, according to the PhRMA Report on Medicines in Development. This means potential treatment options are becoming available and fewer of them are succeeding initially.

	There are currently more investments and products in the funnel, resulting in oncology treatments that are new and novel. The issue is that there is not enough recent or real-time information available. Competition for patients is ever present, and attention and collaboration need to be up front in the research cycle to share learning for the benefit of the patient.

	A prime example of this convergence is the Cancer Moonshot. Two advances are required to break gravity for this initiative. Both are directly related to collaboration. The first is organizational, removing the space between clinical practice and research. This involves agent availability and patient matching. The old paradigm of phased studies and sponsor-lead trials is being outpaced by the urgency of research and the availability of new potential cures, particularly with immunology and oncology. Now, there are multiple agents engaging multiple targets at each of those steps. Some agents accelerate the immune system (pressing on the gas) while others interfere with a tumor’s ability to evade the immune system (releasing the brake). There are a multitude of hypotheses on not only how best to combine mechanisms, but also how to sequence them.

	Allowing clinical research to move at the most efficient pace possible will require skilled researchers to have open access to all of the agents and the data, with current, evidence-based decision support. In combination, this will allow the best patient enrollment decision-making based on available evidence. Review risks and results as a collaborative team and communicate the evidence as quickly and as clearly as possible, without bias of commercial competition or who gets credit. The body of knowledge grows exponentially with clinical research and patient care.

Improve Results with Fully Collaborative Research

	The traditional research model results in researchers working with one or two potential treatments, competing for patients and enrolling them in studies based on inclusion/exclusion criteria defined by the understanding of those products. A fully collaborative model allows researchers to understand all treatment options and have real-time data concerning patient profiles and outcomes, as the data is obtained. Screening, inclusion and enrollment decision support is tweaked with the constant receipt of new data, successes and failures. Enrolling patients in different treatments will be based on the most recent data across patients and treatment options, without limitations or sole focus on a few specific therapies. The commercial rewards can be addressed away from the Launchpad, based on outcomes as the picture of success emerges.

	The timeline for sharing research results and presenting interpretation of the results is accelerated, as research plays out, not as it concludes. Again, the resistance to this earlier and open sharing is prevalent in the existing commercial model, where this information is shared later in the research cycle to avoid early exposure of results of product testing and allow time for competitive marketing. In some cases, sharing results following closure of the study is simply the traditional model for research where conclusions are made in summary. In the new world of the Cancer Moonshot, earlier sharing of results data is allowing everyone to see value in each therapy and either increase or decrease investment accordingly. This early sharing of data is also allowing therapies to be redirected to other targets, different cancers or chronic diseases.

	The second revolution is a technical infrastructure that will allow identification of patients, enrollment and exchange of clinical data in collaboration. Implementing this infrastructure is no longer an innovation as much as it is using technologies that are already available to be used globally in broad collaboration. Patient registries, study enrollment criteria, site locations and potential treatment options all need to come together in a collaboration platform to make cancer research reach its fullest potential for positive outcomes.

	Genomic databases currently exist for both patients and tumors as well. Individually, these data and tools exist and are accessible via the internet, fragmented in silos of commercial and public research. A concerted effort must be made to integrate solutions and aggregate data. Incentives can be drawn up to share data which leaves important players out of the equation.

	The National Cancer Institute’s (NCI) Genomic Data Commons (GDC) (

) is a significant step in this direction. NCI’s Center for Cancer Genomics (CCG) maintains the GDC, which requires contribution of genomic sequence data to the databank where the research is funded by the NCI and includes genotyping of patients and/or tumors. This data is anonymized to protect privacy of patients. It also holds a substantial amount of information on demographics, race and physiology which can be used to analyze disease and health patterns.

	The 21st Century Cures Act, signed into law in December, increases the requirement for collaboration while broadening the type of data brought into the mix. The Cures Act includes a number of investments and measures, and one that is significant is the requirement for inclusion in patient reported outcomes in analysis of research submitted to the FDA for review. The emphasis of “patient-focused drug development” in the Cures Act increases the expectancy of inclusion of patient perspectives and data in regulatory review and proof of the treatment outcomes. This expectation requires more open collaboration with patients and technologies used in studies to collect patient-contributed data.

	Traditionally, much of this patient-provided or collected data has been used in post-market studies to further understand the drug and continue to market it competitively against other options. The trend in recent years has been to consider inclusion of this data in analysis of drug outcomes. It is now becoming expected. Again, this data collection and analysis gets moved forward in the lifecycle, adding to the scope of both process and technology that will change research design and execution.

	The biggest challenge to the future of immunotherapies and treatment of complex diseases is collaboration. Vice President Biden emphasized this in his ‘Cancer Moonshot’ talk at ASCO where he highlighted the need for Big Data, academic collaboration, and worldwide sharing of discoveries as early as possible. Ultimately, the biopharma industry and researchers will need to exercise a level of collaboration that has never been seen but is entirely possible through the use of technology and global cooperation between healthcare product companies, researchers, clinicians and patients.
	
	On the 70th anniversary of the first published, randomized clinical trial for tuberculosis, the global research community now has substantial experience and technology available to build new models for research. Collaboration across all the participants can resolve many of the issues each stakeholder experiences, to include larger-than-necessary gambles with research investments, access to new products and intelligent matching of specific patient criteria to the most likely successful therapy in treating life-threatening diseases.
	
	The future research model will benefit all participants. The value and potential for new therapies are more quickly understood. Investments can be directed to those with the most promise and reduced where the therapy struggles to show value against a specific target. This allows product companies to get a higher return on investment. Patients and doctors have more immediate access to a broader range of options with access to data and tools that help them pair specific patients to therapies that are most likely to succeed for a given situation. Process and collaboration changes that accelerate cancer treatment can be applied to other diseases, as can the therapies themselves. Regulatory agencies can monitor the progress of research as it progresses and make decisions in the best interest of patients as the data shows a product can address serious health issues.
	
	

Mark joined Halloran Consulting Group in 2015 with over 25 years of experience delivering technology, consulting services, and software in the life sciences industry. He has led system implementations for Clinical Trials Management Systems (CTMS), Electronic Data Capture systems (EDC), safety systems, data warehouses, content management systems, and analytics and reporting platforms.

Prior to joining Halloran, Mark was Vice President of Infosario Customer Engagement at Quintiles, Inc., where he founded and was responsible for the professional services organization leading technology integration and services delivery. He has held similar roles at CrossPhase Consulting, Galt Associates, and First Consulting Group. Mark also served as the product manager for CRIX International, a consortium of five pharmaceutical companies, the National Cancer Institute, and FDA with a mission to develop and service a shared platform for registration of physicians conducting clinical studies.

Mark holds a Bachelor of Science from Davis and Elkins College in Elkins, West Virginia. He is also a candidate for a MS in Health Information Management Systems from East Carolina.

		IMS Health Study: Global Market for Cancer Treatments Grows to $107 Billion in 2015, Fueled by Record Level of Innovation, QuintilesIMS, 2 June 2016. Summary available on-line at 

http://www.imshealth.com/en/about-us/news/ims-health-study-global-market-for-cancer-treatments-grows-to-107-billion-in-2015-fueled-by-record-level-of-innovation

		Pharmaceutical Research and Manufacturers of America. Medicines in Development for Cancer: From Hope to Cure. 2015. Report. http://phrma.org/sites/default/files/pdf/oncology-report-2015.pdf. Accessed May 15, 2016. Regulatory Explainer: 21st Century Cures Redux and What it Will Mean for FDA - See more at: 

http://www.raps.org/Regulatory-Focus/News/2016/11/28/26242/Regulatory-Explainer-21st-Century-Cures-Redux-and-What-it-Will-Mean-for-FDA/#sthash.i3Owe7sP.dpuf

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New technologies have accelerated the speed and effectiveness of clinical research and trials. This convergence is making it possible for new innovations and possibilities to bring groundbreaking treatment options to patients.

Mark Vermette

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