11/8/2013 – IN THIS ISSUE:  Tune in to Tunis; EGD on Molecular Diagnostics in Oncology; Green Park Collaborative Update; Upcoming Presentations; New Staff & Promotions; Recent Publications

Tune into Tunis

As CMTP gears up to host the first Green Park Collaborative-USA (GPC-USA) Annual Meeting, we continue to think more deeply and strategically about our Effectiveness Guidance Documents (EGDs), one of the main products of the GPC-USA and its developing consortia. During the last several years, CMTP has produced a variety of EGDs, most recently on Patient Reported Outcomes in Cancer Drug Trials and the Clinical Utility of Molecular Diagnostics, both focused on oncology. These publications are the result of an intensive and highly collaborative process. They guide researchers and product developers about how to develop clinical studies to produce the evidence decision-makers—payers, guideline developers, clinicians, and patients—need to make educated health care decisions.

CMTP released the EGD on Molecular Diagnostics in Oncology last May at the GPC-USA’s annual kickoff, and it has received a good reception. Several industry publications and blogs have reviewed the work positively. We have been invited to present our findings at professional meetings, including the 2013 American Society for Clinical Oncology (ASCO) Breast Cancer symposium. The 75-page document has been downloaded more than 300 times. Importantly, others are talking about the work as well. At the recent Evidence-Based Reimbursement Summit in Bethesda, MD, for example, a leader from the Medicare carrier Palmetto GBA noted that the EGD is serving “in essence like a FDA guidance document,” helping to inform for Palmetto’s coverage decisions around molecular diagnostics programs.

GPC-USA’s new consortia in oncology and endocrine-metabolic diseases have recently selected topics for their first EGDs. We look forward to working with our community of stakeholders not only to develop the best possible guidance, but to create products that are hugely influential, that are considered and used by a wide variety of researchers, product developers, payers, and others as we all seek to create better evidence, better decisions and better health.

News of Note

CMTP recently received two awards through new grants made by the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors. CMTP will be part of a $9 million contract awarded to a Harvard Group Pilgrim Health Care Institute-led consortium to fund the first National Patient-Centered Research Network, which will improve the country’s capacity to conduct comparative effectiveness research (CER). We are also part of a $1 million grant to the Fred Hutchinson Cancer Research Center for “A Structured Approach to Prioritizing Cancer Research Using Stakeholders and Value of Information.” Read More

Green Park Collaborative

The Green Park Collaborative-USA (GPC-USA) has built impressive momentum this Fall, moving ahead on several fronts:

• On September 19, the Collaborative’s Oncology Consortium met for the first time in Baltimore, Maryland. After substantive presentations and considerable discussion, the group chose Treatment sequencing and prioritization as its Year 1 topic priority. The consortium chose Next Generation Sequencing as an alternate/second topic for Year 1 or for Year 2.
• On October 23, GPC-USA’s Endocrine-Metabolic Diseases Consortium gathered, also in Baltimore. Project lead C. Daniel Mullins, Professor at University of Maryland School of Pharmacy, detailed the EGD development process, and presented a summary of findings from key informant interviews. Draft recommendations for the EGD, based on literature reviews, key informant interviews, and initial the technical working group’s initial feedback, were presented to and discussed by the consortium members. The EGD is slated to be released in April 2014.
• The Collaborative will hold its Annual Meeting on November 12 at the World Trade Center in Baltimore, MD. Representatives from federal agencies including CMS, FDA, PCORI, NIH, and AHRQ; patient advocacy groups; private payers; life sciences companies; professional societies; and researchers are expected to attend. The meeting will provide participants with an overview of GPC-USA accomplishments to date, and the opportunity shape and drive current and future GPC-USA initiatives.

For more information or if you are interested in becoming more involved with the GPC-USA, please contact Corinne Warren.

On the Podium: Coming Soon

• CEO Sean Tunis, MD, will present, “Health Economics and the Medical Devices: The New Normal,” November 6 at the 16th Annual Coverage, Reimbursement, and Health Policy Conference, in Linthicum, MD.
• Research Director Donna Messener, PhD, will speak on “Electronic Informed Consent: Notes from an Ongoing Pilot Study of an iPad-based Consent Tool” at The Public Responsibility in Medicine and Research (PRIM&R) 2013 Advancing Ethical Research Conference, November 9 in Boston.

Staff Updates

• Rachael Moloney has been promoted from Senior Research Associate to Research Manager. In her more than two years at CMTP, Rachael has played a critical role on many CMTP projects including the NIH Health Care Systems Research Collaboratory.
• Nora Osowski has been named the new Consumer and Patient Alliance Coordinator at CMTP. She will take on the new responsibility of managing our Patient and Consumer Alliance Council in addition to her role as Research Manager.
• CMTP welcomes Sandra Hwang as our newest Research Assistant. Sandra is currently a student in the Masters of Science in Public Health program at Johns Hopkins Bloomberg School of Public Health. She holds a Bachelors of Science in Biology and Society, Public Health and Policy from Cornell University.

In Print

You can find some of the latest thinking CMTP leaders and staff in a variety of recent publications.

• Gliklich, R., Tunis, S. (2103). Integrating large pragmatic trials into payer decision making. On Drugs.Com.
• Faden, R., Kass, N., Whicher, D, Stewart, W, Tunis, S. (2013). Ethics and informed consent for comparative effectiveness research with prospective electronic clinical data. Medical Care, 51, S53-S57. doi: 10.1097/MLR.0b013e31829b1e4b.
• Hayes, D., Allen, J., Compton, C., Gustavsen, G., Leonard, D., McCormack, Newcomer, L., Pothier, K., Ransohoff, R. L. Schilsky, R., Sigal, E., Taube, S., Tunis, S. (2013). Breaking a Vicious Cycle. Sci. Transl. Med. 5, 196cm6.
• Sonnad, S., Mullins, C.D., Whicher D., Goldsack, J., Mohr, P., Tunis, S. (November 2013). Recommendations for the design of Phase 3 pharmaceutical trials that are more informative for patients, clinicians, and payers Contemp. Cl. Trials, 36:2, 356–36.
• Garrison Jr., L., Towse, A., Briggs, A., de Pouvourville, G., Grueger, J., Mohr, L., J.L., Severens, J.L., Siviero, P., Sleeper, M. (July 2013). Performance-Based Risk-Sharing Arrangements: Good Practices for Design, Implementation, and Evaluation: Report of the ISPOR Good Practices for Performance-Based Risk-Sharing Arrangements Task Force Value in Health: The Journal of the International Society for Pharmacoeconomics and Outcomes Research 6:5 703-719.
• Tunis, S., Messner, D. Medicare Policy on Bariatric Surgery: Decision Making in the Face of Uncertainty (2013). JAMA 310(13):1339-1340. doi:10.1001/jama.2013.278849.
• Sonnad, S., Goldsack, J., Mohr, P., Tunis, S. (2013). Methodological recommendations for comparative research on the treatment of chronic wounds http://www.cbi.nlm.nih.gov/pubmed/24005781 J Wound Care 22(9):470-80.

7/16/13 – IN THIS ISSUE:  Tune in to Tunis; GPC-USA Kicks Off; Events & Speaking Engagements; EGD for MDx, Promotions & Introductions; We’re Hiring

Tune into Tunis

Balancing the needs of various health care stakeholders is central to much of CMTP’s work. An example of this was the focus of a recent discussion on BioCentury Tv, in which I appeared along with Dr. Joel Kupersmith, Chief Research and Development Officer of the Veterans Health Administration, and Dr. Claudia Grossman, Senior Program Officer at the Institute of Medicine’s Roundtable on Value and Science-Driven Health Care (You can see the whole segment here.).

The discussion centered on the tension between our need to protect patients involved with medical research with the broader interest in creating a “learning health care system.” Traditionally, rules and procedures for human subjects protection draw a bright line between routine medical care and scientific inquiry. This protects patients as they enter into potentially risky research on new medications and technologies and ensures they are treated ethically.

In a learning health care system, however, the notion is that we also learn as we go, that we seek quality improvements in the care that is already being delivered. This suggests a significant grey area, as we try to glean new insights from current practices: Is it care or is it research, or something in between?. For example, is it better to take an approved blood pressure medication in the morning or before bed? Patients receive the drug both ways currently, and there are no studies that clearly address this question at present. The answer could significantly improve how effectively the medication controls patients’ hypertension.

There are literally thousands of these kinds of questions waiting to be answered throughout the health care system, but our ability to examine them depend on a kind of practical balance. We need streamlined methods of informed consent so patients in each case understand what they are getting into, but at the same time reduce the barriers to this kind of low risk (and potentially high reward) clinical research. This can help us build the new knowledge that systems need to constantly improve the health care we provide.

CMTP is working on this balance. In partnership with the Johns Hopkins Berman Institute of Bioethics, we are reviewing the effectiveness of various models of informed consent, patient attitude and opinion on each of these consent models, and patient benefits versus risks when involved in practice-based research (See The Research-Treatment Distinction: A Problematic Approach for Determining Which Activities Should Have Ethical Oversight and An Ethics Framework for a Learning Health Care System: A Departure from Traditional Research Ethics and Clinical Ethics). The project is scheduled to conclude late summer 2014, and we hope to have additional useful insights to share at that time.

Sincerely,

Sean Tunis, MD, MSc

News of Note

GPC-USA Kicks Off

CMTP launched the Green Park Collaborative – USA (GPC-USA) at an inaugural meeting in Baltimore, MD, May 1. GPC-USA is a multi-stakeholder forum that develops condition and technology-specific study design recommendations to guide the generation of evidence needed to inform both clinical as well as coverage and payment decisions in the United States. In 2013, this group will focus on methodological standards for clinical development and market access in oncology and diabetes.
The kick off meeting included more than 50 participants from government organizations such as Center for Medicare and Medicaid Services and the Patient Centered Outcomes Research Institute (PCORI); patient organizations such as Friends of Cancer Research, National Health Council, and Friends of the World Health Federation Foundation; payer organizations; and life sciences companies. You can review presentations from the meeting here.

CMTP, Quintiles Team Up to Improve Uterine Fibroid Care

Penny Mohr, MA, Senior Vice President for Program Development, will be supporting Quintiles, the world’s largest provider of biopharmaceutical development and commercial outsourcing services, in a PCORI-funded research study. This work will help patients and the clinical community make better-informed decisions about treatments for uterine fibroids, a common condition affecting women of childbearing age. The study is part of a portfolio of patient-centered research that addresses PCORI’s national research priorities. Read the press release here.

Events, Speaking Engagements

Last week, Sean Tunis presented at the NIH Health Care Systems (HCS) Research Collaboratory Grand Rounds. He discussed highlights of the recent HCS Research Collaboratory Stakeholder Advisory Group meeting. The webinar will be available online at www.nihcollaboratory.org.

CMTP recently worked with the PCORI Methodology Committee and AcademyHealth to convene a mini-course on the PCORI Methodology Standards, as a part of AcademyHealth’s Annual Research Meeting. This mini-course focused on ways researchers in a variety of environments, such as academia and industry, may best utilize PCORI’s methodological standards to improve the relevance, rigor, feasibility, and consistency of patient-centered outcomes research studies. The webinar version of the course is available online here.

On the Podium: Coming Soon

Sean Tunis will be speaking at the Fifth National Comparative Effectiveness Summit, which takes place September 16 – 18, 2013 in Washington, DC. Dr. Tunis will speak on The Role of Pragmatic Trials as a Bridge between Experimental Studies and the Real World as part of the plenary session entitled Real-World Evidence and Decision-Making: Is It Time Yet? Additional details are available here.

On September 27th and 28th, Dr. Tunis will speak at the Second David Sackett Symposium, in Niagra Falls, Ontario. For additional information, please click here. To register, please click here.

Dr. Tunis will travel to London on October 9 to speak on clinical effectiveness research in the USA and lessons learned from international experience at the National Institute on Health Research Health Technology Assessment (HTA) Conference. The event is celebrating 20 years of the HTA Program and the contribution it has made to NIHR’s mission to improve health and wealth of Great Britain. For more information, click here.

In Print

CMTP recently issued a new Effectiveness Guidance Document (EGD) for molecular diagnostics tests in oncology that provides specific study design recommendations for researchers and test developers to evaluate the impact of their tests on patient health outcomes. Download a copy here.

At CMTP

During the last few months, CMTP has welcomed three new staff:

• Chris Kamphaus is the new Manager of Accounting and Finance at CMTP. He has previously worked as an accountant at JPB Partners and RS&F, as well as a Management Associate at Synergy Real Estate. Chris holds a Bachelor of Arts in Economics and a Master of Science in Accounting from Rhodes College.
•  Jennifer Al Nabar joins CMTP as a Senior Research Associate. She is currently supporting a variety of initiatives including the NIH Collaboratory, a PCORI-funded project examining uterine fibroid treatments, and two methods and data infrastructure related proposals. Ms. Al Naber graduated with dual degrees in psychology and sociology from UMBC and received a Master of Science in Counseling Psychology with a concentration in Research from Loyola University Maryland. She is currently pursuing a degree in Health Policy and a certificate in Public Health Informatics from the Johns Hopkins Bloomberg School of Public Health.
• Srijana Rajbhandary is a new Research Associate at CMTP. She will be working with CMTP’s Green Park Collaborative- USA and supporting the FDA Partnership in Applied Comparative Effectiveness Sciences (PACES) project. Ms. Rajbhandary graduated with Master of Public Health (MPH) degree from Rollins School of Public Health, Emory University. She also holds a Bachelor of Dental surgery degree from B.P. Koirala Institute of Health Sciences, Nepal.

And congratulations to Janelle King, who has been promoted from Administrative Assistant to Executive Assistant. Janelle has provided superior support to CMTP executives and project staff for nearly two years. Congratulations, Janelle, and thank you for all of your hard work!

We’re Hiring

CMTP is looking for a Research Manager and Research Director who shares our interest in making health care more effective and affordable. Click here for position descriptions and other opportunities at CMTP.

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4/1/13 – IN THIS ISSUE – Tune In to Tunis; Coming Soon: Green Park Collaborative – USA; In Print:  Hastings Center Report and Genetics in Medicine; On the Podium:  Post Approval Summit; At CMTP: Promotions and Introductions; We’re Hiring!

Tune In to Tunis

Welcome to exCERpts, CMTP’s new e-newsletter. In the months ahead, we look forward to giving you a sense of our continuing efforts to make health care more effective and affordable by improving the quality, relevance, and efficiency of clinical research. As you know, CMTP is committed to collaborating with payers and policymakers, life science companies, clinicians and patients to do this work— developing and publishing research standards and other guidance, improving the processes and products critical to clinical research and facilitating consensus around high priority reimbursement and other policies. We hope this newsletter helps keep you up to date.

Recently, we completed a review of our project and broader communications, interviewing people in and around CMTP’s various endeavors and assessing the outreach efforts of more than a dozen peer organizations. We learned that while colleagues may be deeply engaged in a particular CMTP initiative, they rarely hear about other aspects of our work. And while these colleagues are interested in getting more of this information, what they said they really value is our point of view, our unique perspective on what is happening in comparative effectiveness research. As one informant put it, “Don’t just tell me what CMTP is doing; tell me why it matters, in the context of policy, research, and care delivery.”

So that is what we will try to do, not only with this quarterly newsletter, but in our communications with our various networks more broadly. And while we will give you our viewpoint, we also want to hear yours. We hope our perspective prompts further exchange, lending to valuable, actionable insights. Only together, by listening to each other and integrating different approaches, can we move this critically important work forward. We look forward to more conversation.

Sincerely,

Sean Tunis, MD, MSc

Coming Soon

Later this Spring, CMTP will officially kick off the new Green Park Collaborative – USA (GPC-USA), a multi-stakeholder forum that will develop condition-specific study design recommendations to inform coverage and payment decisions in the United States. The GPC-USA will provide a neutral forum within which payers, life sciences companies, patients, clinicians, researchers, regulators and other stakeholders can engage in dialogue regarding methodological standards for studies intended to demonstrate real world effectiveness and value.

The main products of the GPC-USA will be Effectiveness Guidance Documents (EGDs). These present specific recommendations for the design of prospective clinical research that reflects the evidence expectations of payers, informed by the perspectives of patients, clinicians and other decision-makers.

The EGDs developed through this multi-stakeholder collaborative process will provide well-defined study design “benchmarks” that may be useful to:

• Payers making coverage and pricing decisions;
• Medical professional societies developing clinical guidelines;
• Research funding agencies evaluating grant proposals; and
• Organizations producing educational material for patients and consumers.

In 2013, the work of the GPC-USA will focus on methodological standards for clinical development and market access in oncology and diabetes. CMTP will host the first GPC-USA meeting on May 1 in Baltimore, Maryland.

For more information about GPC-USA, including Membership fees and details about the kick-off meeting, please click here and contact Corinne Warren.

In Print

Issues in ethical oversight
While research today is increasingly integrated into healthcare delivery, the kind and extent of ethical oversight required is generally based on whether a project is considered research or practice. Earlier this year, Sean Tunis, MD, MSc, and others published two papers in the Hastings Center Report—one exploring the implications of the current regulatory environment and a second, proposing a new framework where ethical oversight is instead determined, not by the type of work, but rather by possible patient risk and burden. Abstracts to both papers can be found here.

Citations
Kass, N. E., Faden, R. R., Goodman, S. N., Pronovost, P., Tunis, S. and Beauchamp, T. L. (2013), The Research-Treatment Distinction: A Problematic Approach for Determining Which Activities Should Have Ethical Oversight. Hastings Center Report, 43: S4–S15. doi: 10.1002/hast.133

Faden, R. R., Kass, N. E., Goodman, S. N., Pronovost, P., Tunis, S. and Beauchamp, T. L. (2013), An Ethics Framework for a Learning Health Care System: A Departure from Traditional Research Ethics and Clinical Ethics. Hastings Center Report, 43: S16–S27. doi: 10.1002/hast.134

Stakeholders and Comparative Effectiveness Research
Comparative effectiveness research on genomic tests is critically important given the rapid pace of genomics innovation. Using an explicit priority-setting framework, Pat Deverka, MD, and others highlight key criteria that stakeholders should consider when prioritizing and deciding among many potential research questions and trial designs in this research area. An abstract for this paper can be found here.

Citations
Esmail, L.C., Roth, J., Rangarao, S., Josh, Carlson, J. , Thariani, R., Ramsey, S.D., Veenstra, D.L., and Deverka, P. (February 2013), Getting our priorities straight: a novel framework for stakeholder-informed prioritization of cancer genomics research. Genetics in Medicine, 15:115-122. doi:10.1038/gim.2012.103

On the Podium

Sean Tunis will have two roles at the May 7 Post Approval Summit, presenting on the “Shifting Role of Pragmatic Clinical Trials in Effectiveness Research and moderating the session: “Panel Presentations: The Need for New and Comprehensive Models for Evidence Development.” For more information about the Summit, please click here.

At CMTP

2013 has been a very exciting year thus far at CMTP. Tania Dutta, MS, MPP, who has worked at CMTP since 2011 as a Research Associate, has been promoted to Project Manager. In addition to her outstanding work for CMTP, Tania traveled to India for her wedding. We congratulate Tania on all of her successes.

CMTP welcomes Nora Osowski, MPH as Research Manager. Nora will be working on various qualitative research projects focused on stakeholder engagement in health policy and clinical management decision-making. Prior to her work with CMTP, Ms. Osowski spent six years at the Institute of Medicine of the National Academies where she contributed to numerous consensus reports for the Board on Population Health and Public Health Practice and the Board on Health Sciences Policy.

We’re Hiring

We are hiring a Senior Research Director, Methods Standards, to oversee of the development of comparative effectiveness research methods standards within GPC-USA, as well as a Senior Program Director to plan, develop, and direct all operations of the GPC-USA. Visit www.cmtpnet.org/careers for additional details and opportunities.

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