CER Methods Workgroup monthly call agenda
February 2nd, 2012, 3-4pm EST.
Tunis, S., ( Co-Lead, CMTP) Filart, R., NCATS (NIH Coordinator); Fair, A., (PM C4); Brixner, D., (Utah); Carey, T., (
UNC-Chapel Hill); Collier , E., ( NIH); Concannon, T., (Tufts); Fang, H., (U Mass Med); Hayward, A., (NIH); Harrell,F.,
(Vanderbilt); Hlatky, M., (Stanford); Neumann, P., (Tufts); Ohlssen, P., (Novartis); Shapiro, M., (UCLA); Yu, C,. (
New CER KFC Methods WG Co-Leads
Peter Neumann from Tufts and Jerry Krishnan from University of Illinois-Chicago were introduced as the new
WG Co-leads joining Sean Tunis and Mark Helfand
Presentation: Overview of the Bayesian DIA Group and CER Sub team by David Ohlssen, Novartis
The Bayesian DIA group was formed last year and consists of 40 people comprised of industry, regulators and
The aims of the group are to bring interaction to certain areas were Bayesian statistics might be helpful ( i.e, CER,
safety assessment, joint modeling, program-wide decision making )
Nine individuals in the CER subteam for the Bayesian group, others are welcome to participate. The aim of this
subteam is to look at what Bayesian statistics can be useful in CER. Focus of are Bayesian statistical techniques,
use of joint modeling of benefit risk assessment.
There is a DIA CER group that runs in parallel with the DIA Bayesian group, this group is new and may also be of
interest to the CER KFC Methods WG. The objective of the DIA CER group is to run training and conference
Bayesian evidence synthesis techniques is a main focus of the DIA Bayesian group, with particular focus on
medical product development and how it may be used in CER and also a niche focus on Bayesian evidence
synthesis techniques in practice and engage with other people who will influence policy and decision makers.
Second topic that the DIA Bayesian group is focusing on is use of joint modeling for benefit risk assessment; the
objective is to look at how joint modeling for benefit risk assessment should be used in practice.
Safety-meta-analyses links with both the DIA Bayesian and DIA CER sub-teams, the emphasis is on Bayesian
techniques in safety-meta analysis will be used with evidence synthesis techniques. The FDA is writing the
guidance in this area.
External activities in the DIA Bayesian group, two conference upcoming one with the FDA industry workshop
and the other at the Joint Statistical meeting, on the FDA/ Johns Hopkins applied partnership in comparative
effectiveness ( PACES) initiative.
The interaction with CER KFC Methods WG and the DIA Bayesian group is the first interaction documented
between the DIA group and the CTSA.
Chang Yu offered that within the CTSA there is a need for the ability to determine who is using similar CER
strategies, particularly for the clinicians to compare treatment strategies beyond the traditional randomized clinical
Chang Yu noted that work needed to be done to synthesize the work in CER for the CTSAs clinical
colleagues/health care decision makers in order to answer some of the questions.
Once the questions have been posted by the decision-makers , tools using data analysis to answer
the questions need to be employed , the DIA Bayesian group is developing some of the tools to
answer the clinical questions.
Chang Yu recommended that the CER KFC Methods BERD KFC groups and the DIA Bayesian group continue to
interact together within the CTSA.
Sean Tunis inquired about Bayesian approaches to evidence synthesis, what is in the DIA Bayesian domain,
analyzing prospective clinical studies? Is it prospective data analysis or using pre-specified RCT data for
secondary analysis? Analyses could be analysis of existing data but pre-specifying it in a new trial for secondary
Update on CB-PCTi: Presented by Sean Tunis, CMTP
Develop concrete steps regarding this objective and continue to learn what other groups have going on in specific
therapeutic areas and research networks.
Need to sort out useful domains of people that want to contribute to CB-PCTi project and create a core group
beyond the Co-leads to invest time and energy in the area of CB-PCTi.
The three objectives of the CB-PCTi core group would be to
Establish a robust, efficient national infrastructure for the conduct of community-based pragmatic clinical
trials building on the CTSA consortium platform and sustain this through a new public private
Identify, prioritize and coordinate implementation of a portfolio of activities within and outside the
consortium that will establish and maintain the CB-PCTi.
Develop a public-private partnership to sustain long-term efforts.
This project which is now an aspirational/mega concept that needs to be carved into actionable subcomponents to
improve clinical trials infrastructure nationally. There has been discussion on using ready to use capacity to do
community/practice/primary care based real world trials.
The RFA on Pragmatics Clinical Trials from NHLBI in CVD and pulmonary disease is just one example of how
real world CB-PCTi will be used as a CER tool.
The Helfand et al(2011) paper was referred to as a source to highlight the 3 critical needs/priorities of conducting
A CB-PCTi will allow the CER KFC Methods WG to improve methods for answering questions
about CER research using systematic reviews, public involvement and value of information to
determine research agendas.
CB-PCTi will form a sustainable platform for improving the design, conduct and analysis of
clinical research studies with a diversity of patients.
The CB-PCTi will create a bi-directional flow of information, rapid dissemination and sharing of
best practices to improve decision-making and linking the practice to the CER agenda.
Chang Yu referred to the NEJM commentary article by Jim Weir on pragmatic trials that was conducted in
England on Asthma and the need for more CB-PCTi trials. There is evidence that the level of interest in CB-PCTi
is rising, however, there are a lot of barriers to getting the CB-PCTi done in the US, particularly combating
statistical analysis issues. The challenges do not just stop with conducting CB-PCTis but also in statistical analysis
Several workgroups in the IOM Forum on Drug Discovery and the FDA commissioner’s office are interested in
community based PCAST.
A report from PCAST (the President’s council on Science and Technology) which is
doing a report on drug development. One of the findings on this report is the need for
better access to infrastructure for phase 3, 4, and 4b clinical trials.
Several groups have projects underway, DARTNet, MUSCLE ( Ken Saag at UAB), HMORN, CONCERT ( Jerry
Krishnan, COPD project under CONCERT)
In all four of these projects, people need to have in-kind resources to get projects funded in order to develop the
infrastructure. This is a major challenge for core support to sustain the infrastructure.
Sean posed a question to the group, Does a locus of strategic partnerships need to be made to better sustain the
infrastructure over time?
Tom Concannon suggested incubating infrastructures between investigators and ICs using the questions SGC4 is
working with to link up with I/Cs and become a priority with SGC4.
The incubation above would be illustrated by noting how investigator actions lead to the development proposal or
Sean Tunis, discussed the bottom up approach where investigators projects and activities at the CER KFC
Methods WG level can be the forum exchanging on lessons learned, best practices to move the CB-PCTi imitative
Sean noted the market demand for the ready to go CB-PCTi infrastructure and that is plausible based on the UK
turning their entire national health services into a clinical research network. There is a report on how to do this
and centralize efforts. On a national level the UK has gone a long way to have an infrastructure to do high level
clinical trials at a lower cost.
Sean inquired if the CER KFC Methods WG is the right forum for this initiative.
Elaine Collier suggested that members look at two FOAs on Health Care Systems, Research Collaboratory : 1)
Coordinating Center FOA RFA-RM-11-021 and the 2) FOA for Pragmatic Clinical Trials Demonstration Projects
RFA-RM-12-002. The foci of this infrastructure are to bring together the methods, policies related to IRBs, data
collections, defining patient phenotypes from EMRs and to develop a resource for information. The NIH is trying
to address how to get the research done in real health care settings that needs to be done.
Sean Tunis suggested doing things in a complimentary fashion leveraging relationships that exist in the CTSA
towards this objective and go toward the national infrastructure more efficiently than funding individual
investigators for smaller scale proof-of-concept projects.
Frank Harrell noted there is not a lot of CTSA research going on related to CB-PCTi, there are not many pragmatic
trials going on or RCTs in community settings. This is not to say that the CTSA platform cannot be moved in the
direction of supporting CB-PCTis.
Sean Tunis requested that members review the Next Steps slide and contact him at email@example.com if they
are interested in the following next steps objectives 1) Establishing a planning committee ( less than 20 people) to
draft a CB-PCTi startup plan and plan a F2F methods WG sponsored workshop for summer 2012, 2) Establish a
steering committee to finalize workplan, time and task assignments for the summer 2012 F2F and 3) Be engaged
in the Workshop in some capacity.
# Action Item Responsible Party Due Date
Investigate piloting a public shared space where 18 or so Methods WG Before
Alecia Fair, Rosemarie
1 members can enter their strengths/skills/experience in Methods for use by March 22,
the Methods WG 2012
Outreach to collaborators and Inventory of PBRN colleagues in the
consortium to add to the Methods WG existing list: Phil
Sean Tunis, Jeanne-Marie
2 Greenland,(SGC4); Wilson Pace (Co-Lead, CE PBRN), Paul Meissner, Ongoing
(Co-Lead CER DIR) Kurt Stangey (DartNet), Ken Saag (UAB), Dan Ford
Continue to follow-up with David Ohlssen, DIA Bayesian Analysis group
3 ALL Ongoing
at Novartis on reminder of scope of work for this WG
CER KFC Methods WG members are encouraged to contact Sean Tunis
firstname.lastname@example.org for their a) affirmative indication and interest
4 to continue the CB-PCTi discussion, b)interest to be on the CB-PCTi ALL Ongoing
planning committee c)interest in participating in the F2F workshop in
summer of 2012 and in what specific issues to discuss
CER KFC Methods Before
Have a follow-up in a one-off discussion of the SGC4 priorities for the
5 Leadership, Rosemarie March 22nd,
Filart and Tom Concannon 2012
Enlist support of the PBRN group at UNC-Chapel Hill for future Methods
6 Tim Carey February
WG project on Pragmatic Clinical Trials
Next Meeting Date & Time: Thursday February 23, 2012, from 12:00 PM -- 1:00 PM ET
Recurring Meeting Date & Time: 4th Thursday of every month from 12:00 PM -- 1:00 PM ET