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June 24, 1999 (historical)
Steering Group Meeting
Summary of General Sessions
The general sessions of the meeting were chaired by Dr. Stephen Katz.
General introductions of the 48 participants were made and an overview of the purpose of the meeting was presented. The NIH perspective was presented emphasizing that the osteoarthritis (OA) initiative being explored as a potential partnership with the private sector to serve a common goal of improving public health. Although there are many unresolved issues, NIH has common goals with those of other parties represented at the meeting. Substantial progress has been made since the last meeting of April 16.
OA presents a clear and growing public health need. There are currently no disease modifying pharmacologic interventions and developing new drugs for osteoarthritis is hampered by the lack of surrogate markers of disease activity. There are new technologies, particularly for imaging the joint space, bone, and related tissues even at the molecular level that may offer benefits to measure this disease. Driven by these technologies and opportunities for therapeutic development, this Steering Committee views this as an opportune time to identify populations at risk for OA and proceed to better characterize the disease and define parameters to measure treatment effect.
This meeting follows a series of meetings at the NIH, beginning with GlaxoWellcome in 1998, and subsequently six other companies in April 1998. Not all interested companies are at this meeting, but more will participate in an open meeting to be held in early 2000. A website has been established to keep all interested parties informed about progress: http://www.niams.nih.gov/Funding/Funded_Research/Osteoarthritis_Initiative/default.asp
In the next few months, a "white paper" will be prepared to summarize the current populations and cohorts of OA patients being followed both in the United States and internationally. Another document will be developed to summarize the statistical needs of the project - e.g., establishing cohort size, statistical power, etc.
The goal of this meeting is to discuss a framework for the winter meeting, and to identify those issues that need to be overcome to achieve a partnership. The initiative may be a single study, or a composite of several studies to address needed areas of biomarker research in OA. The focus of this meeting is to identify opportunities for collaborative research to move the goal forward. The winter meeting will identify those goals that can be undertaken in the short term. Milestones for this partnership will be set at short, mid-, and long-term.
Four subcommittees were created to identify issues, questions, and people to address them, including people in dissenting groups. The subcommittees then reported to the full group (see below). Each subcommittee had a chair and an NIH staff person (see agenda).
An overview of the activities of the NIH Office of Technology Transfer were presented and this office will continue to serve as a resource in developing this partnership. The NIH intramural research program has extensive experience with the private sector in patenting and licensing and the NIH has facilitated consortia in novel ways. It is possible and important to work out the plan for this collaboration as it may be the only feasible way to advance osteoarthritis research and encourage the development of therapeutic interventions.
Discussion of Draft Documents on Statement of Purpose
The question of how fundamental science would be incorporated into the collaboration was addressed. Clearly, whatever is encompassed by this initiative needs to stimulate and be fed by developments in basic science. The group acknowledged that this was an important point and remained an open question. The NIH expects that the scientific community as well as the pharmaceutical sector will have access to the resources developed by the initiative and that this will drive new developments.
It was noted in response to discussion that the goal of the initiative is to develop biomarkers and not to evaluate therapeutic interventions. The wisdom of targeting osteoarthritis and not other forms of arthritis was questioned and led to a discussion. There was consensus among the group that the focus of the initiative should be OA and not other bone and joint disorders. The group felt that osteoarthritis requires this big effort of overcome the lack of disease definition and to stimulate research on therapeutics.
Important questions were asked about the development of diagnostic assay systems from the resources developed by the OA initiative. The NIH position is that this should be encouraged by the partnership. Establishing the guidelines for how this is fostered and overseen will be in the domain of the administrative, management, and infrastructure discussions.
Other general issues addressed in the opening session included:
The goal of the Winter 2000 meeting was established as addressing the issues and answers that face the formation of the consortia. Shortly thereafter, each potential partner will need to make a decision about their participation in the collaboration. The issues will be raised at this meeting in the break-out sessions, but not answered until the Winter Meeting.
Can safety biomarkers also be developed from this initiative?. There was general agreement that in some cases, a marker may be reflective of adverse events that relate to bone, cartilage, and joint related tissues.
Funding the partnership was discussed. It was noted that this needed to discussed in depth and that various options should be explored.
Several participants indicated that there will likely be other useful outcomes of the collaboration including: developing metrics of performance in biomarkers that can be extrapolated to other diseases, serve as an attractant for new investigators to the field, and help to inform therapeutic development.
The point was made that many patients with OA are asymptomatic. This needs to be considered when developing recommendations for new cohorts.
Resources developed for this project may serve very important research functions in the future. Looking at this as a single study to accomplish one aim would be short-sighted.
The group will need to discuss the ethical and informed consent issues up front particularly if DNA specimens are collected in some cohorts
Closing Discussion Session.
The chairs from each of the 4 subcommittees (imaging,epidemiology/genetics/biostatistics, biochemical, and administration/infrastructure/management) reported back to the group. Detailed summaries from the committee presentations are presented separately on the website.
General discussions were held on many of the issues regarding the subcommittee presentations. The group raised a series of issues that will need to be addressed in the development of the collaboration.
Major discussion points included the following:
The design of the size and composition of the cohorts will need to determined by the consortium. Biostatistical input is needed for and will be summarized at the next meeting to aid in cohort designs.
Which joint (hip, knee, hand) to examine, particularly for imaging evaluations? Discussion on this will be presented at an upcoming FDA Arthritis advisory group meeting being held on July 21.
It may be that a large cohort would be the most practical and with subsets of the cohort devoted to different risk groups or different joints.
Ongoing studies of OA are useful, but all have weaknesses in clinical data or the samples collected. It was suggested that perhaps the patients in these studies could be used for baseline data and followed prospectively.
Standards for data and sample collection and storage need to be developed. These include: database acquisitions, structure for prospective collection, methodology for samples (storing and cataloguing), assay standards.
There was agreement that a broad view of what could be measured should be identified and then priorities be established. All of the subcommittees raised similar issues about defining cohorts to study, and this will be a major focus of the winter meeting.
A step-wise integration of the analytic studies to be conducted by the consortium was suggested. There was a strong sense that imaging of anatomic markers was ready for evaluation, while biochemical targets are less clear. The group agreed that collecting specimens to conduct future studies from well-defined cohorts was a very important priority.
A suggestion was presented for allowing different partners to be involved in different specific aspects of the project.
What constitutes being a partner? What is the role of academia in the partnership?
The group deemed it important to identify important intellectual property rights positions that will encourage and not establish rules that exclude academic investigators from participating fully.
It was recommended that for finite resources collected in the studies, an oversight committee and application system would be need to be in place; access to imaging information would also need to be discussed amongst the partners
There was general agreement that an administrative/management/infrastructure plan would be developed and shared with the group in organizing the winter meeting to discuss the plans for the consortium. In the meantime, NIH will continue to develop options for the formation of the consortium. In preparing for the winter meeting, it was agreed that each party involved in it will bear some responsibility for sponsoring speakers and additional expenses associated with the meeting. NIH will continue to serve as an information conduit to all interested parties.
The participants in this meeting and their affiliations
are listed with the Sub-committee Reports. Dr. Stephen Katz, NIAMS,
and Dr. Richard Hodes, NIA, participated in each of the break-out sessions,
addressing issues of the NIH perspective.