Objective
The Green Park Collaborative (GPC) is an international initiative that is exploring the scientific feasibility of developing guidance for the life science industry on the design of clinical studies to meet the needs of Health Technology Assessment (HTA) organizations and payers. The aim is to produce prototype “methods guidance” documents which will provide both therapeutic-area specific trial design recommendation and general methodological advice, and will be aligned to the extent possible with related regulatory guidance. The purpose of this guidance is to reduce the uncertainty currently faced by the life sciences industry regarding the evidentiary preferences of HTA groups and payers, to improve the relevance of clinical research, and to speed patient access to useful innovations. Prototype guidance documents produced by the GPC are not intended to represent consensus statements on the part of all participant organizations, but rather demonstrate the feasibility of and inform the subsequent development of guidance beyond the prototype phase.
Background
Over the past decade, there has been increasing recognition of the impact payers and health technology assessment bodies have on the development and adoption of drugs, devices and diagnostics. Historically, the clinical development process in the life sciences industry focused mainly on fulfilling regulatory requirements, with the expectation that positive reimbursement decisions would generally follow. Therefore, clinical studies have been primarily designed to address the information needs of regulators with less attention to generating evidence targeted to payers and the HTA bodies that support them. As a result, a number of important questions were inconsistently addressed: Is a new product better or safer than an existing product? Could benefits be achieved outside of an academic or research setting and sustained over time? What are the impacts on patient-reported outcomes and quality of life? Life sciences companies are now keenly aware that they must rethink their approach to the design of clinical studies to provide this missing information.
As it has become clear that more and different evidence is needed by payers and HTA bodies, these organizations have begun to recognize the importance of clearly communicating to trial designers the information that they expect to see in clinical trials. Improved communication of expectations may result in more efficient trials. Regulatory bodies have been providing such guidance to product developers for many years. In the absence of specific guidance from HTA organizations and payers, pre-approval trial designs often suffer from significant methodological limitations from the perspective of these groups. Recently, several payers and HTA bodies have recognized the need to provide “scientific advice” to product developers on trial design, including several pilot initiatives that provide joint advice from payers and regulators. These efforts have been constructive and well-received, and highlight the potential value of exploring other forms of methodological guidance from payers and HTA bodies to product developers, with particular emphasis on aligning this advice with and building upon guidance provided by regulators.
GPC Organization and Initial Activities
The GPC operates under the direction of a multi-disciplinary, international steering group of individuals with backgrounds in HTA, reimbursement, regulatory policy, patient engagement, clinical care, research design and drug/device development. The steering group is co-chaired by the Center for Medical Technology Policy (CMTP – represented by Sean Tunis, President and CEO) and Health Technology Assessment International (HTAi – represented by Berit Mørland, Past President). In this role, CMTP and HTAi will jointly convene and facilitate collaboration among GPC participants. The project’s initial emphasis is to develop a pilot methods guidance document that will provide recommendations for the design of clinical studies of pharmacologic therapies for Alzheimer’s disease (AD), building on the relevant regulatory guidance. In addition to assessing the scientific feasibility of creating such guidance, the development of the pilot guidance will inform deliberations about how HTA organizations and payers might sustain this work by expanding the effort to other therapeutic domains, and applying the approach to devices and diagnostics. The GPC will also begin exploratory work and a pilot project to develop general (i.e. non-disease-specific) methodological guidance on issues of trial design and evidence generation. It is hoped that the work of the GPC will provide useful experience and inputs into related ongoing and planned initiatives of HTA organizations, payers and regulators.
The methods guidance will be lead by a technical working group of international AD experts that will include researchers, payers, HTAs, and patient representatives. A Life Sciences Advisory Group (LSAG) will provide technical, methodological and logistical input to ensure applicability of guidance on drug development. The GPC steering group includes senior and expert staff from a range of leading payers and HTA bodies from around the world, and the GPC’s work will therefore be highly cognizant of real-world issues faced by such bodies. It is hoped that prototype guidance produced by the GPC will therefore usefully inform related work by these and other bodies.
GPC Funding
Support for the first meeting of the GPC steering group in March 2011 (near Green Park in London) was provided by the California Healthcare Foundation, Kaiser Foundation Community Benefit Program and the United Healthcare Foundation. Partial support for the development of the pilot guidance on AD has been secured from the National Pharmaceutical Council and United Healthcare, along with preliminary commitments of in-kind technical staff support from several national HTA organizations. Further funding is expected from supporters of the work of the Steering Group and the LSAG.
Progress and Next Steps
Since the initial meeting in March, CMTP, HTAi and GPC steering group members have had ongoing discussions to ensure that the work was optimally aligned with related initiatives involving scientific advice to product developers and efforts to increase dialogue between regulators and payers. At a GPC steering group meeting in Sydney Australia in October, 2011, CMTP and the Institute for Clinical and Economic Review presented background research that will support the development of draft guidance for AD, and discussed the process for completing the pilot guidance. Over the next several months, the GPC will convene a Life Sciences Advisory Group and a Technical Working Group to produce and refine that guidance.