Integrate the needs of patients into federal Operational Plan for Comparative Effectiveness Research (CER)

July 28, 2009

Honorable Kathleen Sebelius
Secretary, Department of Health and Human Services
Hubert H. Humphrey Building
200 Independence Avenue, S.W.
Washington, D.C. 20201
Fax: 202-690-7203

Dear Secretary Sebelius:

Mental Health America is the nation’s oldest and largest advocacy organization concerned with all aspects of mental health. In fact, we are celebrating our Centennial this year. Given our longstanding interest in the public’s health, we are the founder of the National Working Group on Evidence-Based Health Care (the Working Group), which is a collaboration of approximately 40 patient and consumer organizations, professional societies, providers and other interested stakeholders who want to help shape the initiatives in evidence-based healthcare (EBH) in order to close the gap between our knowledge and ordinary practice.

Since 2005, we have worked to broaden participation of patient groups in all aspects of EBH to improve the usefulness of evidence for consumers and clinicians as well as to assure that the locus of decision making remains with the patient and his or her clinician. The Working Group believes that people with chronic conditions should have a greater voice and be empowered to provide policy recommendations on issues that will directly impact their care and quality of life.

Through information exchange and engagement in public dialogue, the Working Group has emphasized the importance of balancing an understanding of scientific research with real-world clinician expertise, each individual patient’s needs and history as well as their perspectives and preferences. The Working Group strives to empower patients and consumers by involving them in designing and prioritizing research, as well as reviewing evidence and contributing to its translation, dissemination and implementation.

For your consideration as you develop the Operational Plan for Comparative Effectiveness Research (CER), the Working Group has developed four recommendations for greater patient and consumer involvement in the CER process. Each of these channels represents a distinct and substantive vehicle through which the patient community can contribute its invaluable and unique insights. These recommendations were briefly noted in our oral remarks at the April 14, 2009 public meeting of the Federal Coordinating Council on Comparative Effectiveness Research (FCC-CER).

• Establish an HHS Office of Patient Values and Inclusion.  Patients and consumers should be involved in all aspects of developing the health care delivery systems and therapies for the major medical problems we face as a nation. Each of the HHS agencies is charged with specific aspects in ensuring patients/consumers are part of the CER process. The entire spectrum of HHS agencies must make substantial investments in patient and consumer involvement to more effectively realize the department’s overarching goals.
  
  For example, an Office of Patient Values and Inclusion could assist the NIH and AHRQ to better incorporate patient/consumer input into their basic scientific and health services research studies that will ultimately be applied in the clinical setting. A successful model of patient inclusion in this type of publicly funded research exists in the U.K.’s National Health System (NHS). The INVOLVE program was established by the NHS’ National Institute for Health Research (NIHR) to promote public involvement in research, in order to improve the way that research is prioritized, commissioned, undertaken, communicated and used. INVOLVE translates the public advice it receives into guides for researchers and research funders. The group posts minutes from all of its meetings and generally seeks to operate in a completely transparent manner. An analogous HHS office should seek to implement these best practices.  

• Create a national citizens’ advisory board to help HHS better understand the perspectives and values of the general public in designing and disseminating CER. It is critically important to make sure that the full continuum of issues is considered when making policy recommendations regarding the implementation of CER findings. While an HHS Office of Patient Values would have a broader portfolio including the full spectrum of HHS activities, a citizens’ advisory board would be concerned specifically with the ramifications of the substantial CER efforts that the department will undertake. This citizens’ advisory board could be established and administered by the Office of Patient Values.
  
  A good model for this is the Citizens’ Council convened by the UK's National Institute for Health and Clinical Excellence (NICE). NICE charges the Council with conveying the views of the public both to the Institute and to the groups that NICE commissions for appraisals and clinical guidelines. According to NICE, “the Citizens Council tackles challenging questions about values - such as fairness and need.” These difficult issues may be ignored or improperly addressed in the absence of a formal vehicle for citizen input. The Citizens Council is a small, carefully selected sample of 30 members.  In addition to a public comment period, the small group setting also allows for substantial discussion of difficult issues.

• Establish an explicit channel for HHS to educate patients on CER and, in turn, for patients to advise HHS. The FDA’s Patient Representative Program is an excellent model for this kind of participation. The program educates patients to be effective advisors, then includes them on advisory boards that correspond to their particular medical experience. HHS could similarly inform patients on some basic scientific and methodological concerns regarding CER and then solicit their input. These patients should be trained and supported in order to effectively engage in this process.   
  
  The FDA program has had a tangible impact on the way clinical research is conducted and has informed patient healthcare decision-making. The program shows that public involvement is a two-way street; the public would benefit from being educated on the details of CER, while the agency would benefit from listening to patients’ and consumers’ unique voices on CER matters.

• Sponsor research designed to improve clinical decision making by both clinicians and patients. Research that considers the individuality of health conditions and factors such as family history, individual experience in treatment and patient values and preferences is needed. It would be unfortunate if good clinical comparative research resulted in inappropriate, broad population-based treatment strategies that do not work for everyone. Designing CER trials that mine the heterogeneity of response for identifiable sub-groups, and which include outcome variables aligned with patient preferences, should improve the clinical applicability of the work. Patient involvement in trial design would assist in improving the relevance of their findings.
  
  Furthermore, significant effort is needed to leverage emerging data systems that are at the research and patient communities’ disposal to move CER forward. Integration with the developing HIT architecture should ensure that decision support tools informed by CER and systematic reviews are included in this new technology – moving knowledge to the bedside in order to craft individual treatment plans for individual patients. Patient-driven sites such as Patients Like Me may provide a blueprint for obtaining highly relevant comparative data in an efficient manner. Patients Like Me is a community of patients who interact and report their own clinical outcomes for a variety of therapeutics and disease states; while not as rigorous as controlled clinical trials, these large datasets incorporate the patients’ voices and results in information that is useful to them.

Greater roles for patient advocates are essential to ensuring HHS agencies more effectively evaluate and improve health care services and therapies. As taxpayers and end-users, citizens should have the opportunity to be actively involved in shaping federally-sponsored research and the organization and the delivery of health and social care services for their communities. Changes that are made to the evaluation of therapies and delivery of services should be based on their experiences and views.

The Working Group seeks to serve as a resource to ensure that the needs of patients are integrated into federal CER decision-making and studies. We host a website (www.evidencebasedhealthcare.org) that is a central repository of information for patients/consumers about evidence-based health care. Through our website, newsletter, forums and conference calls, we publicize opportunities for patient involvement. The Working Group has also issued several reports which include principles for patient inclusion throughout the CER process and models for meaningful patient /consumer inclusion (see attached). We are in the process of finalizing a toolkit to assist patients/consumers in becoming more conversant on topics such as evidence-based health care and CER. We welcome the opportunity to work with you and the FCC-CER moving forward.

The National Working Group on Evidence-Based Health Care appreciates the opportunity to provide specific input on our national investment in CER.

We welcome the opportunity to discuss our ideas further.

Sincerely,

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