Call for Abstracts on Research Methods for Evaluating Patient Health Outcomes in Rare Diseases

The Effective Health Care Program within the US Department of Health and Human Services’ Agency for Healthcare Research and Quality is accepting abstract submissions for a journal supplement on research methods for evaluating patient health outcomes in rare diseases. The proposed supplement will focus on innovative patient-centered health outcomes research methodology and its application to rare diseases or drugs/devices used to treat such patients.  Topics areas of interest include:

1. Rare disease patients’ health outcomes in clinical trials, including outcomes arising from novel trial designs, such as adaptive clinical trials or n-of-1 trials

2. Emerging data sources for observational research on rare disease patients’ health outcomes, including the process of data aggregation and building disease registries or federated data networks

3. Novel analytic methods for observational research and prospective observational monitoring for rare disease patients’ health outcomes

4. Integrating stakeholder involvement in assessing rare disease patient health outcomes, particularly studies that describe or test how patient-centered health outcomes can be applied to enhance the development or evaluation of therapeutics for rare diseases

5. Economic, regulatory, ethical, legal, and social issues related to the study of rare disease health outcomes.  Authors may present empirical data or seek to collaborate in developing consensus statements outlining basic principles in these areas.

Proposals for original research articles and insightful reviews will be given highest priority.  Submissions are encouraged from authors with expertise in a broad range of study designs and from across multiple disciplines, including authors with methodological expertise in clinical trials, health outcomes studies, health services research, health policy, biostatistics, epidemiology, social science, and qualitative research.  The selected authors will be invited to submit a complete manuscript for peer-review and publication in the Journal of General Internal Medicine, a widely indexed journal with an international audience among practicing clinicians and general medical researchers. Abstract submissions are due by May 31, 2013.

More information and the submission form are available on the Effective Health Care Web site at:

Effective Health Care Program

The Petrie-Flom Center Staff

The Petrie-Flom Center staff often posts updates, announcements, and guests posts on behalf of others.

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