Benefits of Collaboration with Lay Advocacy Group Community

There are creative examples of research studies whose protocol design, from the very beginning, encourage mutual commitment to, and shared responsibilities for, research tasks. Among these approaches are lay advocacy group efforts to fund, select and direct research of their particular rare diseases. One of the first and best prototypes for collaborative research teamwork between investigators and lay advocacy groups is the March of Dimes's unrelenting and successful campaign to find a cure for polio.

Other more recent successes point out the unique research vantage point enjoyed by researchers funded by lay advocacy group foundations. They work from start to finish inside the rare disease community and within sight of the concerns of families and participants. Through close proximity to the day to day rare disease specialists, i.e. families living with that rare disease, there is enhanced potential for achieving a better understanding of the complex physical and emotional layers of the rare disease. These direct connections between lay advocacy groups and clinical researchers can enhance opportunities for communication, collaborative, and trust, and support successful completion of the research study to the satisfaction of all partners.

Alliance Experience

Out of 600 lay advocacy groups in the Alliance database in 2003, over 150 groups reported direct involvement in research on their rare disease, maintenance of a database of affected persons, and encouragement of frequent research/family interface. It is important to get a more accurate picture of the process of these collaborative efforts, that is, to what extent consumers are involved from the beginning of the research protocol, whether a medical advisory committee reviews the research proposals and selects research protocols and the degree to which informed consent, educational and counseling components are considered standard practice. Groups that have already been collaborating successfully with researchers serve as critical models from which there is much to be learned. The Alliance can help determine existing models of collaboration and introduce them to the research community for active role modeling.

The Alliance stays aware of which groups are supporting collaborative research, how these efforts are proceeding, and to what extent these groups might be willing to train, advise and mentor new groups venturing into the research field. Umbrella organizations like the Alliance are also well suited, along with the larger lay advocacy groups, to provide workshops for the leaders of lay advocacy groups interested in directing their own research. These workshops could benefit from participation of the research community to explain IRB functions, research design protocols, the need for privacy safeguards with respect to patient registries, informed consent procedures, advice on participant expectations, and issues of confidentiality, privacy and genetic discrimination. The Alliance started this process at the September 1998 membership conference.

The Alliance can also be used as a referral source for families and individuals looking for on-going clinical research trials for their particular rare disease. We currently refer to the clinical trials at NIH and also refer to private research trials. Individual lay advocacy groups and research organizations also play a central role in linking researchers to families through conferences, newsletters, help lines, websites and patient registries.

Vision

A research partnership will develop between the consumer and research communities as respective visions, passions, interests and concerns are shared, appreciated and integrated. Optimal rare disease research occurs within the context of shared collaboration and enlightenment, mutual respect and on-going, two-way communication at every stage of the research protocol, from start to finish.

The bottom line is that rare disease research will be enhanced through full partnership, collaboration and cooperation between the consumer and research communities. The Alliance is ready to do its part to help make this happen. In our next section, we will look at three models for using many different approaches to pull together a research plan.

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