programs for patients/families Webinar (existing): Advocating for legislation • Volunteering at conferences Webinar (existing): • Webinar (existing): Advocating for improved quality of life • Organizing and sponsoring formal educational Advocating for education Patient care and support • • activities and training programs for health care Literature (existing): Advocating for clinics and patient care services • professionals, researchers, and policymakers Providing social support Facilitating Advocacy and • • patients’ individual Participating in standardized patient programs Awareness efforts • Patient organization development Advocacy and Awareness Literature (existing): Conferences and workshops Literature (existing): Establishing patient organizations Literature (existing): • Advocating for drug access/coverage Participating in conferences and workshops • • Webinar (existing): Organizing conferences • Advocating for research Funding conferences • • Starting awareness campaigns (e.g., fundraisers) • Patient care and support Conferences and workshops Literature (existing): Literature (existing): Providing social support • Participating in conferences and workshops Providing financial support • • Webinar (existing): Providing clinical care support • Presenting at conferences Providing support to patients participating in clinical • • Sitting on organizing committees trials • Patient care and support Patient organization development Literature (existing): Literature (existing): Providing social support Providing advice on develop patient organizations • • Monitoring own clinical care Fundraising to support further development of the • • Webinar (existing): organization Providing clinical care support • Regulatory decision-making Patient organization development Literature (existing): Literature (existing): Provide input in pre-submission advice given on trial • Establishing patient organizations protocol • Regulatory approval Research Research Research Literature (existing): Literature (existing): Literature (existing): Participating as subjects in studies outside of clinical Initiating research studies Participating as subjects in studies outside of clinical • • • trials Providing assistance to researchers conducting trials • Setting research priorities studies Setting research priorities • • Initiating research studies Leading research Initiating research studies • • • Providing assistance to researchers conducting Participating in research organizations/networks Providing assistance to researchers conducting studies • • • studies Disseminating research-related information Leading research • • Leading research Literature (proposed): Funding research • • Participating in research organizations/networks Participate in all decisions about research on rare Participating in research organizations/networks • • • Disseminating research-related information diseases Disseminating research-related information • • Literature (proposed): Participate in decision-making processes within •
programs for patients/families Webinar (existing): • Organizing and sponsoring formal educational activities and training programs for health care professionals, researchers, and policymakers • Participating in standardized patient programs Advocacy and Awareness Literature (existing): • Advocating for drug access/coverage Webinar (existing): • Advocating for research • Starting awareness campaigns (e.g., fundraisers) Conferences and workshops Literature (existing): • Participating in conferences and workshops Webinar (existing): • Presenting at conferences • Sitting on organizing committees Patient care and support Literature (existing): • Providing social support • Monitoring own clinical care Webinar (existing): • Providing clinical care support Patient organization development Literature (existing): • Establishing patient organizations Webinar (existing): • Volunteering at conferences Patient care and support Literature (existing): • Providing social support Patient organization development Literature (existing): • Establishing patient organizations • Advocating for legislation Webinar (existing): • Advocating for improved quality of life • Advocating for education • Advocating for clinics and patient care services • Facilitating patients’ individual Advocacy and Awareness efforts Conferences and workshops Literature (existing): • Participating in conferences and workshops • Organizing conferences • Funding conferences Patient care and support Literature (existing): • Providing social support • Providing financial support • Providing clinical care support • Providing support to patients participating in clinical trials Patient organization development Literature (existing): • Providing advice on develop patient organizations • Fundraising to support further development of the organization Regulatory decision-making Literature (existing): • Provide input in pre-submission advice given on trial protocol Regulatory approval Research Literature (existing): • Participating as subjects in studies outside of clinical trials • Setting research priorities • Initiating research studies • Providing assistance to researchers conducting studies • Leading research • Participating in research organizations/networks • Disseminating research-related information Literature (proposed): Research Literature (existing): • Initiating research studies • Providing assistance to researchers conducting studies • Leading research • Participating in research organizations/networks • Disseminating research-related information Literature (proposed): • Participate in all decisions about research on rare diseases • Participate in decision-making processes within Research Literature (existing): • Participating as subjects in studies outside of clinical trials • Setting research priorities • Initiating research studies • Providing assistance to researchers conducting studies • Leading research • Funding research • Participating in research organizations/networks • Disseminating research-related information