Literature (existing): Awareness efforts Advocating for drug access/coverage Patient care and support • Webinar (existing): Literature (existing): Conferences and workshops Advocating for research Providing social support Literature (existing): • • Starting awareness campaigns (e.g., fundraisers) Participating in conferences and workshops • • Patient organization development Organizing conferences • Conferences and workshops Literature (existing): Funding conferences • Literature (existing): Establishing patient organizations • Participating in conferences and workshops Patient care and support • Webinar (existing): Literature (existing): Presenting at conferences Providing social support • • Sitting on organizing committees Providing financial support • • Providing clinical care support • Patient care and support Literature (existing): Patient organization development Providing social support Literature (existing): • Monitoring own clinical care Providing advice on develop patient organizations • • Webinar (existing): Fundraising to support further development of the • Providing clinical care support organization • Patient organization development Literature (existing): Establishing patient organizations • Regulatory decision-making Literature (existing): Reporting adverse events • Reimbursement Research Research Research Literature (existing): Literature (existing): Literature (existing): decision-making 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 • Participate in all decisions about research on rare research collaborations/networks Patient reported outcome measures • diseases Webinar (existing): Literature (existing): Participate in decision-making processes within Funding research Assisting researchers in conducting studies to develop • • • research collaborations/networks and validate PROMs Webinar (existing): Patient registries and biorepositories
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 Regulatory decision-making Literature (existing): • Reporting adverse events Patient care and support Literature (existing): • Providing social support Patient organization development Literature (existing): • Establishing patient organizations 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 Patient organization development Literature (existing): • Providing advice on develop patient organizations • Fundraising to support further development of the organization Reimbursement decision-making 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): • Participate in all decisions about research on rare diseases • Participate in decision-making processes within research collaborations/networks Webinar (existing): 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 collaborations/networks Webinar (existing): • Funding research Patient registries and biorepositories 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 Patient reported outcome measures Literature (existing): • Assisting researchers in conducting studies to develop and validate PROMs
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