Advocating for research Providing social support Patient care and support • • Starting awareness campaigns (e.g., fundraisers) Literature (existing): • Patient organization development Providing social support • Literature (existing): Providing financial support Conferences and workshops • Literature (existing): Establishing patient organizations Providing clinical care support • • Participating in conferences and workshops • Webinar (existing): Patient organization development Presenting at conferences Literature (existing): • Sitting on organizing committees Providing advice on develop patient organizations • • Fundraising to support further development of the • organization Patient care and support Literature (existing): Providing social support Regulatory decision-making • Monitoring own clinical care Literature (existing): • Webinar (existing): Providing data for orphan drug designation • Providing clinical care support applications • Provide input in pre-submission advice given on trial • protocol Patient organization development Literature (existing): Membership on advisory or decision-making • Establishing patient organizations committees • Providing input on proposed regulation decision or • Regulatory decision-making guidelines Literature (existing): Participate in the assessment of benefits and harms • Consideration of PROs by decision-makers Providing input on plans for post-market approval • • Membership on advisory or decision-making pharmacovigilance • committees Providing input on consumer information (e.g., • Providing input on proposed regulation decision or labelling) • guidelines Literature (proposed): Participate in benefit/harm assessment Participate on regulatory committees that do not • • Reporting adverse events currently have patient representation • Literature (proposed): Identify laws, regulations, and policies that need to be • Provide input on acceptable risk changed in order to encourage product approval • Consult with regulatory bodies to establish greater • certainty in the approval process, especially in regards to trial design and endpoint selection Real world studies 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 • •
• 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): • Consideration of PROs by decision-makers • Membership on advisory or decision-making committees • Providing input on proposed regulation decision or guidelines • Participate in benefit/harm assessment • Reporting adverse events Literature (proposed): • Provide input on acceptable risk • Providing social support Patient organization development Literature (existing): • Establishing patient organizations 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 Regulatory decision-making Literature (existing): • Providing data for orphan drug designation applications • Provide input in pre-submission advice given on trial protocol • Membership on advisory or decision-making committees • Providing input on proposed regulation decision or guidelines • Participate in the assessment of benefits and harms • Providing input on plans for post-market approval pharmacovigilance • Providing input on consumer information (e.g., labelling) Literature (proposed): • Participate on regulatory committees that do not currently have patient representation • Identify laws, regulations, and policies that need to be changed in order to encourage product approval • Consult with regulatory bodies to establish greater certainty in the approval process, especially in regards to trial design and endpoint selection Real world studies 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 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): 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
13023_2017_738_MOESM4_ESM.docx