Webinar (existing): • Facilitating relationships between stakeholders • Establishing relationships with stakeholders Education Literature (existing): • Helping to develop educational material/training 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 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 activities and training programs for health care professionals, researchers, and policymakers • Participating in standardized patient programs Advocacy and Awareness Webinar (existing): • Advocating for research • Starting awareness campaigns (e.g., fundraisers) Conferences and workshops Literature (existing): • Participating in conferences and workshops Webinar (existing): • Volunteering at conferences Patient care and support Literature (existing): • Providing social support Patient organization development Literature (existing): • Establishing patient organizations Advocacy and Awareness Literature (existing): • Advocating for research • Advocating for legislation • Starting awareness campaigns 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 Patient organization development Literature (existing): • Providing advice on develop patient organizations • Fundraising to support further development of the organization Clinical trials Research Literature (existing): • Participating as subjects in studies outside of clinical trials • Setting research priorities • Initiating research studies • Providing assistance to researchers conducting Research Literature (existing): • Initiating research studies • Providing assistance to researchers conducting studies • Leading research • Participating in research organizations/networks Research Literature (existing): • Participating as subjects in studies outside of clinical trials • Setting research priorities • Initiating research studies • Providing assistance to researchers conducting studies
• 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
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
• 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 Reimbursement decision-making Literature (existing): • Submitting topics for evaluation • Providing information for preparation of evaluation report • Directly consulted during review process • Membership on advisory/decision-making committee • Providing feedback on evaluation report and/or proposed recommendations • Preparing a patient submission • Presenting views during committee meeting Literature (proposed): • Providing input on benefits and risks during topic selection by horizon scanning producing organizations • Providing input at the earliest stages of the HTA process • Presenting a summary of patient submissions to reimbursement evaluation committees • Consulting in the outlining of post-evaluation research recommended by reimbursement • Provide input into any re-assessments of benefits and risks 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 Literature (existing): • Submitting topics for evaluation • Directly consulted during review process • Membership on advisory/decision-making committee • Preparing a patient submission Literature (proposed): • Consulting in topic identification and selection • Assuring reimbursement of off-label drug use Routine clinical use Research Research Research
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 Regulatory decision-making Literature (existing): • Reporting adverse events • Advocating for research • Starting awareness campaigns (e.g., fundraisers) Conferences and workshops Literature (existing): • Participating in conferences and workshops Webinar (existing): • Volunteering at conferences Patient care and support Literature (existing): • Providing social support Patient organization development Literature (existing): • Establishing patient organizations • Advocating for drug access/coverage • Advocating for research • 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 Patient organization development Literature (existing): • Providing advice on develop patient organizations • Fundraising to support further development of the organization Obsolescence/ replacement with a new therapy 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
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 Reimbursement decision-making Literature (existing): • Providing information for preparation of evaluation report • Directly consulted during review process • Membership on advisory/decision-making committee • Providing feedback on evaluation report and/or proposed recommendations • Preparing a patient submission • Presenting views during committee meeting Literature (proposed): • Providing input at the earliest stages of the HTA process • Presenting a summary of patient submissions to reimbursement evaluation committees • Provide input into any re-assessments of benefits and 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 Literature (existing): • Directly consulted during review process • Membership on advisory/decision-making committee • Preparing a patient submission Literature (proposed): • Assuring reimbursement of off-label drug use