Initiate Clinical and Research Partnerships 74;75

Table 5. Classification of opportunities for patient input according to stage in technology life-cycle and type of uncertainty they aim to reduce
Step in the technology lifecycle / Type of Uncertainty
Clinical Benefit / Value for Money / Affordability / Adoption/Diffusion
Pre-clinical phase /

Fund research [70;71;73;76;80;81;87;138]
Initiate clinical and research partnerships[74;75]
Establish biotechnology/biopharmaceutical companies [80]
Initiate pre-clinical research [80]
Develop patient registries and on-line patient communities [70;81;85;86;90;93]
Collaborate:
In creating biobanks [70;86;87]
With researchers and physicians [70;72;74;80;138]
Participate:
In the executive or scientific committees of research institutions/networks [70;137]
In the development of disease specific outcome measures[88]
The steering committees of biotechnology companies [138]
The development of genetic research policy
The prioritization of treatments on the basis of benefits to and needs of the patient before clinical trial design [92]
In projects to coordinate rare disease research [70]
In clinical practice guideline development[86]
Provide:
Samples to biobanks [70;86;87]
Potential participants for studies through registries[72;81;82]
Information on potential/available treatments and their effects to other patients and physicians [71]
Data to patient registries [71;81;82;84-86;93]
Information to online patient communities [70;90]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies[136;138]

Information on patients’ priorities for research [77-79]
Clinical trials /

Fund research [70;71;73;76;80;81;87;138]
Initiate clinical and research partnerships[74;75]
Design and conduct clinical trials [70;87;90]
Develop patient registries and online patient communities [70;81;85;86;90;93]
Establish biotechnology/biopharmaceutical companies [80]
Recruit participants for clinical trials [70;93;137;138]
Create information registries to guide clinical trial design [86]
Participate in the development of disease specific outcome measures[88]
Run training programs for patient representatives [70]
Identify motivations to participate in clinical trials [94]
Identify and validate relevant patient-reported outcomes [70;86]
Establish agreements on collaboration between clinical trial sponsors and patient organizations
Collaborate:
In creating biobanks [70;86;87]
With researchers and physicians [70;72;74;80;138]
Provide:
Samples to biobanks [70;86;87]
Potential participants for studies through registries[72;81;82]
Information on potential/available treatments and their effects to other patients and physicians [71]
Data to patient registries [71;81;82;84-86;93]
Information to online patient communities [70;90]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies [136;138]
The prioritization of treatments on the basis of benefits to and needs of the patient before clinical trial design [92]
Information and counselling to trial participants [70]
Information to contact registries for clinical trial participation
Input into web-based clinical records [83]
Accurate information to professionals via the internet [138]

Fund research [70;71;73;80;87;138]
Design and conduct clinical trials [70;87;90]
Develop patient registries and online patient communities [70;81;85;86;90;93]
Establish biotechnology/biopharmaceutical companies [80]
Recruit participants for clinical trials [70;137;138]
Create information registries to guide clinical trial design [86]
Run training programs for patient representatives [70]
Identify and validate relevant patient-reported outcomes [70;86]
Establish agreements on collaboration between clinical trial sponsors and patient organizations
Collaborate:
In creating biobanks [70;86;87]
With researchers and physicians [70;72;74;80;138]
Provide:
Samples to biobanks [70;86;87]
Information on potential/available treatments and their effects to other patients and physicians [71]
Data to patient registries [71;81;84-86;93]
Information to online patient communities [70;90]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies; [136;138]
The prioritization of treatments on the basis of benefits to and needs of the patient before clinical trial design [92]
Information and counselling to trial participants [70]
Information to contact registries for clinical trial participation
Input into web-based clinical records [83]
Accurate information to professionals via the internet [138]

Develop patient registries [81;85;86;93]
Provide data to registries [71;81;82;84-86;93]

Develop patient registries and online patient communities [70;81;85;86;90;93]
Participate in contact registries for clinical trial participation
Provide:
Information on potential/available treatments and their effects to other patients and physicians [71]
Data to patient registries [71;81;82;84-86;93]
Information to online patient communities [70;71;84;90]
Input into web-based clinical records [83]
Accurate information to professionals via the internet [138]

Regulatory approval /

Run training programs for patient representatives [70]
Develop patient registries [81;85;86;93]
Participate in:
Advisory committees [21;22;27;34;37;38;70;70;90;136-139]
In clinical practice guideline development[86]
In decision-making committees [21;30;40]
The establishment of orphan drug regulations [137]
Provide:
Data to patient registries [71;81;82;84-86;93]
Input on proposed regulatory decision or preparation of guidelines [27;30;36;39-42]
Input in protocol assistance [21;43;99]
Input in benefit-harm assessment [21;25;27;43;44]
Input on the assessment of a risk management plan [21;43]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies; [71;136;138]
Accurate information to professionals via the internet [138]

Run training programs for patient representatives [70]
Develop patient registries [81;85;86;93]
Participate in:
Advisory committees [21;22;27;34;37;38;70;137-139]
The establishment of orphan drug regulations [137]
Provide:
Data to patient registries [71;81;82;85;86;93]
Input on proposed regulatory decision or preparation of guidelines [27;30;36;39-42]
Input in protocol assistance [21;43;99]
Input in benefit-harm assessment [21;25;27;43;44]
Input on the assessment of a risk management plan [21;43]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies; [136;138].
Accurate information to professionals via the internet [138]

Develop patient registries [81;85;86;93]
Provide data to registries [81;85;86;93] [71;82;84]

Develop patient registries [81;85;86;93]
Provide:
Data to patient registries [71;81;82;84-86;93]
Accurate information to professionals via the internet [138]

Post-marketing/real world studies /

Run training programs for patient representatives [70]
Develop patient registries [81;84-86;93]
Collaborate with researchers and physicians [70;72;74;75;80;138]
Participate In clinical practice guideline development[86]
Provide:
Data to patient registries [71;81;82;84-86;93]
Input into web-based clinical records [83]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies [136;138]
Accurate information to professionals via the internet [138]
Input to post-marketing surveillance [26;27;33;35;43;45-49]

Run training programs for patient representatives [70]
Develop patient registries [81;84-86;93]
Collaborate with researchers and physicians [70;72;74;75;80;138]
Provide:
Data to patient registries [71;81;82;84-86;93]
Input into web-based clinical records [83]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies [136;138]
Accurate information to professionals via the internet [138]

Develop patient registries [81;84-86;93]
Provide data to registries [81;82;84-86;93]

Develop patient registries [81;84-86;93]
Provide:
Data to patient registries [81;82;84-86;93]
Input into web-based clinical records [83]
Accurate information to professionals via the internet [138]

Reimbursement decision-making /

Run training programs for patient representatives [70]
Submit application for consideration [50;52;63-66;68;117]
Develop patient registries [81;84-86;93]
Participate:

In advisory or decision-making committees [50-60;62;70;136]
In clinical practice guideline development[86]

Provide:
Additional comments/evidence on evaluation report and/or recommendations [51-53;57;67-69]

[61-66]

Patient views during committee/board meetings [52]
Input into evaluations [57;69]
Data to patient registries [81;82;84-86;93]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies [136;138]
Input on the relative importance of different criteria for decision-making[95]
Accurate information to professionals via the internet [138]

Run training programs for patient representatives [70]
Submit application for consideration
Develop patient registries [81;84-86;93;140]
Participate in advisory or decision-making committees[70;136]
Provide:
Additional comments/evidence on evaluation report and/or recommendations [51-53;57;67-69]

[61-66]

Patient views during committee/board meetings [52]
Consultation for evaluations [57;69]
Data to patient registries [81;82;84-86;93]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies [136;138]
Accurate information to professionals via the internet [138]

Develop patient registries [81;84-86;93]
Provide data to registries [81;82;84-86;93]

Develop patient registries [81;84-86;93]
Provide:
Accurate information to professionals via the internet [138]
Data to patient registries [81;82;84-86;93]

Introduced as insured service/ use in routine clinical practice /

Develop patient registries and online patient communities [70;81;85;86;90;93]
Design patient route maps [89]
Collaborate with researchers and physicians [70;72;74;75;80;138]
Participate in online patient communities [70;71;90]
Provide:
Input into web-based clinical records [83]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies [136;138]
Accurate information to professionals via the internet [138]

Develop patient registries and online patient communities [70;81;85;86;90;93]
Design patient route maps [89]
Collaborate with researchers and physicians [70;72;74;80;138]
Participate in online patient communities [70;90]
Provide:
Input into web-based clinical records [83]
Input on topics such as: disease impact; treatment benefits that are important; adequacy, side effects and optimum doses of available therapies [136;138]
Accurate information to professionals via the internet [138]

Develop patient registries [81;85;86;93]
Provide data to registries [81;82;85;86;93]

Develop patient registries and online patient communities[81;85;86;93]
Provide:
Data to patient registries [81;82;85;86;93]
Information to online patient communities[70;90]
Input into web-based clinical records[83]
Accurate information to professionals via the internet[138]