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]