endpoints Reimbursement decision-making Input into the reimbursement decision-making process European Union Haemophilia Patients It was stated at the EHC Congress satellite Not applicable O’Mahony • (2014) symposium that it is necessary to begin engaging [96] HTA agencies, possibly through EU Network for HTA (EUnetHTA), to allow for patient input at Proposed the earliest stage of the HTA process was also suggested by a panel member that •It there is an opportunity for patient engagement in current efforts to develop common EUnetHTA’s practices across the EU that ensure consistency of data collection e.g., it is essential that the system is capable of • understanding the impact of an intervention in the context of the experience of hemophilia patients European Union Haemophilia Patients, To ensure that health technology assessments Not applicable O’Mahony • (2014) families, and (HTAs) consider factors beyond cost (i.e., factors [96] patient that matter to patients), a panel member at the organizations EHC Congress satellite symposium suggested that Proposed patient advocates and clinicians must work together to create a shared consensus on how to effectively evaluate the efficacy of therapies and what experiential data are of the greatest value Genetic United Kingdom Rare diseases in general Patients Nominated patient experts should be permitted to Not applicable • Alliance UK present a summary of the patient evidence (2014) submission to the Evaluation Committee [97] Proposed Genetic United Kingdom Rare diseases in general Patients Patient should be formally consulted during the Not applicable • Alliance UK National Institute of Health and Care Excellence (2014) (NICE) Highly Specialized Technology (HST) [97] evaluation process, prior to the final Committee meeting, to outline the nature Proposed and timeline of any post-evaluation research recommended by NICE Genetic United Kingdom Rare diseases in general Patients If the benefit/risk of a product is to be Not applicable • Alliance UK by NICE, patient input should be (2014) included and all information from patient
endpoints Reimbursement decision-making Input into the reimbursement decision-making process O’Mahony (2014) [96] Proposed European Union Haemophilia Patients • It was stated at the EHC Congress satellite symposium that it is necessary to begin engaging HTA agencies, possibly through EU Network for HTA (EUnetHTA), to allow for patient input at the earliest stage of the HTA process •It was also suggested by a panel member that there is an opportunity for patient engagement in EUnetHTA’s current efforts to develop common practices across the EU that ensure consistency of data collection • e.g., it is essential that the system is capable of understanding the impact of an intervention in the context of the experience of hemophilia patients Not applicable O’Mahony (2014) [96] Proposed European Union Haemophilia Patients, families, and patient organizations • To ensure that health technology assessments (HTAs) consider factors beyond cost (i.e., factors that matter to patients), a panel member at the EHC Congress satellite symposium suggested that patient advocates and clinicians must work together to create a shared consensus on how to effectively evaluate the efficacy of therapies and what experiential data are of the greatest value Not applicable Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Nominated patient experts should be permitted to present a summary of the patient evidence submission to the Evaluation Committee Not applicable Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Patient should be formally consulted during the National Institute of Health and Care Excellence (NICE) Highly Specialized Technology (HST) evaluation process, prior to the final Committee meeting, to outline the nature and timeline of any post-evaluation research recommended by NICE Not applicable Genetic Alliance UK (2014) United Kingdom Rare diseases in general Patients • If the benefit/risk of a product is to be by NICE, patient input should be included and all information from patient Not applicable
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