Table 3.

Initiatives for incorporation of PPs in BRA decision making.

Institutional level	Scope	Institution	Year	Purpose of the initiative	References
Governmental agencies	USA	FDA (CDRH)	2012	Pilot study (DCE eliciting PPs regarding a weight loss) to explore feasibility of PP studies and implementation of quantitative PPs in BRA	(4)
			2013–2017	Financial support for establishing CDER's Patient-Focused Drug Development initiativePublication of priority list of 20 disease areas that could benefit from the initiative	(5, 28)
		FDA	2015	Guidance on DMD and related disorders mentioning consideration of patient and caregiver risk tolerance in light of the life-threatening nature of the condition	(28)
	Europe	EMA	2009	Coordination of PROTECT collaborative project in partnership with IMINo specific guidance issued by the EMA	(41)
			2013–2015	EMA Patients' and Consumers' Organizations meeting conduction of a pilot study to assess feasibility and usefulness of systematically eliciting PPs for inclusion in BRA	(33, 42)
Private/public partnerships	Europe	IMI	2009	Launch of PROTECT collaborative project including two work packages dedicated to public/patient involvement in BRARecommendation of DCEs as preferred “utility survey technique”	(41, 42)
			2015	Launch of the 5th Call under IMI 2: Patient perspective elicitation on benefits and risks of medicinal products, from development through the entire life cycle, to inform the decision-making process by regulators and HTA bodies (PREFER project)	(42)
		EUPATI	2016	Patient training modules and guidance document development on PP elicitation and use in decision making (R&D, BRA, and HTA)	(10)
	USA	MDIC	2012	A framework to support FDA and industry in integration of PP in BRA of innovative medical devicesMDIC Methods Catalog: a general overview of available methods to quantify PP for BRA	(35)
Patient organizations and advocacy groups	USA	Parent Project Muscular Dystrophy Advocacy Group	2013	Development of the first patient-advocacy-initiated draft guidance for inclusion of PPs in regulatory decision making	(28)
			2015	PP pilot study that elicits preferences for a therapeutic agent that has demonstrated pulmonary benefit in a phase III clinical trial	(29)

FDA, US Food and Drug Administration; EMA, European Medicines Agency; IMI, Innovative Medicines Initiative; EUPATI, European Patients' Academy; MDIC, Medical Device Innovation Consortium; CDRH, Center for Devices and Radiological Health; DMD, Duchenne muscular dystrophy; DCE, discrete choice experiment; BRA, benefit–risk assessment; HTA, health technology assessment; R&D, research and development; PP, patient preference.