Situation	Reason why patient preferences are valuable	Source
Special disease areas and patient populations
Rare diseases, areas of unmet medical need and new disease areas	Because limited information available and limited expertise of doctors; “we don't quite know what we're doing” (IN7_EU).	IN, RE
Pediatric populations	Because of challenges in clinical trials and disease management related to formulation and route of administration.	IN, RE
Chronic diseases and oncology	Because of the time patients have to live with the disease. Although some found it as important in acute diseases, and did not think it would be valuable in well-known and well-studied common diseases.	IN
Diseases with a range of disease stages and manifestations	Because the disease heterogeneity and differences in treatment effects can cause split views between regulators as they may not be thinking of the same types of patients.	RE
Suspected preference heterogeneity and subgroups	Because patient preference studies could reveal subgroups for which the treatment is of different value, although regulators also mentioned this could complicate assessments.	RE, HTA
Special side and therapeutic effects
Special side effects	Because of unexpected side effects or safety issues, e.g. very toxic oncology treatments.	IN, RE
Novel benefits	Because it is uncertain what patients think about these novel benefits.	RE
Symptom relief	Because symptom relief is something that can be perceived in a different way among patients, especially if drugs have a delayed impact on symptoms.	IN
Prolongation of life	Because life prolongation of two or three months “might be considered by HTA bodies or payers as not so impressive” (IN3_EU), while patients can perceive this in a different way and may need to trade-off quality versus prolongation of life.	IN
Minor modifications in quality of life	Because it is difficult for evaluators to understand the impact of these minor modifications.	HTA
Special types of treatments
Novel therapies	Because it is uncertain what patients think about these novel technologies like precision medicine where it could help identify “the right patient”; “if you look at twins, identical twins. They both have the same disease. They are both offered the same treatment. The treatment, essentially, is going to react the same way with both of them but because of their different preferences one may choose it, one may not” (RE1_US).	RE
Treatments that preclude other treatments	Because the treatment would preclude the patient from getting alternative treatments in the future.	RE
Availability of other treatments	In some disease areas where a lot of treatments are available patient preferences were found to be not very valuable by regulators. In contrast, HTA representatives found patient preferences important in cases where alternatives are available that has different effects.	RE, HTA
Regulatory requirements	Because patient preferences could become more important than those of regulators when regulatory requirements are “old fashioned or even limited” (IN3_EU) due to unfamiliarity with the topic. However, in the case of strict regulatory pathways or “routine” (IN7_EU) clinical studies and disease areas there is no added value of patient preferences.	IN
Borderline benefit-risk profiles	Because regulators may not reach consensus on a decision due to presence of uncertainties with regards to clinical endpoints.	IN, RE
Borderline cost-effectiveness ratios	Because the result of the assessment is unclear.	HTA

HTA, health technology assessment focus group; IN, industry focus group; RE, regulatory focus group.