MISSED OPPORTUNITIES IN THE NETHERLANDS- THE NEGLIGENCE OF NON-STATISTICAL UNCERTAINTY IN CED POLICY
Author(s)
Pouwels XG1, Grutters JP2, Bindels JA1, Ramaekers BL1, Joore MA3
1Maastricht University Medical Center+, Maastricht, The Netherlands, 2Radboud university medical center, Nijmegen, The Netherlands, 3Maastricht University Medical Center, Maastricht University, Maastricht, The Netherlands
OBJECTIVES: Coverage with evidence development (CED) has been introduced in the Netherlands to regulate reimbursement of expensive drugs. CED aims at reducing uncertainty and providing better value for money when coverage decisions of medicines have to be made. During CED, patients were granted access to the drug for four years, while research was undertaken into the (cost-)effectiveness of the drug. This research uses the following categorisation of uncertainty: 1) non-statistical uncertainty because of absence of evidence; 2) non-statistical uncertainty because of lack of relevance of available evidence (e.g. indirect evidence, relevance of outcome measure) and; 3) statistical uncertainty (i.e. precision) surrounding available evidence. To identify which uncertainties played a role in three CED dossiers, assess whether these uncertainties were investigated during CED research, and identify issues and ways forward related to uncertainty in CED according to stakeholders. METHODS: Three Dutch CED dossiers (initial and final assessment reports) were analysed and interviews were conducted with sixteen stakeholders (decision makers, clinicians, patient representatives and industry representatives) involved in those dossiers. We used the initial reports and interviews to identify which uncertainties played a role in those dossiers. Final reports were used to ascertain whether these uncertainties were researched during CED. Issues and ways forward with uncertainty in CED policy were discussed in the interviews. RESULTS: Eighteen non-statistical and nine statistical uncertainties were identified. Statistical uncertainties were relatively more often researched than non-statistical uncertainties (56% versus 22% respectively). Stakeholders advocated for more flexible CED research design and better agreements (on research outcomes) before CED research initiation. CONCLUSIONS: Dutch CED policy focused on reducing statistical uncertainty. A systematic identification of all uncertainties is required to target CED research on elements which will improve coverage decisions. Expert opinion and qualitative evidence should be accepted as CED research designs in order to handle non-statistical uncertainty.
Conference/Value in Health Info
2016-10, ISPOR Europe 2016, Vienna, Austria
Value in Health, Vol. 19, No. 7 (November 2016)
Code
PCN229
Topic
Health Policy & Regulatory
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways
Disease
Oncology, Rare and Orphan Diseases