Healthcare Priority Setting and Rare Diseases What Matters When Reimbursing Orphan Drugs

................................................................................1 SVENSK SAMMANFATTNING .................................................. 3 LIST OF PAPERS ....................................................................... 5 ABBREVIATIONS ...................................................................... 7 INTRODUCTION ....................................................................... 9 Aims ...................................................................................................... 12 Outline of the thesis ............................................................................. 12 BACKGROUND ......................................................................... 13 Rare diseases and orphan drugs ...........................................................13 Rare diseases ........................................................................................... 13 Orphan drugs .......................................................................................... 15 Priority setting in healthcare ................................................................ 17 Health economic perspectives on orphan drugs ................................. 18 Diffusion and budget impact of orphan drugs ........................................ 18 Cost-effectiveness and orphan drugs ...................................................... 20 Ethical perspectives on orphan drugs ................................................. 22 Theories of distributive justice in healthcare .......................................... 23 Views on whether rarity should matter in priority setting ...................... 24 Procedural justice and public preferences .............................................. 27 Psychological perspectives on orphan drugs ....................................... 28 Identifiability & Singularity .................................................................... 29 Attribute framing ..................................................................................... 30 Proportion dominance ............................................................................. 31 Individual vs. group level decision making ............................................. 31 METHODS ............................................................................... 33 Use of methods ..................................................................................... 33 Healthcare priority setting and rare disases Quantitative, experimental methods: Papers I and II ........................ 34 Paper I: Population survey ..................................................................... 34 Paper II: Decision experiments ............................................................... 37 Qualitative methods ............................................................................. 40 Paper III: Focus group discussions ........................................................ 40 Paper IV: Semi-structured interviews ..................................................... 43 RESULTS ................................................................................. 47 Preferences for rarity and psychological effects influencing such preferences (Paper I) ............................................................................ 47 Data collection and respondent characteristics ...................................... 47 General preferences for rarity ................................................................ 47 Effects of the psychological manipulations ............................................. 48 The influence of identifiability and singularity on decision making (Paper II) ............................................................................................... 51 Descriptive results ................................................................................... 51 The influence of identifiability and singularity on choice ....................... 52 Follow-up questions ................................................................................ 56 Factors to consider when making reimbursement decisions regarding orphan drugs (Paper III) ...................................................................... 57 Components related to the patient........................................................... 58 Components related to the treatment ...................................................... 59 Economic components ............................................................................. 59 Societal components ................................................................................ 60 Perceived challenges and solutions when setting priorities regarding orphan drugs (Paper IV) ...................................................................... 61 Perceived challenges ............................................................................... 61 Perceived solutions .................................................................................. 64 DISCUSSION ........................................................................... 67 Does rarity matter in healthcare decision making? ............................ 67 Should rarity matter in healthcare decision making? ......................... 68 How rarity influences decision making ............................................... 70 Recommendations and further reflections .......................................... 73 CONCLUSIONS ........................................................................ 77 ACKNOWLEDGEMENTS ......................................................... 79 REFERENCES ......................................................................... 83 PAPERS I-IV .............................................................................91 Healthcare priority setting and rare disases