Orphan Drug Pricing and Reimbursement
Orphan Drug Pricing and Reimbursement
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Orphan drugs account for a growing portion of payers’ budgets – and comprise some of the most expensive treatments available.
Publisher: Datamonitor Healthcare
- Description
- Contents
- Samples
Orphan drugs account for a growing portion of payers’ budgets – and comprise some of the most expensive treatments available. With some therapies racking up multiple indications – and hundreds of millions of dollars in annual sales — payers are on guard, especially in Europe.
US insurers are not yet pushing back, but it is only a matter of time. The arrival of more personalized medicines and genetically defined disease subtypes will mean that eventually all treatments may look like orphans, as currently defined. The challenges these high-priced drugs face today are those that the entire industry will face tomorrow.
Table of Contents
5 EXECUTIVE SUMMARY
5 The orphan drug success story
5 Overview of orphan reimbursement in key markets
5 Orphan drug case studies highlight opportunities – and challenges
5 Real world evidence and outcomes key to valuing orphans
6 Orphans showcase industry-wide reimbursement and trust challenges
7 THE ORPHAN DRUG SUCCESS STORY
7 Number of approved orphan drugs has increased due to several incentives
7 Favorable reimbursement has boosted orphans, but is changing
9 Bibliography
11 ORPHAN REIMBURSEMENT PROCESSES IN KEY MARKETS
12 UK: only “ultra-orphans” get special treatment
14 France: tightening the screws on orphan reimbursement
15 Germany’s orphan sales threshold may be challenged
17 Italy: new P&R system may subject more orphans to managed entry
18 Spain: orphans suffer from wider economic woes
18 The Netherlands: conditional reimbursement does not work
19 US: no pressure on orphans yet, but it will not be long
20 Japan grants price premiums and supports intractable diseases
21 Bibliography
25 ORPHAN DRUG CASE STUDIES HIGHLIGHT OPPORTUNITIES – AND CHALLENGES
25 Alexion’s Soliris: multiple (rare) indications make a big drug
26 Pfizer’s Elelyso: an orphan too many for one rare disease?
27 Intermune’s Esbriet: testing orphan P&R in Germany
28 Novartis’s Ilaris: start small, get bigger strategy can stumble
29 NPS’s Gattex: driving a corporate turnaround
30 Bibliography
34 REAL WORLD EVIDENCE AND OUTCOMES KEY TO VALUING ORPHANS
34 The limitations of registries
35 Hurdles to orphan risk shares
36 Bibliography
38 ORPHANS SHOWCASE INDUSTRY-WIDE REIMBURSEMENT AND TRUST CHALLENGES
38 Orphans’ acute need for harmonized HTA
39 Orphans drive stakeholder dialog around “value” challenge
40 Bibliography
42 APPENDIX
42 About the author
LIST OF TABLES
42 Scope
42 Methodology
11 Table 1: Summary of orphan reimbursement in analyzed markets
16 Table 2: Orphan drug pricing in Germany: inconsistency between additional benefit and price
discount
Table 2: Orphan drug pricing in Germany: inconsistency between additional benefit and price discount
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