Acute myeloid leukemia (AML)

CONTENTS

13 FORECAST: ACUTE MYELOID LEUKEMIA (Published on 13 November 2017)
13 EXECUTIVE SUMMARY
14 MARKET OVERVIEW AND TRENDS
29 MARKET DEFINITION AND METHODOLOGY
37 ATIR101
42 DACOGEN (DECITABINE)
47 GILTERITINIB
53 GUADECITABINE
58 IDASANUTLIN
62 IDHIFA (ENASIDENIB)
68 IOMAB-B
73 IVOSIDENIB
79 MYLOTARG (GEMTUZUMAB OZOGAMICIN)
84 ORAL VIDAZA (AZACITIDINE)
88 PRACINOSTAT
92 QUIZARTINIB
97 RYDAPT (MIDOSTAURIN)
102 VENCLEXTA (VENETOCLAX)
106 VIDAZA (AZACITIDINE)
112 VYXEOS ([CYTARABINE + DAUNORUBICIN])
117 ZELTHERVA (GALINPEPIMUT-S)
122 PRIMARY RESEARCH METHODOLOGY

124 TREATMENT: ACUTE MYELOID LEUKEMIA (Published on 03 May 2017)
124 EXECUTIVE SUMMARY
125 PRIMARY RESEARCH METHODOLOGY
127 DISEASE DEFINITION AND DIAGNOSIS
141 PATIENT SEGMENTATION
143 CURRENT TREATMENT OPTIONS
146 PRESCRIBING TRENDS

167 EPIDEMIOLOGY: ACUTE MYELOID LEUKEMIA (Published on 13 December 2016)
167 EXECUTIVE SUMMARY
168 DISEASE BACKGROUND
173 METHODOLOGY
193 FORECAST
196 SUPPLEMENTARY ANALYSIS
212 BIBLIOGRAPHY
217 APPENDIX: ADDITIONAL SOURCES

218 MARKETED DRUGS: ACUTE MYELOID LEUKEMIA (Published on 06 July 2017)
218 EXECUTIVE SUMMARY
219 PRODUCT OVERVIEW
220 PRODUCT PROFILE: DACOGEN
227 PRODUCT PROFILE: RYDAPT
235 PRODUCT PROFILE: VIDAZA

243 ACUTE MYELOID LEUKEMIA PRICING, REIMBURSEMENT, AND ACCESS (Published on 15 December 2017)
243 EXECUTIVE SUMMARY
244 REGULATORY LABELS
248 PAYER ARCHETYPES
273 ACCESS TO RECENTLY LAUNCHED AND PIPELINE PRODUCTS
284 CLINICAL TRIAL DESIGN AND EVIDENTIARY REQUIREMENTS
290 US PRICING
291 US REIMBURSEMENT
307 JAPAN
309 PRICING IN THE FIVE MAJOR EU MARKETS
310 FRANCE
322 GERMANY
333 ITALY
343 SPAIN
351 UK
364 APPENDIX

367 PIPELINE: ACUTE MYELOID LEUKEMIA (Published on 06 July 2017)
367 EXECUTIVE SUMMARY
368 CLINICAL PIPELINE OVERVIEW
374 PRODUCT PROFILE (LATE STAGE): ATIR101
382 PRODUCT PROFILE (LATE STAGE): IDHIFA
393 PRODUCT PROFILE (LATE STAGE): IOMAB-B
400 PRODUCT PROFILE (LATE STAGE): VENCLEXTA
412 PRODUCT PROFILE (LATE STAGE): VYXEOS
422 PRODUCT PROFILE (LATE STAGE): ZELTHERVA
430 PRODUCT PROFILE (LATE STAGE): GILTERITINIB
442 PRODUCT PROFILE (LATE STAGE): GUADECITABINE
451 PRODUCT PROFILE (LATE STAGE): IDASANUTLIN
459 PRODUCT PROFILE (LATE STAGE): IVOSIDENIB
469 PRODUCT PROFILE (LATE STAGE): ORAL VIDAZA
477 PRODUCT PROFILE (LATE STAGE): QUIZARTINIB

LIST OF FIGURES
15 Figure 1: AML sales across the US, Japan, and five major EU markets, by country, 2016–25
18 Figure 2: AML sales across the US, Japan, and five major EU markets, by treatment setting, 2016–25
21 Figure 3: AML sales across the US, Japan, and five major EU markets, by class, 2016–25
24 Figure 4: AML sales for FLT3 inhibitors across the US, Japan, and five major EU markets, by drug, 2016–25
26 Figure 5: AML sales for IDH inhibitors across the US, Japan, and five major EU markets, by drug, 2016–25
27 Figure 6: AML sales in the US, by class, 2016–25
32 Figure 7: Methodology flow for Datamonitor Healthcare’s AML forecast
33 Figure 8: Price sources and calculations, by country
38 Figure 9: ATIR101 sales for AML across the US and five major EU markets, by country, 2016–25
43 Figure 10: Dacogen sales for AML across the US and five major EU markets, by country, 2016–25
49 Figure 11: Gilteritinib sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
54 Figure 12: Guadecitabine sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
59 Figure 13: Idasanutlin sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
64 Figure 14: IDHIFA sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
69 Figure 15: Iomab-B sales for AML across the US and five major EU markets, by country, 2016–25
75 Figure 16: Ivosidenib sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
80 Figure 17: Mylotarg sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
85 Figure 18: Oral Vidaza sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
89 Figure 19: Pracinostat sales for AML across the US and five major EU markets, by country, 2016–25
93 Figure 20: Quizartinib sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
98 Figure 21: Rydapt sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
103 Figure 22: Venclexta sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
108 Figure 23: Vidaza sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
113 Figure 24: Vyxeos sales for AML across the US, Japan, and five major EU markets, by country, 2016–25
118 Figure 25: Zeltherva sales for AML across the US and five major EU markets, by country, 2016–25
136 Figure 26: Proportion of AML patients referred for specific cytogenetic and mutational tests, by age
141 Figure 27: Proportion of AML patients according to age and disease stage, by country
142 Figure 28: Proportion of AML patients in each risk status category, by age
147 Figure 29: First-line induction treatment types for newly diagnosed AML patients below 60, by country
148 Figure 30: First-line induction treatment types for newly diagnosed AML patients aged 60 and above, by country
150 Figure 31: Top three first-line induction therapies for newly diagnosed AML patients below 60, by country
151 Figure 32: Top three first-line induction therapies for newly diagnosed AML patients aged 60 and above, by country
152 Figure 33: Second-line induction treatment types for newly diagnosed AML patients below 60, by country
153 Figure 34: Second-line induction treatment types for newly diagnosed AML patients aged 60 and above, by country
154 Figure 35: Top three second-line induction therapies for newly diagnosed AML patients under 60, by country
155 Figure 36: Top three second-line induction therapies for newly diagnosed AML patients aged 60 and above, by country
156 Figure 37: Post-remission treatment types for newly diagnosed AML patients below 60, by country
157 Figure 38: Post-remission treatment types for newly diagnosed AML patients aged 60 and above, by country
158 Figure 39: Top three post-remission pharmacological therapies for AML patients below 60, by country
159 Figure 40: Top three post-remission pharmacological therapies for AML patients aged 60 and above, by country
160 Figure 41: Treatment types for relapsed and/or refractory AML patients below 60, by country
161 Figure 42: Treatment types for relapsed and/or refractory AML patients aged 60 and above, by country
162 Figure 43: Top three pharmacological regimens for relapsed or treatment-refractory AML patients below 60 receiving pharmacological therapy alone, by country
163 Figure 44: Top three pharmacological regimens for relapsed or treatment-refractory AML patients below 60 receiving pharmacological therapy followed by HSCT, by country
164 Figure 45: Top three pharmacological regimens for relapsed or treatment-refractory AML patients aged 60 or above receiving pharmacological therapy alone, by country
165 Figure 46: Top three pharmacological regimens for relapsed or treatment-refractory AML patients aged 60 and above receiving pharmacological therapy followed by HSCT, by country
195 Figure 47: Trends in incident cases of acute myeloid leukemia in the US, Japan, and five major EU markets, by country, 2015–35
223 Figure 48: Dacogen for acute myeloid leukemia – SWOT analysis
224 Figure 49: Datamonitor Healthcare’s drug assessment summary of Dacogen for acute myeloid leukemia
225 Figure 50: Datamonitor Healthcare’s drug assessment summary of Dacogen for acute myeloid leukemia
231 Figure 51: Datamonitor Healthcare’s drug assessment summary of Rydapt for acute myeloid leukemia
232 Figure 52: Datamonitor Healthcare’s drug assessment summary of Rydapt for acute myeloid leukemia
238 Figure 53: Vidaza for acute myeloid leukemia – SWOT analysis
239 Figure 54: Datamonitor Healthcare’s drug assessment summary of Vidaza for acute myeloid leukemia
240 Figure 55: Datamonitor Healthcare’s drug assessment summary of Vidaza for acute myeloid leukemia
365 Figure 56: Price sources and calculations, by country
378 Figure 57: ATIR101 for acute myeloid leukemia – SWOT analysis
379 Figure 58: Datamonitor Healthcare’s drug assessment summary of ATIR101 for acute myeloid leukemia
380 Figure 59: Datamonitor Healthcare’s drug assessment summary of ATIR101 for acute myeloid leukemia
388 Figure 60: IDHIFA for acute myeloid leukemia – SWOT analysis
389 Figure 61: Datamonitor Healthcare’s drug assessment summary of IDHIFA for acute myeloid leukemia
390 Figure 62: Datamonitor Healthcare’s drug assessment summary of IDHIFA for acute myeloid leukemia
396 Figure 63: Iomab-B for acute myeloid leukemia – SWOT analysis
397 Figure 64: Datamonitor Healthcare’s drug assessment summary of Iomab-B for acute myeloid leukemia
398 Figure 65: Datamonitor Healthcare’s drug assessment summary of Iomab-B for acute myeloid leukemia
408 Figure 66: Venclexta for acute myeloid leukemia – SWOT analysis
409 Figure 67: Datamonitor Healthcare’s drug assessment summary of Venclexta for acute myeloid leukemia
410 Figure 68: Datamonitor Healthcare’s drug assessment summary of Venclexta for acute myeloid leukemia
417 Figure 69: Vyxeos for acute myeloid leukemia – SWOT analysis
418 Figure 70: Datamonitor Healthcare’s drug assessment summary of Vyxeos for acute myeloid leukemia
419 Figure 71: Datamonitor Healthcare’s drug assessment summary of Vyxeos for acute myeloid leukemia
426 Figure 72: Zeltherva for acute myeloid leukemia – SWOT analysis
427 Figure 73: Datamonitor Healthcare’s drug assessment summary of Zeltherva for acute myeloid leukemia
428 Figure 74: Datamonitor Healthcare’s drug assessment summary of Zeltherva for acute myeloid leukemia
437 Figure 75: Gilteritinib for acute myeloid leukemia – SWOT analysis
438 Figure 76: Datamonitor Healthcare’s drug assessment summary of gilteritinib for acute myeloid leukemia
439 Figure 77: Datamonitor Healthcare’s drug assessment summary of gilteritinib for acute myeloid leukemia
447 Figure 78: Guadecitabine for acute myeloid leukemia – SWOT analysis
448 Figure 79: Datamonitor Healthcare’s drug assessment summary of guadecitabine for acute myeloid leukemia
449 Figure 80: Datamonitor Healthcare’s drug assessment summary of guadecitabine for acute myeloid leukemia
455 Figure 81: Idasanutlin for acute myeloid leukemia – SWOT analysis
456 Figure 82: Datamonitor Healthcare’s drug assessment summary of idasanutlin for acute myeloid leukemia
457 Figure 83: Datamonitor Healthcare’s drug assessment summary of idasanutlin for acute myeloid leukemia
464 Figure 84: ivosidenib for acute myeloid leukemia – SWOT analysis
465 Figure 85: Datamonitor Healthcare’s drug assessment summary of ivosidenib for acute myeloid leukemia
466 Figure 86: Datamonitor Healthcare’s drug assessment summary of ivosidenib for acute myeloid leukemia
472 Figure 87: Oral azacitidine for acute myeloid leukemia – SWOT analysis
473 Figure 88: Datamonitor Healthcare’s drug assessment summary of oral Vidaza for acute myeloid leukemia
474 Figure 89: Datamonitor Healthcare’s drug assessment summary of oral Vidaza for acute myeloid leukemia
483 Figure 90: quizartinib for acute myeloid leukemia – SWOT analysis
484 Figure 91: Datamonitor Healthcare’s drug assessment summary of quizartinib for acute myeloid leukemia
485 Figure 92: Datamonitor Healthcare’s drug assessment summary of quizartinib for acute myeloid leukemia

LIST OF TABLES
16 Table 1: AML sales across the US, Japan, and five major EU markets, by country ($m), 2016–25
19 Table 2: AML sales across the US, Japan, and five major EU markets, by treatment setting ($m), 2016–25
22 Table 3: AML sales across the US, Japan, and five major EU markets, by class ($m), 2016–25
30 Table 4: Summary of drug classes and molecules included in the AML patient-based forecast
34 Table 5: Exchange rates used for calculating prices
34 Table 6: Patent and market exclusivity expiry dates for key marketed brands in AML in the US, Japan, and five major EU markets, by region
35 Table 7: Forecast launch dates of key late-stage pipeline products and generics in AML in the US, Japan, and five major EU markets, by region
39 Table 8: ATIR101 sales for AML across the US and five major EU markets, by country ($m), 2016–25
41 Table 9: ATIR101 patient numbers for AML across the US and five major EU markets, by country, 2016–25
44 Table 10: Dacogen sales for AML across the US and five major EU markets, by country ($m), 2016–25
46 Table 11: Dacogen patient numbers for AML across the US and five major EU markets, by country, 2016–25
50 Table 12: Gilteritinib sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
52 Table 13: Gilteritinib patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
55 Table 14: Guadecitabine sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
57 Table 15: Guadecitabine patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
60 Table 16: Idasanutlin sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
62 Table 17: Idasanutlin patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
65 Table 18: IDHIFA sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
67 Table 19: IDHIFA patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
70 Table 20: Iomab-B sales for AML across the US and five major EU markets, by country ($m), 2016–25
72 Table 21: Iomab-B patient numbers for AML across the US and five major EU markets, by country, 2016–25
76 Table 22: Ivosidenib sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
78 Table 23: Ivosidenib patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
81 Table 24: Mylotarg sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
83 Table 25: Mylotarg patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
86 Table 26: Oral Vidaza sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
88 Table 27: Oral Vidaza patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
90 Table 28: Pracinostat sales for AML across the US and five major EU markets, by country ($m), 2016–25
92 Table 29: Pracinostat patient numbers for AML across the US and five major EU markets, by country, 2016–25
94 Table 30: Quizartinib sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
96 Table 31: Quizartinib patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
99 Table 32: Rydapt sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
101 Table 33: Rydapt patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
104 Table 34: Venclexta sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
106 Table 35: Venclexta patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
109 Table 36: Vidaza sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
111 Table 37: Vidaza patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
114 Table 38: Vyxeos sales for AML across the US, Japan, and five major EU markets, by country ($m), 2016–25
116 Table 39: Vyxeos patient numbers for AML across the US, Japan, and five major EU markets, by country, 2016–25
119 Table 40: Zeltherva sales for AML across the US and five major EU markets, by country ($m), 2016–25
121 Table 41: Zeltherva patient numbers for AML across the US and five major EU markets, by country, 2016–25
122 Table 42: Hematologists and hemato-oncologists surveyed for the AML primary research study, 2016
125 Table 43: Hematologists and hemato-oncologists surveyed for the AML primary research study, 2016
131 Table 44: 2016 revision of the World Health Organization classification of myeloid neoplasms and acute leukemia
133 Table 45: AML risk status based on cytogenetics and molecular abnormalities
143 Table 46: Leading brands available for AML across the US, Japan, and five major EU markets
144 Table 47: Common chemotherapies used for the treatment of AML
170 Table 48: 2008 revision of the World Health Organization classification of myeloid neoplasms and acute leukemia
174 Table 49: Acute myeloid leukemia disease definition
177 Table 50: Acute myeloid leukemia disease definition data processing summary
179 Table 51: Data sources used for the analysis of acute myeloid leukemia incidence in the US, Japan, and five major EU markets, by country
184 Table 52: Forecasting methodology for acute myeloid leukemia incidence, by country, age group, and gender
188 Table 53: US National Comprehensive Cancer Network 2016 acute myeloid leukemia risk stratification
189 Table 54: US National Comprehensive Cancer Network 2010 acute myeloid leukemia risk stratification
190 Table 55: European LeukemiaNet acute myeloid leukemia prognostic system
192 Table 56: Sources of survival data used to estimate acute myeloid leukemia prevalence, by country
194 Table 57: Incident cases of acute myeloid leukemia in the US, Japan, and five major EU markets, by country, 2015–35
197 Table 58: 2008 revision of the World Health Organization classification of myeloid neoplasms and acute leukemia, and corresponding ICD-O-3 codes
200 Table 59: Proportion of cases of myeloid neoplasms and acute leukemia according to WHO classification, by age and gender, US, 2015
208 Table 60: Estimated case numbers of myeloid neoplasms and acute leukemia according to WHO classification, by age and gender, US, 2015
219 Table 61: Key marketed drugs for acute myeloid leukemia
221 Table 62: Dacogen drug profile
222 Table 63: Dacogen pivotal trial data in acute myeloid leukemia
228 Table 64: Rydapt drug profile
230 Table 65: Rydapt pivotal trial data in acute myeloid leukemia
236 Table 66: Vidaza drug profile
237 Table 67: Vidaza pivotal trial data in acute myeloid leukemia
245 Table 68: Marketed products and approved indications for acute myeloid leukemia in the US, Japan, and five major EU markets
253 Table 69: Factors influencing the pricing and reimbursement of Vidaza and Dacogen in the US and five major EU markets
259 Table 70: Pricing and reimbursement of mutationally targeted therapies in the US and five major EU markets
264 Table 71: Investigated populations for recently approved and pipeline AML therapies
280 Table 72: The impact of in-patient funding mechanisms on Vyxeos’s use
290 Table 73: US pricing of key intravenous AML drugs
290 Table 74: US pricing of key oral AML drugs
295 Table 75: Formulary placement of marketed AML drugs in selected commercial formularies
299 Table 76: Formulary placement of marketed AML drugs in select Medicare Part D formularies
304 Table 77: Prior authorization criteria for marketed AML drugs in six major health plans
307 Table 78: Pricing premiums given to medicines that can demonstrate benefit over comparators
308 Table 79: Pricing of key marketed intravenous AML drugs in Japan
309 Table 80: Pricing of key AML drugs in the five major EU markets
311 Table 81: Transparency Committee’s ASMR ratings and pricing implications
312 Table 82: Transparency Committee’s SMR ratings and pricing implications
313 Table 83: Transparency Commission’s assessment of AML treatments
324 Table 84: G-BA assessments of key AML drugs
334 Table 85: Reimbursement conditions for AML treatments in Italy
336 Table 86: Italian regional formulary decisions for AML drugs
337 Table 87: AML drug assessments by the Emilia-Romagna region
339 Table 88: AIFA Technical Scientific Committee innovation algorithm
345 Table 89: National and regional reimbursement decisions in Spain
352 Table 90: NICE assessments of key AML therapies
357 Table 91: SMC decisions on key AML therapies
366 Table 92: Exchange rates used for calculating branded drug prices
368 Table 93: Phase II/III pipeline products in development for acute myeloid leukemia
370 Table 94: Phase III clinical trial data for Vidaza, Dacogen, and Mylotarg in acute myeloid leukemia
375 Table 95: ATIR101 drug profile
376 Table 96: ATIR101 late-phase trials in acute myeloid leukemia
378 Table 97: ATIR101 Phase II trial data in acute myeloid leukemia
383 Table 98: IDHIFA drug profile
385 Table 99: IDHIFA Phase III trial in acute myeloid leukemia
387 Table 100: IDHIFA Phase I/II data in acute myeloid leukemia
394 Table 101: Iomab-B drug profile
395 Table 102: Iomab-B late-phase trials in acute myeloid leukemia
401 Table 103: Venclexta drug profile
403 Table 104: Venclexta Phase III trials in acute myeloid leukemia
406 Table 105: Venclexta early phase data in acute myeloid leukemia
413 Table 106: Vyxeos drug profile
415 Table 107: Vyxeos late-phase data in acute myeloid leukemia
423 Table 108: Zeltherva drug profile
425 Table 109: Zeltherva Phase II data
431 Table 110: Gilteritinib drug profile
433 Table 111: Gilteritinib late-phase trials in acute myeloid leukemia
436 Table 112: Gilteritinib Phase I/II data in acute myeloid leukemia
443 Table 113: Guadecitabine drug profile
444 Table 114: Guadecitabine Phase III trial in acute myeloid leukemia
446 Table 115: Guadecitabine Phase II data in acute myeloid leukemia
452 Table 116: Idasanutlin drug profile
453 Table 117: Idasanutlin Phase III trial in acute myeloid leukemia
454 Table 118: Idasanutlin Phase I/Ib data in acute myeloid leukemia
460 Table 119: ivosidenib drug profile
461 Table 120: ivosidenib clinical trials in acute myeloid leukemia
463 Table 121: ivosidenib Phase I data in acute myeloid leukemia
470 Table 122: Oral Vidaza drug profile
471 Table 123: Oral azacitidine Phase III trial in acute myeloid leukemia
478 Table 124: quizartinib drug profile
480 Table 125: quizartinib Phase III trials in acute myeloid leukemia
482 Table 126: quizartinib Phase II data in acute myeloid leukemia