Budget impact analysis for avatrombopag in the treatment of chronic primary immune thrombocytopenia in adult patients refractory to other treatments

Figures & data

Figure 1. Budget impact model structure.

Note: Legend: TPO-RAs: Thrombopoietin receptor agonists

Table 1. Target population^*†.

	Year 1–3	Reference
Italian population
Adult population (≥18 years) – A	49,796,335	ISTAT
Prevalent PTS
Prevalence of chronic ITP – B	23.6/100,000 adults	Feudjo-Tepie 2008
pITP (%) – C	80.0%	Lambert 2017
Adult Prevalent PTS with chronic pITP (n) – D = A*B*C	9,402	Estimate
Prevalent PTS with chronic pITP who have required treatment to stop the bleeding (%) – E	71.0%	Palandri 2016
Prevalent PTS with chronic pITP who have required treatment to stop the bleeding (n) – F = D*E	6,675	Estimate
Prevalent PTS with chronic pITP who have required a treatment after 1^st line (%) – G	55.2%	Palandri 2016
Prevalent PTS with chronic pITP who have required a treatment after 1^st line (n) – H = F*G	3,685	Estimate
Prevalent PTS with chronic pITP treated with TPO-RAs after 1^st line (%) – I	38.3%	IQVIA Market Research
Prevalent PTS with chronic pITP treated with TPO-RAs after 1^st line (n) J = H*I	1,411	Estimate
Incident PTS
Incident of ITP (%) – K	3.3/100,000 adults	Rodeghiero 2020
pITP (%) – L	80%	Lambert 2017
Incident PTS with pITP (n) – M = A*K*L	1,314	Estimate
Incident PTS with pITP who develop a chronic form (%) – N	80.0%	Kistanguri 2013
Incident PTS with pITP who develop a chronic form (n) – O = M*N	1,051	Estimate
Incident PTS with chronic pITP who have required treatment to stop the bleeding (%) – P	71.0%	Palandri 2016
Incident PTS with chronic pITP who have required treatment to stop the bleeding (n) – Q = O*P	746	Estimate
Incident PTS with chronic pITP who have required treatment after 1^st line (%) – R	55.2%	Palandri 2016
Incident PTS with chronic pITP who have required treatment after 1^st line (n) – S = Q*R	412	Estimate
Incident PTS with chronic pITP treated with TPO-RAs after 1^st line (%) – T	80.0%	Expert Opinion
Incident PTS with chronic pITP treated with TPO-RAs after 1^st line (n) – U = S*T	330	Estimate
Target population
Total prevalent and incident PTS with chronic pITP treated with TPO-RAs after 1^st line (n) -V = J + U	1,741	Estimate

Legend: ITP: immune thrombocytopenia; pITP: primary immune thrombocytopenia; PTS: patients; n: number; TPO-RAs: Thrombopoietin receptor agonists; Expert Opinion: all the hypotheses used in the analysis were validated by two Italian clinical experts and one health economic expert.

*Number of patients rounded at the first integer. ^†Percentages rounded to the first decimal number.

Table 2. Estimated patients and market shares broken down by year and therapy: base case and sensitivity analysis ^*†.

	Year 1–2023	Year 2–2024	Year 3–2025
Reference Scenario
Avatrombopag – Total	0 (0.0%)	0 (0.0%)	0 (0.0%)
Of which 1^st year treatment	0 (0.0%)	0 (0.0%)	0 (0.0%)
Of which ≥ 2^nd year treatment	0 (0.0%)	0 (0.0%)	0 (0.0%)
Eltrombopag – Total	1,137 (65.3%)	1,137 (65.3%)	1,137 (65.3%)
Of which 1^st year treatment	216 (12.4%)	216 (12.4%)	216 (12.4%)
Of which ≥ 2^nd year treatment	921 (52.9%)	921 (52.9%)	921 (52.9%)
Romiplostim – Total	604 (34.7%)	604 (34.7%)	604 (34.7%)
Of which 1^st year treatment	114 (6.5%)	114 (6.5%)	114 (6.5%)
Of which ≥ 2^nd year treatment	490 (28.1%)	490 (28.1%)	490 (28.1%)
TPO-RAs – Total	1,741 (100.0%)	1,741 (100.0%)	1,741 (100.0%)
Of which 1^st year treatment	330 (19.0%)	330 (19.0%)	330 (18.9%)
Of which ≥ 2^nd year treatment	1,411 (81.0%)	1,411 (81.0%)	1,411 (81.1%)
Alternative Scenario
Avatrombopag – Total	212 (12.2%)	326 (18.7%)	463 (26.6%%)
Of which 1^st year treatment	212 (12.2%)	114 (6.5%)	137 (7.9%)
Of which ≥ 2^nd year treatment	0 (0.0%)	212 (12.2%)	326 (18.7%)
Eltrombopag – Total	1,024 (58.8%)	957 (55.0%)	871 (50.0%)
Of which 1^st year treatment	194 (11.1%)	181 (10.4%)	165 (9.5%)
Of which ≥ 2^nd year treatment	830 (47.7%)	776 (44.6%)	706 (40.5%)
Romiplostim – Total	505 (29.0%)	458 (26.3%)	407 (23.4%)
Of which 1^st year treatment	96 (5.5%)	87 (5.0%)	77 (4.4%)
Of which ≥ 2^nd year treatment	409 (23.5%)	371 (21.3%)	330 (19.0%)
TPO-RAs – Total	1,741 (100.0%)	1,741 (100.0%)	1,741 (100.0%)
Of which 1^st year treatment	502 (28.8%)	382 (21.9%)	379 (21.7%)
Of which ≥ 2^nd year treatment	1,239 (71.2%)	1,359 (78.1%)	1,362 (78.3%)

Legend: TPO-RAs: Thrombopoietin receptor agonists.

*Number of patients rounded at the first integer. ^†Percentages rounded to the first decimal number.

Table 3. Monitoring costs per patient.

		Unit Costs
Year	Visit OR exams n	Specialistic Visit €	Platelet Counts €	Liver Function €	venous blood collection €	Biochemical Analyses (only for the 1^st visit) €	Total Monitoring €
Avatrombopag
Year 1	16	€20.66	€3.17	€0.00	€2.58	€172.70	€598.71
Year ≥2	12	€20.66	€3.17	€0.00	€2.58	€0.00	€316.92
Eltrombopag
Year 1	16	€20.66	€3.17	€3.45	€2.58	€172.70	€650.46
Year ≥2	12	€20.66	€3.17	€3.45	€2.58	€0.00	€358.32
Romiplostim
Year 1	20	€20.66	€3.17	€0.00	€2.58	€172.70	€704.35
Year ≥2	12	€20.66	€3.17	€0.00	€2.58	€0.00	€316.92

Table 4. Cost per patient/year: base case and sensitivity analysis.

		Drug Cost
Drug	Year	Pack	Price	Cost €/mg	Dose	Drug cost patient/year €	Monitoring cost	Total Cost
BaseCase Analysis
Avatrombopag	1	30 tabs x 20 mg	€2.466.53	€4.11	17.70 mg daily	€26,558.36	€598.71	€27,157.07
	≥2						€316.92	€26,875.28
Eltrombopag	1	28 tabs x 50 mg	€2,170.51	€1.55	51.30 mg daily	€29,029.80	€650.46	€29,680.26
	≥2						€358.32	€29,388.12
Romiplostim	1	1 vial of 250 mcg	€543.76	€2,175.04	329.75 mcg weekly*	€37,295.25	€704.35	€37,999.60
		1 vial of 500 mcg	€1,087.51	€2,175.02
	≥2	1 vial of 250 mcg	€543.76	€2,175.04			€316.92	€37,612.17
		1 vial of 500 mcg	€1,087.51	€2,175.02
Sensitivity Analysis
Avatrombopag	1	30 × 20 mg tabs	€2.466.53	€4.11	20.00 mg daily	€30,009.45	€598.71	€30,608.16
	≥2						€316.92	€30,326.37
Eltrombopag	1	€2,170.51	€1.55	€1.55	50.00 mg daily	€28,294.15	€650.46	€28,944.61
	≥2						€358.32	€28,652.47
Romiplostim	1	1 vial of 250 mcg	€543.76	€2,175.04	350.00 mcg weekly†	€39,585.73	€704.35	€40,290.08
		1 vial of 500 mcg	€1,087.51	€2,175.02
	≥2	1 vial of 250 mcg	€543.76	€2,175.04			€316.92	€39,902.65
		1 vial of 500 mcg	€1,087.51	€2,175.02

 *68.1% of patients treated with 250 mcg weekly and 31.9% treated with 500 mcg weekly. ^†5 mcg/kg Weekly per an average patient of 70 kg..

Figure 2. Budget Impact and healthcare expenditure variation per year, linked with avatrombopag reimbursement (Alternative – Reference scenario) per year: base case analysis.

Table 5. Budget impact analysis: base case analysis.*

	Year 1–2023	Year 2–2024	Year 3–2025
Reference Scenario
Avatrombopag – Total	€0	€0	€0
Of which drug therapy	€0	€0	€0
Of which monitoring	€0	€0	€0
Eltrombopag – Total	€33,477,390	€33,477,390	€33,477,390
Of which drug therapy	€33,006,878	€33,006,878	€33,006,878
Of which monitoring	€470,512	€470,512	€470,512
Romiplostim – Total	€22,761,918	€ 22761,918	€ 22761,918
Of which drug therapy	€22,526,331	€22,526,331	€22,526,331
Of which monitoring	€235,587	€235,587	€235,587
TPO-RAs – Total	€56,239,308	€56,239,308	€56,239,308
Of which drug therapy	€55,533,209	€55,533,209	€55,533,209
Of which monitoring	€706,099	€706,099	€706,099
Alternative Scenario
Avatrombopag – Total	€5,757,299	€8,793,466	€12,481,861
Of which drug therapy	€5,630,373	€8,658,026	€12,296,522
Of which monitoring	€126,927	€135,440	€185,339
Eltrombopag – Total	€30,150,106	€28,177,304	€25,645,252
Of which drug therapy	€29,726,511	€27,781,515	€25,284,952
Of which monitoring	€423,595	€395,790	€360,300
Romiplostim – Total	€19,031,339	€17,260,080	€15,337,985
Of which drug therapy	€18,834,101	€17,081,225	€15,179,167
Of which monitoring	€197,238	€178,856	€158,819
TPO-RAs – Total	€54,938,744	€54,230,851	€53,465,098
Of which drug therapy	€54,190,985	€53,520,765	€52,760,641
Of which monitoring	€747,759	€710,085	€704,458
Δ Alternative Scenario – Reference Scenario
Total cost per year	-€1,300,564	-€2,008,457	-€2,774,210
Of which drug therapy cost per year	-€1,342,224	-€2,012,444	-€2,772,569
Of which monitoring cost per year	+€41,660	+€3,987	-€1,641
Total cost at 3 years	-€6,083,231

Legend: TPO-RAs: Thrombopoietin receptor agonists.

*Costs rounded at the first integer.

Figure 3. Budget Impact and healthcare expenditure variation per year, linked with avatrombopag reimbursement (Alternative – Reference scenario) per year: sensitivity analysis.

Table 6. Budget impact analysis: sensitivity analysis.*

	Year 1–2023	Year 2–2024	Year 3–2025
Reference Scenario
Avatrombopag – Total	€0	€0	€0
Of which drug therapy	€0	€0	€0
Of which monitoring	€0	€0	€0
Eltrombopag – Total	€32,640,959	€32,640,959	€32,640,959
Of which drug therapy	€32,170,447	€32,170,447	€32,170,447
Of which monitoring	€470,512	€470,512	€470,512
Romiplostim – Total	€24,145,366	€24,145,366	€24,145,366
Of which drug therapy	€23,909,780	€23,909,780	€23,909,780
Of which monitoring	€235,587	€235,587	€235,587
TPO-RAs – Total	€56,786,325	€56,786,325	€56,786,325
Of which drug therapy	€56,080,226	€56,080,226	€56,080,226
Of which monitoring	€706,099	€706,099	€706,099
Alternative Scenario
Avatrombopag – Total	€6,488,930	€9,918,521	€14,079,715
Of which drug therapy	€6,362,003	€9,783,081	€13,894,375
Of which monitoring	€126,927	€135,440	€185,339
Eltrombopag – Total	€29,396,804	€27,473,291	€25,004,504
Of which drug therapy	€28,973,210	€27,077,502	€24,644,205
Of which monitoring	€423,595	€395,790	€360,300
Romiplostim – Total	€20,188,031	€18,309,119	€16,270,210
Of which drug therapy	€19,990,793	€18,130,263	€16,111,391
Of which monitoring	€197,238	€178,856	€158,819
TPO-RAs – Total	€56,073,765	€55,700,931	€55,354,429
Of which drug therapy	€55,326,006	€54,990,846	€54,649,971
Of which monitoring	€747,759	€710,085	€704,458
Δ Alternative Scenario – Reference Scenario
Total cost per year	-€712,560	-€1,085,394	-€1,431,896
Of which drug therapy cost per year	-€754,221	-€1,089,381	-€1,430,255
Of which monitoring cost per year	+€41,660	+€3,987	-€1,641
Total cost at 3 years	-€3,229,850

Legend: TPO-RAs: Thrombopoietin receptor agonists.

*Costs rounded at the first integer.