Abstract Number: PB0678
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Novel Biotherapeutics in Hemophilia
Background: Treatment of people with haemophilia A and inhibitors (PwHAi) has improved considerably in Brazil since the introduction of immune tolerance induction (ITI) in 2012. Recently, emicizumab (EMI) was incorporated by the Brazilian Public Healthcare System (SUS) for prophylaxis of PwHAi who failed ITI.
Aims: We aimed to compare the costs and the outcomes of the Brazilian ITI Protocol (BIP) with recombinant factor VIII concentrate (FVIII; α-octocog) and prophylaxis with bypassing agents (BPA; ITI+BPA) or EMI (ITI+EMI), using a decision-three model, from the SUS (payer) perspective.
Methods: The BIP recommends starting ITI at low-dose rFVIII regimen (50IU/kg 3x/week) for all PwHAi and, upon poor response, increasing rFVIII dose to 100IU/kg/day. BPA can be prescribed to treat or prevent bleeding during ITI. Outcomes were success (inhibitor titre <2 BU/mL and FVIII responsiveness) and failure. The success rate of the BIP was 71%. In this analysis, the price of EMI was considered as the amount paid by the Ministry of Health in the last purchase, which is almost twice as high as the price proposed by the industry for incorporation.
| Parameter | ITI+EMI | ITI+BPA |
|---|---|---|
| Age and mean body weight at ITI start | 2 years/15 kg | 2 years/15 kg |
| Prophylaxis regimen | EMI attack 3.0 mg/kg/wk for 4 weeks and maintenance 1.5 mg/kg/wk | aPCC (50%) 75 U/kg q48h or rFVIIa (50%) 90 µg/kg q24h |
| Mean total treated bleedings (success/failure) | 0.54/0.93 | 6/16 |
| Episodic regimen | rFVIIa 180 µg/kg/bleeding | aPCC (for those on aPCC prophylaxis) 262.5 U/kg/bleeding or rFVIIa (for those on rFVIIa prophylaxis) 180 µg/kg/bleeding |
| Need of increase of rFVIII regimen (success/failure) | 14%/74% | 14%/74% |
| Proportion of the duration of each rFVIII regimen, when increase is needed (low-dose:high-dose) | 50%:50% | 50%:50% |
| Mean ITI duration (success/failure) | 1.8 year/3.1 years | 1.8 year/3.1 years |
| Success rate | 71% | 71% |
| Products costs (US$/unit)rFVIII cost | 0.21/IU (rFVIII); 0.46/U (aPCC); 0.37/µg (rFVIIa); 64.84/mg (EMI purchase); 30.99/mg (EMI incorporation) | 0.21/IU (rFVIII); 0.46/U (aPCC); 0.37/µg (rFVIIa); 64.84/mg (EMI purchase); 30.99/mg (EMI incorporation) |
Table 1. Model assumptions. rFVIIa, activated recombinant factor VII; EMI, emicizumab; ITI, immune tolerance induction; aPCC, partially activated prothrombine complex concentrate; rFVIII, recombinant factor VIII, α-octocog.
Results: In the cost-effectiveness analysis, the costs were US$414,773.84 (ITI+BPA) and US$243,244.25 (ITI+EMI), generating an additional cost of US$171,529.59 for PwHAi receiving ITI. ITI+BPA resulted in 8.25 bleeds over ITI+EMI, and each additional bleed cost, US$20,799.28. By deterministic sensitivity analysis, the most impacting variable to the incremental cost was the EMI price: if the incorporation proposed price was used, the savings generated using EMI could reach US$254,319.20 for each PwHAi on ITI.
| Treatment | Cost (US$) | Incremental cost (US$) | Total treated bleedings | Incremental treated bleedings | Incremental cost per treated bleeding (US$) |
|---|---|---|---|---|---|
| Current EMI price (US$ 64.84/mg) | |||||
| ITI+EMI | 243,244.25 | 0.65 | |||
| ITI+BPA | 414,773.84 | 171,529.59 | 8.9 | 8.25 | 20,799.28 |
| Previously proposed EMI price (US$ 30.99/mg) | |||||
| ITI+EMI | 160,454.64 | 0.65 | |||
| ITI+BPA | 414,773.84 | 254,319.20 | 8.9 | 8.25 | 30,838.16 |
Table 2. Cost-effectiveness analysis of the Brazilian Immune Tolerance Induction Protocol using α-octocog and prophylaxis with bypassing agents or emicizumab. BPA, bypassing agent; EMI, emicizumab; ITI, immune tolerance induction.
Conclusions: In conclusion, our model showed ITI+BPA is more expensive and people in this arm are more likely to experience bleeding than ITI+EMI. The inclusion of EMI in the BIP can reduce costs for the SUS and bleedings among PwHAi.
To cite this abstract in AMA style:
Camelo RM, Rezende SM, Álvares-Teodoro J. Cost-effectiveness Analysis of the Brazilian Immune Tolerance Induction Protocol with Recombinant Factor VIII and Prophylaxis with Bypassing Agents or Emicizumab [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/cost-effectiveness-analysis-of-the-brazilian-immune-tolerance-induction-protocol-with-recombinant-factor-viii-and-prophylaxis-with-bypassing-agents-or-emicizumab/. Accessed December 11, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/cost-effectiveness-analysis-of-the-brazilian-immune-tolerance-induction-protocol-with-recombinant-factor-viii-and-prophylaxis-with-bypassing-agents-or-emicizumab/