Fellows research: Generic anti-cancer drug arsenic trioxide helps cut costs of treating a blood cancer

12 Feb 2020

Fellows research: Generic anti-cancer drug arsenic trioxide helps cut costs of treating a blood cancer

Dr Vikram Mathews. Senior Fellow, Christian Medical College. Vellore

In our recently published work, we report the cost effectiveness of treating acute promyelocytic leukaemia using generic arsenic trioxide, as compared to conventional chemotherapy.

Acute promyelocytic leukaemia (APL) is a distinct subtype of acute myeloid leukaemia (AML) and makes up 10–12% of AML cases. Once a disease with dismal prognosis, APL now shows excellent cure rates (>80% five-year overall survival) with arsenic trioxide (ATO). ATO-based regimens, thus, have become the standard of care and replaced the more intensive chemotherapy-based strategy. However, the cost of patented ATO is prohibitive, and remains a major hurdle in widespread use of ATO for treatment of APL.

Arsenic and its derivatives have been used in medicine for thousands of years. The first clinical use of ATO in APL was reported in the Chinese literature, but its production was patented in the USA after successful replication of its efficacy data in the western world. This increased the cost of ATO to $550 per 10 mg dose. As a result, the cost of ATO is now the major driver of cost of treatment of APL with ATO based regimens.

At our center, ATO has been used since 1998 to treat APL even prior to the application of the patent. Initially, it was manufactured in the pharmacy of the author’s hospital but later transferred to industry for generic manufacture of ATO. Concerns with generic ATO include breach of intellectual property rights and the quality of the product. Experts have argued that neither of these are valid though, as generic ATO has been used in several centers before the patent, including that of the authors, with equivalent reported clinical outcomes.

Given the potential of ‘generic’ ATO in increasing the access and reducing the cost of APL treatment in both developed and low- and middle-income (LMICs) countries, we undertook a study to systematically evaluate its cost effectiveness and compare it with alternative chemotherapy based regimen. The aims of this study were (i) to estimate the total healthcare costs and health resource utilization (HRU) for APL patients treated using generic ATO, and (ii) to evaluate the cost effectiveness of generic ATO in comparison to a chemotherapy-based regimen.

Fig 1: Cost flow during different phases of treatment of acute promyelocytic leukaemia

In this study we used a bottom-up activity-based costing method to estimate the cost of treatment of APL patients using the ATO-based regimen. The results demonstrate that the primary driver of the costs in APL treated with ATO was blood product administration (30%of the treatment cost). In contrast, the major costs in APL treated with conventional chemotherapy were chemotherapy administration (42%) and management of infectious complications (38%) related to prolonged myelosuppression.

The ATO-based regimen reduced the cost incurred for inpatient administration of chemotherapy during induction, consolidation for newly diagnosed APL and during reduction for relapsed cases. This is important for LMICs where inpatient facilities are often limited in terms of population-to-bed ratio. Finally, the Markov analysis using the survival data on generic ATO estimated a significantly lower five-year cost for treating APL patients using generic ATO.

In conclusion, our study shows that the generic ATO based regimen without chemotherapy is the most cost-effective way to deliver treatment for patients with APL and probably the best strategy to achieve the goal of universal access to treatment for APL.


Resource utilization and cost effectiveness of treating acute promyelocytic leukaemia using generic arsenic trioxide. Aniket Bankar, Anu Korula, Uday P. Kulkarni, Anup J. Devasia, Fouzia NA, Sharon Lionel, Aby Abraham, Poonkuzhali Balasubramanian, Nancy Beryl Janet, Sukesh C. Nair, Sezlian S, Visali Jeyaseelan, Jeyaseelan N, Jasmine Prasad, Biju George, Vikram Mathews. Br J Haematol. 2019 Dec 21. doi: 10.1111/bjh.16343