Drug Discovery and Development in Emerging Markets: A Way to More Affordable Cancer Treatments?

Drug Discovery and Development in Emerging Markets: A Way to More Affordable Cancer Treatments?

Gilberto Lopes, MD, FASCO, MBA

Mar 17, 2014

“Innovation distinguishes between a leader and a follower.”—Steve Jobs

Frugal innovation is about turning constraints into opportunities, integrating new ways of thinking about a problem with potentially lower costs of discovery, development, and production in and for low- and middle-income countries. As an example, Embrace Global, a social enterprise based in India, developed a baby warmer that costs $200, approximately 1% of what usual models sell for in the United States and Europe. With capitalized expenditures to bring a new drug to market reaching an all-time high at around $2 billion, such an approach is sorely needed if we want to have more affordable anticancer medications in the future in emerging markets.

As an example of potential drug discovery and development outside of the US-Europe-Japan axis, we have recently published our experience developing a new compound, acetyltanshinone A (ATA), identified through high throughput screening of natural products used in traditional Chinese medicine. This modified molecule has activity against estrogen receptor positive breast cancer cells in vitro and in vivo—with a novel mechanism and more potency than tamoxifen—and we hope to move it forward to human trials once safety studies are completed. [1] Many other colleagues are searching for new drugs in Asia and Latin America.

Indeed, a number of pharmaceutical companies have started to develop drugs that are not intended, at least initially, to be sold in high-income economies. For example, BetaPharma (Branford, CT), in combination with Zhejiang Beta Pharma (Beijing, China) has developed icotinib, an EGFR inhibitor that is non-in­ferior to gefitinib in the treatment of patients with lung cancer who have failed to respond to palliative chemo­therapy. As drug development is 10 to 40 times more expensive in the United States and Europe, as compared to China (where costs of less than $1 million, $2 to $3 million, and $3 to $7 million for phase I, II, and III trials, respec­tively, have been reported), icotinib is a cheaper alternative to erlotinib and gefitinib. Icotinib costs approxi­mately 30% less than gefinitib. Since the Chinese Food and Drug Administration (SFDA) approved its marketing in June 2011, tens of thousands of patients have received the drug, with sales expected to reach $158 million annually. The Chinese Ministry of Health is currently considering adding ico­tinib to its basic drug list, and the companies plan to apply for marketing approval in the United States in the future as well. [2]

Another example is Nanoxel, a nanoparticle-based paclitaxel formulation developed by Dabur Pharma, an Indian outfit that has since been acquired by the German health care company Fresenius Kabi. Based on the results of a small randomized controlled trial, the Indian drug regulatory authority approved its use as an alterna­tive to cremophor-based paclitaxel in 2006. The drug is currently available in several countries in Asia and Latin America; and, in India, Nanoxel is cheaper than branded paclitaxel (for example, Taxol, which is manufactured by Bristol–Myers Squibb). As it does not require premedica­tion (for example, to prevent nausea) and is not associ­ated with infusion reactions (based on our review of 562 infusions), we estimated that Nanoxel is $400 cheaper to administer per cycle than paclitaxel. Despite these findings, the lack of adequately sized randomized controlled trials in other countries comparing Nanoxel to paclitaxel and nab-paclitaxel (the albumin-bound form of the drug) has hindered its approval in other markets and our knowledge of its true potential benefits or shortcomings. [3]

A final example is that of nimotuzumab, an anti-EGFR monoclonal antibody, which was initially developed at the Center for Molecular Immunology in Havana, Cuba. It is approved for use and is currently marketed in several countries in Latin America and Southeast Asia, based on early randomized clinical trials showing potential benefits in head-and-neck cancer and gliomas.

Although developments such as these could go some way to driving down the costs of medications, several caveats must be raised. First, scrutiny of new medications in low- and middle-income countries seems to be less rigorous than in the United States, Europe, and Japan, raising the possibility of safety and efficacy concerns. For example, clinical trials that led to the approval of Nanoxel in India have not yet been published in peer-reviewed journals. Secondly, as compa­nies take these drugs to larger and more lucrative markets in high-income countries, their development costs will likely increase in the future. Finally, it is unlikely that a significant number of new drugs will come through this route because pharmaceutical and biotech companies in developed nations are still more likely to generate the largest number of clinically significant new compounds.

One can hope, however, that low- and middle-income countries will search for frugal innovation in drug discovery and development as well and that this might become one of many ways to increase access to cancer medications in resource-constrained and other settings.


1.   Yu T, Zhou Z, Mu Y, de Lima Lopes G, Luo KQ. A novel anti-cancer agent, acetyltanshinone IIA, inhibits oestrogen receptor positive breast cancer cell growth by down-regulating the oestrogen receptor. Cancer Lett. 2014 Apr 28; 346(1):94-103

2.   Lopes G, de Souza JA, Barrios C. Access to cancer medications in low- and middle-income countries. Nat Rev Clin Oncol. 2013 Jun;10(6):314-22

3.   Ranade AA, Joshi DA, Phadke GK, Patil PP, Kasbekar RB, Apte TG, Dasare RR, Mengde SD, Parikh PM, Bhattacharyya GS, Lopes GL. Clinical and economic implications of the use of nanoparticle paclitaxel (Nanoxel) in India. Ann Oncol. 2013 Sep;24 Suppl 5:v6-12



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