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Cell & Gene Therapy

The challenge - and opportunity - of gene therapies in emerging markets

Posted by on 18 November 2019
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By Jeremy Schafer, PharmD, MBA, SVP, Precision for Value

In October, I was fortunate enough to speak at a conference in Brasilia[1], with the goal of beginning the conversation on how to bring gene and cell therapies to Brazil, where currently none are approved. The challenges in providing access to these remarkable products were discussed —challenges uniquely Brazilian, those common to emerging markets, and other struggles that all nations face. With approximately 300 gene therapy products in the pipeline, time is of the essence to overcome these barriers so that patients the world over have an opportunity for cure.[2]

Challenges likely to impact all emerging markets fell into several buckets:

  1. Financial: Gene therapies have come to the US and European markets with prices in the hundreds of thousands and even millions of dollars. For emerging nations with smaller economies and limited healthcare budgets, such prices are daunting.
  2. Structural: An issue facing emerging markets is actually delivering the treatment to patients. Brazil for example is the world’s fifth largest nation by landmass and has over 200 million citizens. Healthcare services and practitioners are not equally distributed, with shortages in rural areas and specialists generally concentrated in the private sector.[3] Other emerging markets will likely face the same challenge of bringing care to poor or rural populations where proper healthcare facilities and qualified providers may be in short supply.
  3. Educational: An issue in all nations is the level of healthcare literacy among the population. This issue is particularly acute in emerging markets where education and technological solutions may be limited in certain areas. Developing and delivering information on gene therapy to populations that may have limited education and potential distrust of healthcare services is a barrier to care.

Progress is being made on the financial front. New payment solutions for gene therapy have been proposed and are being tested in the United States and in Europe. Innovative payment models being used include outcomes based, which either decreases the payment or refunds it in the event of subpar efficacy; annuity, which breaks the payment into installments over time; and creation of a separate fund to pay for gene therapy.[4] Nations in Europe have leveraged outcomes-based agreements and payment over time to provide access to CAR-T therapies. In the US market, products like Zolgensma for spinal muscular atrophy have offered similar arrangements to payers.[5] For emerging markets, a payment solution that combines an outcomes-based arrangement with payment over time may help ensure that countries with limited healthcare resources only pay when the product is efficacious, as well as in installments that will not overwhelm the nation’s budget. In addition, creating a dedicated gene therapy fund could help emerging nations plan for gene therapy expenses and could even be done in cooperation among several nations in a geographic region to both share risk and negotiate as a group on cost.

The structural and educational challenges can only be surmounted by continued investment in healthcare infrastructure and outreach programs. The recent announcement of a $200 million collaboration between the NIH and the Gates Foundation to develop and bring gene therapies for HIV and sickle cell disease to impoverished nations underscores the challenge.[6] Critics have pointed out that generic antiretroviral therapy for HIV and hydroxyurea for sickle cell are significantly underutilized in many African nations due to limited healthcare infrastructure and inadequate patient education.[6] The development of gene therapies will not cure patients if the drugs cannot reach them. Thankfully, the partnership’s focus on “access, scalability, and affordability” is meant to help address structural and educational barriers. More collaborations like NIH-Gates are needed and should explore unique ways to deliver care in emerging markets—including how to bring the patient to the drug versus the other way around.

The future of the gene therapy market will depend on several factors, including durability of drug response, long-term drug safety, and affordable returns on investment. For the gene therapy market to grow, manufacturers, governments, and private payers should continue to collaborate on new ways to finance gene therapies, as well as how to deliver these drugs in the most cost-effective manner. Emerging markets will benefit from this innovation in payment options and healthcare delivery. In particular, conferences and publications on lessons learned in reimbursing and delivering gene therapy in nations of varying economic and healthcare levels will be valuable and ensure that best practices can be developed and shared around the world.


  1. Gene and Advanced Therapy: Creating Value in the Patient Journey and in the Health System. October 23-24, 2019. Brasilia, Brazil.
  2. Powaleny A. New report shows nearly 300 cell and gene therapies in development. PhRMA. Published December 6, 2018. Accessed November 5, 2019.
  3. Massuda A et al. The Brazilian health system at crossroads: progress, crisis and resilience. BMJ Glob Health. 2018;3:e000829. doi:10.1136/ bmjgh-2018-000829.
  4. Cassidy B. How will we pay for the coming generation of potentially curative gene therapies? STAT. Published June 12, 2019. Accessed November 5, 2019.
  5. Novartis. AveXis announces innovative Zolgensma® gene therapy access programs for US payers and families. Published May 24, 2019. Accessed November 5, 2019.
  6. Begley S. NIH and Gates Foundation launch effort to bring genetic cures for HIV, sickle cell disease to world’s poor. STAT. Published October 23, 2019. Accessed November 5, 2019.
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