clinical trial researchers

Significant differences remain between countries in terms of their health policy, cultural beliefs, and medical infrastructure. This is perhaps best elucidated by the myriad of national responses to the COVID-19 pandemic. Some countries, like China, had a zero-COVID policy from the very onset; other countries are more lax about the idea of living with the virus. 

The point here is that a single disease can elicit a variety of responses from different nations. Although there is some consensus on best practices and evidence from clinical studies with regards to a particular disease, the creation and implementation of health policies by different nations are always filtered through the lens of each nation’s governing policy and their perception of how health care is best delivered. 

With regard to hemophilia, the basic facts are indisputable: it is a bleeding disorder caused by the lack of certain clotting factors. The mainstay of treatment today is the exogenous replacement of clotting factors. Experimental therapies, such as genome editing, are still being developed and refined for mass usage. 


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Given that hemophilia is a fairly straightforward disease without many unknowns (compared to other rare diseases, such as idiopathic pulmonary fibrosis), one could be forgiven to assume that most countries have nearly identical treatment protocols for this disease. Tiede and 11 colleagues from around the globe published a paper detailing “international recommendations” for the diagnosis and treatment of acquired hemophilia A, suggesting that a unified approach towards dealing with hemophilia is both possible and desirable.

However, differences between countries stubbornly remain. We will compare the hemophilia response between two Eastern countries: Japan and China. 

Factor Replacement Therapy in Japan 

Statistics show that the severity of hemophilia in Japan and its impact on quality of life is comparable to those in Western countries.

Japan has a universal health coverage system in which patients pay 30% of the medical fees incurred. The remaining fees are paid for by the government or the health insurance program that an employer participates in. Uniquely, hemophilia treatment is not subject to this copayment mechanism, meaning the full medical expenditure is covered by public funds. 

In Japan, factor replacement therapy is the mainstay of treatment for hemophilia. Reiss and Ohmori wrote, “Presently, no gene therapy products using adeno-associated virus (AAV) vectors have been approved in Japan.”

Despite AAV-mediated gene therapy being unavailable for the time being, research in Japan into this mode of treatment continues. In fact, significant discoveries have been reported by studies carried out in Japanese laboratories. For example, Reiss and Ohmori wrote, “Work at Jichi Medical University confirmed that therapeutic FIX levels were maintained for >10 years after AAV vector administration in cynomolgus monkeys.” 

If gene therapy becomes available as a treatment option for hemophilia in the future, there is no guarantee that it will be covered by public funds, since gene therapy drugs incur high medical costs. However, gene therapy may still prove to be more cost-effective in the long term. 

Modernizing Hemophilia Treatment in China 

China’s medical infrastructure has modernized at a dizzying pace over the last few decades. Among its achievements is the setting up of the Hemophilia Treatment Center Collaboration Network of China (HTCCNC), which has sped up the modernizing of hemophilia treatment in the country. 

In terms of medical research, the Tianjin Institute of Hematology and Blood Diseases Hospital has made significant contributions to our collective understanding of hemophilia.

“Several publications . . . have explored aspects of AAV-induced immunogenicity and methods of reducing immune response to AAV vectors,” Reiss and Ohmori wrote. ”When transducing cells with AAV vector, empty vector particles themselves or mixed with full particles were found triggering a stronger immune response than the lack of empty particles.”

Read more about hemophilia treatment 

Gene therapy is also under investigation in China, but bureaucratic hurdles remain significant. The Chinese government allows small, investigator-initiated clinical trials that need to be sponsored by a hospital and registered with local government agencies. This means that oversight into hemophilia research is more rigid than in other countries that have a laissez-faire approach to clinical trials. 

It is estimated that 95% of the Chinese population has access to basic health insurance, although the levels of reimbursement caps and copay requirements vary greatly. At a local government level, health care for hemophilia patients has generated considerable support. However, the question of who pays for future gene therapy treatments remains unresolved. 

Anticipating the Future 

Regardless of the state of hemophilia healthcare around the world, a consensus is building among the scientific community that gene therapy represents the future of hemophilia treatment. Despite its potentially high costs, it is a one-time disease-altering treatment. This means that patients with hemophilia may soon have the chance to live completely normal lives after gene therapy has been administered. 

Lindsey A. George of the Children’s Hospital of Philadelphia in Pennsylvania wrote, “Numerous ongoing rAAV–mediated hemophilia gene transfer clinical trials suggest that the goal of gene therapy to alter the paradigm of hemophilia care may soon be realized.”

References

Tiede A, Collins P, Knoebl P, et al. International recommendations on the diagnosis and treatment of acquired hemophilia A. Haematologica. 2020;105(7):1791-1801. doi:10.3324/haematol.2019.230771

Reiss UM, Zhang L, Ohmori T. Hemophilia gene therapy-new country initiatives. Haemophilia. Published online July 7, 2020. doi:10.1111/hae.14080

George LA. Hemophilia gene therapy comes of age. Hematology Am Soc Hematol Educ Program. Published online December 8, 2017. doi:10.1182/asheducation-2017.1.587