Frequently Asked Questions
35. What are the current patient treatment needs?
Two immediate needs exist. First, a pediatric strength treatment which is affordable, age-adapted, safe, and efficacious. Second, a new drug for the chronic stages of the disease that would ideally also be effective in every stage.
36. What is DNDi doing to address unmet treatment needs?
In the short term, DNDi and LAFEPE are working together to develop a pediatric formulation of benznidazole, which will be available in 2010. DNDi is also working on clinical development of azoles and other combinations.
In the long term, DNDi is focusing on new drugs and improved research & treatment capacities. By 2014, DNDi aims to make available one new pediatric strength treatment and have one new drug registered from its Chagas-specific portfolio..
37. What is DNDi's role in the R&D for Chagas disease?
DNDi is currently the only product development partnership with a research agenda for Chagas disease. The absence of a profitable market for Chagas drugs necessitates the development of innovative mechanisms to stimulate and finance R&D, as well as serious political commitments to foster an environment that encourages and pushes research innovation for Chagas.
38. How much is invested in R&D for new drugs to treat Chagas disease?
Of the neglected diseases, Chagas disease is among the diseases that receive the least investment for R&D. In 2007 only about US$10 million of the US$2,560 million R&D investments for neglected diseases went to Chagas disease.
39. To which phase of research is the majority of funding allocated?
Of the approximately US$10 million in R&D investments in Chagas disease in 2007, only 10% was allocated to drug development.
40. What is the reason for the lack of R&D on treatment and diagnostics?
The main reason for lack of R&D is the lack of funds. Some research laboratories might have good initiative but most of them are stymied because funding is limited.
The second factor is the poor access to diagnosis and treatment. National control programs tend to focus on screening for congenital and blood transfusion transmission, rather than diagnosis and treatment.
41. How can R&D be boosted?
Governments can do much to change the current landscape, to ensure international cooperation between public and private actors, and to mobilize tremendous resources for medical research and development .
42. How difficult is it to find partnerships?
The key factor to find partnerships is to be able to bring together individual, public, and private actors, regardless of their different motivations. One of the main principles in these partnerships is to share a common needs-driven objective with the actors from these different spheres. To this end, DNDi's partnerships with donors, including governments, international organizations, non-governmental organizations, private foundations and individuals, play a major role. Today DNDi manages 250 research, technical, and funding agreements with both public and private partners across all diseases that we work with. However, the successful conclusion of these agreements also depends on solving critical issues, such as licensing rights, confidentiality, intellectual property rights management, and lack of profitability.
43. How does a new drug get registered?
There is no international regulation regarding the registration of drugs and therefore it differs from country to country. In general, it is a long and complex process, involving elaborate documentaion and compliance to stringent quality standards. Adoption of a new drug can be a very slow prcess.
44. What has to be done to make the new drugs accessible to the most neglected patients?
The first step for patients to access treatment requires identifying and reporting the Chagas disease cases, critical today as actual cases are significantly under-reported. In addition, there needs to be consensus on treatment guidelines, training of health workers, and finally, the political will to address the problem.
In parallel, it is essential to ensure a continuous, affordable supply of drugs. This requires agreements from drug manufacturers whereby they commit to manufacture the product over the long term and sell it at an affordable price. In order to ensure affordability, maufacturers must be able to obtain active pharmaceutical ingredients at the lowest cost possible. In addition, medicines need to be distributed via a reliable supply chain to health centers that may be in very remote, rural areas.
In the mid to long term, it will be important to monitor treatment safety and efficacy in large Phase IV trials, and to update treatment guidelines accordingly.
