Report provides real and estimated costs of repurposing drugs and new chemical entities, evoking the lessons learned based on alternative pathways and partnerships
At a scientific meeting at Institut Pasteur, France, entitled 'Best Science for the Most Neglected: Where Do We Stand Ten Years On?', co-organized with Institut Pasteur and MSF and in collaboration with PLOS, the Drugs for Neglected Diseases initiative (DNDi) marks its 10-year anniversary by issuing a report that explores the lessons learned from a decade of research and development (R&D) of new treatments for neglected diseases via a cost-effective, innovative, not-for-profit drug development model. The report also comes at the time of discussions at the WHO aiming at gaining Member State agreement on 'demonstration projects' meant to provide evidence for the feasibility and sustainability of collaborative and open approaches to R&D for the health needs of developing countries.
The DNDi report, entitled 'An Innovative Approach to R&D for Neglected Patients: Ten Years of Experience and Lessons Learned by DNDi', provides elements to stimulate current discussions on the way forward for sustainable mechanisms to provide health tools for developing countries. The report describes four key pillars of the open R&D model: patient-centricity; open access to knowledge and patient access to treatments; financial and scientific independence; and building and sustaining solid alliances with public and private partners, including in endemic countries.
DNDi was founded in 2003 to address a systemic lack of R&D for certain neglected diseases. A recent Lancet Global Health publication showed a persistent deficiency in new therapies for neglected diseases, despite some progress over the past decade. From 2000 to 2011, of all new drugs and vaccines approved for all diseases, 4% were for neglected diseases - which represent an 11% health burden - with progress being mainly in reformulating already existing drugs. Of the new drugs, only 1% were for neglected diseases.
'MSF was the initiator of the DNDi experiment and we see concrete results for patients already, which are improving and saving lives in the field today', said Dr Joanne Liu, President of MSF International. 'MSF is proud to renew its commitment, both in terms of funding and collaboration in the field, to the initiative for the years to come.'
In ten years, DNDi has established over 350 collaborations in 43 countries, including with 20 pharmaceutical and biotechnology companies, and with over 50 universities and research institutes. With its partners, DNDi has conducted 25 clinical studies from Phase I until Phase IV implementation / pharmacovigilance studies, while strengthening research capacities in the countries where the diseases occur, notably through the set-up of clinical research platforms. These efforts have led to the implementation of six improved treatments to answer urgent needs of patients with malaria, sleeping sickness, visceral leishmaniasis, and Chagas disease. DNDi has invested significantly in upstream research and access to compound libraries, data, and knowledge sources to identify new chemical entities, 12 of which are currently in pre-clinical and clinical development.
'We are convinced of the relevance and role of Institut Pasteur and its international network in upstream research on neglected diseases and our decade of engagement in DNDi is set to take on a new dimension', said Professor Christian Bréchot, President of Institut Pasteur. 'Fundamental knowledge of these diseases and translational research still have much to contribute to the science applied to the benefit of these patients.'
Since its inception, DNDi has raised, from public and private funding sources, EUR 277 million of the EUR 400 million needed to deliver 11 to 13 new treatments by 2018, according to the DNDi Business Plan. The analysis of the business model, through selected case studies of treatments delivered and new chemical entities underway, offers insight into the DNDi's R&D costs: EUR 12 million to develop and monitor implementation of a fixed-dose combination therapy for malaria (ASAQ - over 250 million treatments distributed by Sanofi); EUR 6.8 million to develop an improved treatment option for sleeping sickness (NECT - now used to treat 96% of all late-stage patients); EUR 11.5 million to develop a new combination therapy for kala-azar in Africa (SSG&PM - 23,000 patients treated in East Africa since 2010); and estimates for development and registration, EUR 38.3 million for a new chemical entity (SCYX-7158) and EUR 26.5 million for a rediscovered new chemical entity (fexinidazole), both oral treatments for sleeping sickness.
Based on its experience, DNDi concludes that its cost of development ranges from EUR 6-20 million for an improved treatment, and EUR 30-40 million for a new chemical entity. However, the usual attrition in the field of R&D for infectious diseases, and the inherent risk of failure, should be taken into account, bringing the cost range of an improved treatment to EUR 10-40 million, and EUR 100-150 million for a new chemical entity. These estimations do not include the in-kind contributions from DNDi's many partners.
'Today, after ten years, we know that when we push the boundaries of innovative business models, we can develop and deliver safe, adapted, and affordable treatments for diseases affecting millions of the patients in neglected disease-endemic countries, who lack access to health technologies because they can't pay', said Dr Bernard Pécoul, Executive Director of DNDi. 'But we have yet to deliver the breakthroughs needed for radical change, and we need to consolidate the successes of the past to create truly sustainable mechanisms, including financing and coordination, for R&D in the field of neglected diseases.'