Applying a range of “highly targeted, yet complementary, push and pull delinking mechanisms” at key stages of the biopharmaceutical R&D process is the best way to tackle systemic gaps in the research and development model for neglected and tropical diseases (NTDs) and specific Type II diseases such as malaria and tuberculosis, a new report suggests.
The gaps identified in the Pugatch Consilium study commissioned by the International Federation of Pharmaceutical Manufacturers and Associations include insufficient commitment to basic research efforts targeting diseases of the developing world; inadequate financial and commercial incentives for further investment in these diseases during the applied stages of R&D; and the possibility that even drugs emerging the pipeline will prove too costly for populations in developing countries.
Hence efforts by global health policymakers to uncouple the cost of R&D from the price of medicines through mechanisms such as open databases, research grants and advanced market commitments.
No ‘one size fits all’
But there is there is no ‘one size fits all’ approach, stresses Meir Perez Pugatch, lead author of the report on Assembling the pharmaceutical R&D puzzle for needs in the developing world.
“Addressing R&D needs in the developing world is complex, and solutions differ from one setting to another,” Pugatch comments
“Our study accounts for these complexities and concludes that increasing R&D is best achieved with a combination of mechanisms …The upcoming World Health Assembly will discuss how to better leverage resources to stimulate further R&D and address key funding and capacity gaps in these disease areas.”
Principles are sound
The underlying principles behind the existing biopharmaceutical R&D model “remain sound”, even if the model is in “a process of evolution to fit new conditions, demands and capabilities – economic, social, scientific and structural”, the report says.
Those principles include robust scientific and technological capabilities and infrastructure; facilitative regulatory and clinical environments; effective exclusivity periods derived from intellectual property rights; and market incentives for the launch of both innovative and generic products.
The Pugatch Consilium study proposes a ‘Blueprint for Success’ in evaluating the available mechanisms for incentivising R&D into NTDS and specific Type 2 diseases. These success factors are:• Accurate identification and definition of systemic gaps in the R&D process
• Mitigation of the costs and risks of relevant R&D
• Leveraging partners’ capabilities to translate research into clinical outcomes
• The sustainability of R&D funding for specific disease areas
• Effective access to end-products
• Compatibility with other mechanisms
Delinking models operate at different points in the pharmaceutical innovation process, including research and discovery, preclinical and clinical R&D, and post-marketing/delivery, the report notes.
It provides an overview of the following key mechanisms:
• Open databases or compound libraries: giving other R&D actors access to proprietary databases of technologies and know-how to facilitate drug discovery.
• R&D grants: additional funding in advance of R&D aimed at specific research outcomes.
• R&D prizes: payments to R&D entities in lieu of sales, conditional on achieving a particular outcome.
• Targeted R&D tax credits: a direct contribution to research entities to promote R&D in specific research areas by increasing financial returns in those areas.
• Orphan drug-like schemes: a combination of additional market exclusivity, tax credits, accelerated market authorisation and other funding support to incentivise product development and marketing
• Patent pools: platforms for cross-licensing of intellectual property for use in R&D.
• Product development partnerships: public-private partnerships involving a combination of grant funding and R&D partnerships focused on product development.
• Advanced market commitments: agreements to develop and supply a product in exchange for a temporary purchase guarantee.
• R&D treaties: international agreements to increase funding commitments targeted at open innovation and delinking mechanisms for R&D into NTDs and specific Type II diseases.
Using the Blueprint criteria, as well as existing evidence on delinking mechanisms, to make a preliminary assessment of these strategies, the researchers conclude that “certain mechanisms, most notably prizes and patent pools, may not be as effective as suggested, particularly compared to other mechanisms analysed in the report”.
“Specifically, open compound databases, R&D grants, product development partnerships and advanced market commitments have demonstrated a success in stimulating significant R&D activities in various stages,” they add.
According to director general Eduardo Pisani, IFPMA “aims to contribute to this important policy debate through an evidence-based approach”.