Human African trypanosomiasis, Chagas disease, Buruli ulcer and many other conditions that cause death, disability and economic destitution for millions of people across the developing world don’t attract the research effort their public health impact suggests they deserve.
Last year, a Lancet article by Chirac and Torreele reported that only 1.3% of new drugs developed over the past 30 years were for neglected tropical diseases and tuberculosis. Yet these conditions account for 12% of the disease burden worldwide. And even if pharma and biotech companies develop vaccines and medicines, developing countries may not be able to afford them - as the acrimonious row over funding of medicines for HIV and AIDS illustrated. The problem taints the sector’s reputation and raises questions over its ethics.
In the March issue of Nature Medicine, Carl Nathan, Chairman of the Department of Microbiology and Immunology, and R.A. Rees Pritchett Professor of Microbiology at Weill Cornell Medical College in New York City, suggests that solving these problems may involve developing ‘open access’ drug companies and restructuring the patent system.
Fee for service within pharma
Nathan envisages open-access drug companies as “fee-for-service sites” within pharma organisations that facilitate collaborations between academics and biotechnology and pharmaceutical companies. Users and governments would fund the collaboration. Nathan believes that the approach will bring “new ideas and expertise” to drug development that is independent of market forces.
The first seeds may have been sown. There are now, Nathan notes, around 24 public-private partnerships (PPPs) that manage around $900 million donated by philanthropists and approximately $244 million. PPPs enter contracts with biotechnology and pharmaceutical companies to perform various aspects of development. This approach resulted in more than 47 potential products for diseases encountered almost solely in developing countries. Unfortunately, Nathan comments, “philanthropy lacks the means to carry this load indefinitely”. He suggests that “Open-access drug companies funded by users and government could institutionalize and improve the best features of PPPs”.
Pfizer sharing 12,000 medicines
Some companies (including GlaxoSmithKline, AstraZeneca and Novartis) have established up R&D units dedicated to diseases that affect the developing world solely or predominately. Pfizer, Nathan notes, is sharing 12,000 compounds with scientists affiliated with the WHO’s Special Program for Research and Training in Tropical Diseases (TDR). Scientists from TDR and Pfizer work at one of the company’s sites to develop lead compounds. At least 13 other companies provide compounds to TDR and several offer medicinal chemistry and pharmacokinetic services.
Nathan also suggests splitting patents into two tracks, which would run concurrently. Governments of developed and developing countries would contribute to a fund over many years, which would eventually need to be tens of billions of dollars annually. The fund would reward the patent owners in proportion to the impact on the global burden of disease. The payments could last longer than the 20-year patent monopoly, which may attract investors and, as medicines would need to compete for the payments, would encourage continued innovation.
This approach running along the current patent system would, Nathan argues, result in drug prices close to the production costs. The new track would also encourage governments and businesses to ensure as many people as possible receive the drug. It may even help some people in developed countries. Nathan notes that despite the spread of drug resistance, there is insufficient investment in antibiotic R&D, partly because of a convergence between “monopoly-based drug development and the perception of relatively small markets."
Nathan comments that several issues remain to be resolved including the extent of government’s financial contribution and the mechanisms to collect reliable information about changes in the burden of disease and attribute any improvement to a particular product. Furthermore, the ability to conduct clinical trials in less developed countries may need to be enhanced, while governments may need to overcome variation in regulatory requirements. However, as Nathan concludes: “Such challenges are manageable, in contrast to the calamitous consequences of the present course."
Reference: Nathan C Aligning pharmaceutical innovation with medical need Nat Med 2007;13:304-8 doi:10.1038/nm0307-304