Combining health and social protection measures to reach the ultra ...

Research resourcescompounds from many different sources even if there is nointerested industry partner and no commercial potential. Thisruns against the oft-stated maxims that only commercialmarket returns can catalyse drug development; and that onlypharmaceutical companies (and perhaps only largemultinational companies) have the requisite experience tomanage the lengthy, complex and expensive process of drugdevelopment.Equally important is that this flexible, modular approachnot only allows but stimulates different (and often farcheaper) models of drug development. For example, byactively pairing small Western companies or academics withCROs and developing country manufacturers, PPPs create aneglected disease pipeline that encourages and allowssmaller-sized groups to participate, and at a substantiallylower cost than the traditional commercial approach. We notethat the anti-malarial synthetic peroxide has moved fromlaboratory into clinical at a cost of less than US$ 12 million;while the new fixed-dose antimalarial, pyronaridineartesunate,is expected to be registered for US$ 20 million orless. The TB Alliance also estimates that its anti-TB drug, PA-824, will cost less than US$ 90 million to complete clinicaldevelopment. All these projects involve academic, publicdomain or small company leads teamed up with contractorsor a developing country manufacturing partner. PPP codevelopmentof multinational drug company leads can alsobe substantially cheaper, since costs of capital (estimated toroughly double the cost of R&D) are largely avoided, andcompanies are able to minimize their risk and financialoutlays – and therefore their final prices. Either or both ofthese alternative approaches may provide interesting lessonsfor other Western diseases where the profit motive is weak orabsent: antibiotics for drug resistant bacteria and products fororphan diseases are two examples that spring to mind.The upshot of these different approaches and choices isthat, as noted above, a great deal of PPP activity deviatesmarkedly from the standard perception of what PPPs are andhow they operate. It is true that many PPP deals, and themajority of those with large multinational pharmaceuticalcompanies, involve classical partnerships that are built onjoint decision-making, majority funding from the PPP partnerand in-kind donations of effort and skills from the privatepartner. Under these deals, the PPP can be very much theweaker party, since it relies on the charitable or strategicmotivations of its private partner, which may change with thenext merger or acquisition. However, an equally large numberof PPP deals do NOT look like this, particularly when the PPPhas control over the R&D process or the background IP.Indeed, these deals may have neither joint decision-making,in-kind donations nor private input – and may not eveninvolve partnerships at all.The net effect of these new models, approaches anddynamics is that the new PPP product developmentorganisations are, in many ways, PPPs in name only. Bylumping them together under a generalized “partnerships”umbrella that encompasses everything from charity tobusiness-funded health programmes, we risk failing tounderstand – and therefore capitalize on – the very specificstrengths and opportunities that these groups offer. In thiscontext, we note particularly their ability to deliver highhealth-value new drugs to neglected disease patients, theircapacity to reduce costs and risks to industry andgovernments, and their catalytic role in translating basicinto applied research even in the absence of acommercial market.ChallengesThe first challenge is to provide policy-makers andgovernment donors with a better differentiated understandingof PPPs generally, and a far better understanding of whatproduct development PPPs are and how they operate – andperhaps a better name for the PDPPPs.The second is to urgently encourage governments totranslate this understanding into policies that support theseproduct development organizations, in particular policies thatspecifically encourage and reward industry involvement inthese groups (no such policies now exist) and new fundingstreams to address the noticeable, even embarrassing, lack ofpublic funding for them, despite the fact that they are nowresponsible for three quarters of all neglected disease drugdevelopment. Further, industry policies need to be tailored tosuit different industry groups: not only multinational drugcompanies, but also the smaller biotechs and CROs who areplaying an increasingly active role.PPPs also face internal challenges, the greatest – but notthe only one – of which is their funding gap. Even the bestperformingPPP cannot continue to contract and pursue R&Dprojects in the face of funding deficits of up to 50% in thenear future. PPPs are also not all the same, with someperforming better than others. While much of this reflects thevarying difficulty of the different disease targets these groupsaddress, all PPPs nevertheless need to seek to match industrylevels of efficiency and productivity if they are to secure fundsfrom risk-averse public donors. This is likely to require notjust public health expertise, but also high in-house levels ofindustry expertise and understanding, including through thecomposition of Scientific Committees, Boards and staff; andthe willingness (and funds) to contract in the necessary skillswhen gaps become apparent. For instance, Medicines forMalaria Venture (MMV), which already has high levels of inhouseskills, has readily moved to secure CRO assistance onindividual projects to maintain their performance standards.Industry, likewise, has challenges to address, in particularmultinational drug companies who have more flexibility toparticipate than do many smaller enterprises. Although fourof the top twelve multinational companies now have activeneglected disease programmes collectively employing over200 scientists, others do little or nothing in terms of neglecteddisease R&D. Those companies with modest activity couldreview whether this could be increased – and in particularwhether partnering could offer a lower-risk, more costeffectiveway of pursuing greater activity. On the other hand,companies with little or no in-house expertise in infectious orveterinary diseases – who are likely to be unwilling, andperhaps unsuited, to full-blown neglected disease R&D – canalso take up the challenge by contributing creatively in other144 ✜ Global Forum Update on Research for Health Volume 4