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

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compounds from many different sources even if there is no
interested industry partner and no commercial potential. This
runs against the oft-stated maxims that only commercial
market returns can catalyse drug development; and that only
pharmaceutical companies (and perhaps only large
multinational companies) have the requisite experience to
manage the lengthy, complex and expensive process of drug
development.
Equally important is that this flexible, modular approach
not only allows but stimulates different (and often far
cheaper) models of drug development. For example, by
actively pairing small Western companies or academics with
CROs and developing country manufacturers, PPPs create a
neglected disease pipeline that encourages and allows
smaller-sized groups to participate, and at a substantially
lower cost than the traditional commercial approach. We note
that the anti-malarial synthetic peroxide has moved from
laboratory 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 or
less. The TB Alliance also estimates that its anti-TB drug, PA-
824, will cost less than US$ 90 million to complete clinical
development. All these projects involve academic, public
domain or small company leads teamed up with contractors
or a developing country manufacturing partner. PPP codevelopment
of multinational drug company leads can also
be substantially cheaper, since costs of capital (estimated to
roughly double the cost of R&D) are largely avoided, and
companies are able to minimize their risk and financial
outlays – and therefore their final prices. Either or both of
these alternative approaches may provide interesting lessons
for other Western diseases where the profit motive is weak or
absent: antibiotics for drug resistant bacteria and products for
orphan diseases are two examples that spring to mind.
The upshot of these different approaches and choices is
that, as noted above, a great deal of PPP activity deviates
markedly from the standard perception of what PPPs are and
how they operate. It is true that many PPP deals, and the
majority of those with large multinational pharmaceutical
companies, involve classical partnerships that are built on
joint decision-making, majority funding from the PPP partner
and in-kind donations of effort and skills from the private
partner. Under these deals, the PPP can be very much the
weaker party, since it relies on the charitable or strategic
motivations of its private partner, which may change with the
next merger or acquisition. However, an equally large number
of PPP deals do NOT look like this, particularly when the PPP
has 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 even
involve partnerships at all.
The net effect of these new models, approaches and
dynamics is that the new PPP product development
organisations are, in many ways, PPPs in name only. By
lumping them together under a generalized “partnerships”
umbrella that encompasses everything from charity to
business-funded health programmes, we risk failing to
understand – and therefore capitalize on – the very specific
strengths and opportunities that these groups offer. In this
context, we note particularly their ability to deliver high
health-value new drugs to neglected disease patients, their
capacity to reduce costs and risks to industry and
governments, and their catalytic role in translating basic
into applied research even in the absence of a
commercial market.
Challenges
The first challenge is to provide policy-makers and
government donors with a better differentiated understanding
of PPPs generally, and a far better understanding of what
product development PPPs are and how they operate – and
perhaps a better name for the PDPPPs.
The second is to urgently encourage governments to
translate this understanding into policies that support these
product development organizations, in particular policies that
specifically encourage and reward industry involvement in
these groups (no such policies now exist) and new funding
streams to address the noticeable, even embarrassing, lack of
public funding for them, despite the fact that they are now
responsible for three quarters of all neglected disease drug
development. Further, industry policies need to be tailored to
suit different industry groups: not only multinational drug
companies, but also the smaller biotechs and CROs who are
playing an increasingly active role.
PPPs also face internal challenges, the greatest – but not
the only one – of which is their funding gap. Even the bestperforming
PPP cannot continue to contract and pursue R&D
projects in the face of funding deficits of up to 50% in the
near future. PPPs are also not all the same, with some
performing better than others. While much of this reflects the
varying difficulty of the different disease targets these groups
address, all PPPs nevertheless need to seek to match industry
levels of efficiency and productivity if they are to secure funds
from risk-averse public donors. This is likely to require not
just public health expertise, but also high in-house levels of
industry expertise and understanding, including through the
composition of Scientific Committees, Boards and staff; and
the willingness (and funds) to contract in the necessary skills
when gaps become apparent. For instance, Medicines for
Malaria Venture (MMV), which already has high levels of inhouse
skills, has readily moved to secure CRO assistance on
individual projects to maintain their performance standards.
Industry, likewise, has challenges to address, in particular
multinational drug companies who have more flexibility to
participate than do many smaller enterprises. Although four
of the top twelve multinational companies now have active
neglected disease programmes collectively employing over
200 scientists, others do little or nothing in terms of neglected
disease R&D. Those companies with modest activity could
review whether this could be increased – and in particular
whether partnering could offer a lower-risk, more costeffective
way of pursuing greater activity. On the other hand,
companies with little or no in-house expertise in infectious or
veterinary diseases – who are likely to be unwilling, and
perhaps unsuited, to full-blown neglected disease R&D – can
also take up the challenge by contributing creatively in other
144 ✜ Global Forum Update on Research for Health Volume 4