Public Interest in Private Science: New Pathways to Measuring R&D Success

Today’s big product story - the apparent end to a decade-long drought in the drug development pipeline - is the prep stage for tomorrow’s big policy question:  are industry R&D priorities in synch with the changing burden of disease?

With FDA new drug approvals on the rise and as the unlocking of the genome advances the science on potential treatments for conditions ranging from Alzheimers to MS, there is suddenly more interest in just what big pharma is bringing to the table, its relevance to unmet health needs, and at what cost. Institutions now plotting trends in drug commercialization include the UK National Health Service, with its horizon scanning unit at the University of Birmingham; the US National Institutes of Health [NIH]; the World Health Organization [WHO]; private academic groups like the Tufts Center for the Study of Drug Development; and leading industry trade groups – PhRMA itself publishes an annual roster of compounds in development in a dozen different therapeutic segments.

What is compelling is how the surge of interest in industry’s pipeline progress is not confined to Wall Street. R&D is becoming “democratized,” with an expanded audience of stakeholders focused on the public interest around a good – new medicines – that is essential to health. The desire now is to find ways to match that interest with some metrics that provide a more precise understanding of whether private investment is geared to outcomes that might lessen the current and future burden of disease. At present, there are no agreed metrics and lack of data is compounded by the transition underway in the global disease profile, from communicable to chronic, non-communicable conditions associated with population aging. Here, new drugs can make a significant contribution – if there is adequate translation of the gains made in our understanding of molecular biology into new therapeutics that improve a patient’s functional capabilities enough to prevent costly institutionalizations.

A new research analysis* just completed by Quintiles – a leading CRO and analytics firm that is now itself a major player in drug development – attempts to fill the metrics gap. It was featured at a discussion panel at the annual meeting of the International Society of Pharmacoeconomics and Outcomes Research [ISPOR] in Washington in June. The research investigated the relationship between the global disease burden, as ranked by the WHO and the NIH according to standardized Disability Adjusted Life Years [DALY], against the spending and development targets of drug developers, mainly in the private sector; this latter was obtained from the Adis R&D Insights data base of compounds in Phase I to III. “The point was to take a stab at providing a definitive answer to this basic question – is drug development linked to the real burden of disease?” Quintiles Senior Vice President John Doyle told Pharm Exec.
The Quintiles work concluded that the current drug pipeline is “not optimized” to addressing the biggest contributors of disease burden, a trend that is likely to accelerate over the next two decades without a realignment of economic incentives that bring more focused attention to fighting key chronic conditions like diabetes, dementia and ischemic heart disease. “Our findings suggest that there is not going to be much improvement in the correlation between the disease burden in areas where DALYs are highest and the drugs in development for those conditions, without a reprioritization of the pipeline based on public health need,” Doyle said.

To address the disconnect, the study proposes taking a page from the work of various multilateral organizations, by developing a “public health index” that includes variables like number of available alternative interventions, economic impact of diseases, adherence levels in treatment, and education and prevention links to establish a roster of those key disease priorities for which drugs would be ideally suited to create a maximum public health impact.

Quintiles doesn’t address the issue of how such an index might be used in practice, but it is certain to attract the attention of those who favor a more prominent public sector role in shaping the R&D spending priorities of big Pharma. The WHO has established a consultative Working Group on R&D Financing and Coordination, many of whose members are on record as favoring some form of global benchmark that WHO member states could use to hold big Pharma more accountable for its R&D investments. Its work is due to drive action at the May 2013 World Health Assembly on whether the WHO should adopt a global R&D Treaty that asks each member to set national priorities for drug R&D spending, as a form of “moral suasion” in getting the drug majors to move beyond market incentives in placing their drug bets.  Simply put, standards for responsible investment are coming – and the decision suite for the industry’s scientists is destined to get more crowded.

*”Public Health Innovation: Biopharmaceuticals Lost in Translation?” John Doyle and Nader Halim; Quintiles Consulting; June 2012.