Measuring progress in neglected disease drug development.

BACKGROUND

Since the late 1990s, funding for development of neglected disease drugs has increased with an influx of resources from product development partnerships (PDPs). Previous research showed modest gains in drug approvals and products in Phase III of clinical development in the period 2000-2008.

OBJECTIVE

We assessed the 2009-2013 period in terms of numbers of products in Phase III development and numbers of approvals. Subsequently, we calculated the PDP share in terms of sponsorship of new approvals. We also identified the numbers of 2000-2013 approvals included in the World Health Organization's Essential Drug List (EDL).

METHODS

We identified new approvals and Phase III products targeting neglected diseases in the period 2009-2013 by searching ClinicalTrials.gov, IMS R&D Focus, and Investigational Drugs Database, as well as drug regulatory agency websites. Subsequently, we determined which products approved between 2000 and 2013 have been included in the most recent version of the EDL.

RESULTS

We found 20 new approvals targeting neglected diseases in the period 2009-2013. PDPs were the primary sponsor of 57% of new approvals in this time frame. Approvals included 1 new molecular entity, 5 vaccines, 2 new indications, 9 fixed-dose combinations, and 3 new formulations. HIV/AIDS (pediatric indications) and malaria accounted for 60% of approvals in 2009-2013. The average number of new approvals per year for neglected diseases rose from 2.6 in 2000-2008 to 4.9 in 2009-2013. The World Health Organization included 44% of 2000-2013 approved products on the EDL. We found 18 products currently in Phase III of clinical development. Products in Phase III testing included 3 new molecular entities, 6 vaccines, 2 fixed-dose combinations, 5 new indications, and 2 new formulations.

CONCLUSIONS

Increased funding through PDPs for neglected disease drug development seems to be producing results. Approvals and products in Phase III testing have shown a steady increase since 2000, with nearly a doubling of products in 2009-2013, compared with 2000-2008, in terms of the annual average yield. However, only 3 new molecular entities have been approved in 14 years. In addition, malaria and HIV (pediatric indications) seem to have benefited most from increased funding, whereas less success has occurred with other diseases. Inclusion of newly approved products on the EDL has been slow and limited, with only 44% of new approvals added to the list. Uneven progress suggests funding could be better targeted. In addition, PDPs could do more to facilitate access, in particular by working closely with the World Health Organization to assess the clinical effectiveness and cost-effectiveness of new approvals.