Intended for healthcare professionals

Rapid response to:

Feature Drugs for Neglected Diseases

US incentive scheme for neglected diseases: a good idea gone wrong?

BMJ 2014; 349 doi: (Published 21 July 2014) Cite this as: BMJ 2014;349:g4665

Rapid Response:

As a medical humanitarian organisation that has been working to diagnose and treat people suffering from neglected diseases for almost 30 years (1), Médecins Sans Frontières (MSF) understands only too well that the dearth of research & development (R&D) for new tools to treat these diseases is directly attributable to a lack of funding and effective incentives. These diseases affect some of the poorest and most marginalised populations in the world. The imbalance between their medical needs and the availability of suitable medical tools is too often fatal. In spite of representing more than 10% of the global disease burden, over the past decade, less than 3.8% of new drugs approved across the world were indicated for neglected diseases (2). This represents very little progress from the previous decade, especially as it relates to new chemical entities for neglected diseases.

Innovative incentive mechanisms for R&D, including “pull” mechanisms such as the US FDA Priority Review Voucher (PRV), are crucial to spurring innovation in areas lacking the conventional market “pull” of revenue derived from high product prices and high-volume sales.

However, the FDA’s award of a PRV to Knight Therapeutics for a product that Knight neither developed nor manufactures – and which is not easily procured at affordable prices – highlights the shortfalls of the PRV mechanism as currently designed. The PRV’s effectiveness as an incentive for neglected diseases is limited by its failure to ensure it rewards genuine innovation, a lack of obligations to guarantee affordability and patient access to treatments, and administrative restrictions that limit its potential value to innovators and therefore its effectiveness. MSF is urging the US Congress to amend the PRV legislation to ensure that these shortfalls are fixed and the PRV can fulfil its role in stimulating meaningful investments in neglected disease R&D.

Amendments to the PRV legislation are vital to ensuring it delivers benefits to neglected patients, yet even if fixed, the PRV mechanism alone is not enough to address unmet medical needs in the area of neglected diseases. Additional funding, as well as incentives that promote research collaboration and efficiencies, “de-link” the costs of R&D from the price of the end product, and ensure access and the affordability of treatments, should be prioritised. For example, the 3P Project (3), developed by MSF in collaboration with other organisations, would employ an open collaborative R&D framework and a combination of push, pull and pool mechanisms to promote the timely development of more effective treatments for drug-resistant tuberculosis (DR-TB).

MSF calls on the US Congress to amend the PRV mechanism to close loopholes and ensure that benefits reach patients suffering from neglected diseases, and to consider additional mechanisms to incentivise R&D for neglected diseases that ensure all patients needing treatment are able to access the resulting products.

(1) An overview of the current innovation and access challenges MSF faces for NTDs:
(2) Pedrique, et al. 'The drug and vaccine landscape for neglected diseases (2000—11): a systematic assessment'. Lancet (2013):
(3) 'PUSH, PULL, POOL: Accelerating Innovation and Access to Medicines for Tuberculosis',

Competing interests: No competing interests

31 July 2014
Jennifer Reid
Julien Potet, Katy Athersuch, Maisy Grovestock, Judit Rius Sanjuan
Médecins Sans Frontières- Access Campaign
Medecins Sans Frontieres/ Doctors Without Borders (MSF) 333, 7th Avenue, 2nd Floor, New York, NY 10001 USA