R&D for rare and neglected diseases
Rare and neglected diseases have traditionally received little attention from researchers. Fortunately, that is changing – and Novartis has developed dedicated research organizations, collaborations and financing mechanisms to help address this challenge.
Targeting unmet medical needs
Our research priorities are not determined by potential market size. Our research organization, the Novartis Institutes for BioMedical Research (NIBR), is to actively target rare diseases where we have clear understanding of the disease’ underlying cause and where there is real unmet medical need.
An example is Muckle-Wells syndrome, a rare disease with potentially fatal complications. Muckle-Wells syndrome is part of a group of autoinflammatory diseases called cryopyrin-associated periodic syndrome (CAPS). Novartis developed the first treatment option for the 6 000 patients worldwide affected by CAPS.
Innovation for diseases of the developing world
Novartis is committed to researching vaccines and medicines for diseases of the developing world through two institutes within NIBR:
- Novartis Institute for Tropical Diseases (NITD) in Singapore
- Novartis Vaccines Institute for Global Health (NVGH) in Italy
These institutes are fully dedicated to discovering vaccines and treatments for neglected diseases.
Our commitments in action
Applying novel research to diseases of the developing world
In collaboration with OneWorld Health, a US-based not-for-profit pharmaceutical company, NIBR is applying expertise from research targeting cystic fibrosis to tackle secretory diarrhea — a disease that kills more than 1.6 million children a year in developing countries.
Funding neglected disease R&D
Donations for neglected disease research have increased in the last decade, generating an extensive portfolio of compounds to fight these diseases. As more compounds from this pipeline move from research to more costly drug development, there is a significant funding shortage approaching. Novartis has encouraged an initiative to finance R&D for neglected diseases; called FRIND, the proposal would generate additional funding rather than redirect limited existing funds.
Responding to the unmet needs of children
In collaboration with Medicines for Malaria Venture, the Novartis Malaria Initiative developed a sweet-tasting artemisinin-based combination therapy (ACT) to treat malaria in children. Launched in 2009, the medicine is the first ACT meeting WHO requirements for pediatric use.
