Rare diseases: shaping a future with no-one left behind

Rare diseases affect up to 400 million people across the world – and, for many, there are no treatment options available.

Rare diseases policy


Despite the severe, debilitating and sometimes life-threatening nature of rare diseases, many countries have no policies to help advance research and improve the management of these conditions.

Rare diseases pose numerous unique challenges to patients, carers, healthcare professionals and health systems. Patients often lack access to adequate treatment, information and support.

There is a need for clear policy frameworks and strategic priorities to improve care for people affected by these conditions.

Policy priorities include ensuring that rare disease are a public health priority; empowering patients to influence research agendas and valuation of new treatments; promoting continued research and development; and securing sustainable access to diagnosis, treatment and care.

What we’ve achieved

HPP worked with the Rare Disease Working Group of the International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) to develop a report on rare diseases. Alongside desk research, we interviewed experts from patient groups and research organisations across the world.

The report explores the challenges that rare diseases present to healthcare systems and outlines clear policy priorities to improve care and treatment options for patients.

The report, Rare diseases: shaping a future with no-one left behind, was launched on Rare Disease Day 2017.

Key partners and stakeholders

  • International Federation of Pharmaceutical Manufacturers & Associations (IFPMA)
  • International Rare Diseases Research Consortium (IRDiRC)
  • Rare Diseases International
  • Findacure, UK
  • Tuberous Sclerosis Alliance, USA
  • Organisation for Rare Diseases India
  • Advocacy Service for Rare and Intractable Disease multi-stakeholders, Japan

Project funding

The project was initiated and funded by the IFPMA.