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.


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 systems. Patients often lack access to adequate treatment, information or 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 rare disease are a public health priority; empowering patients to influence research agendas and valuation of new treatments; promoting continued research and development; and ensuring sustainable access to diagnosis, treatment and care.

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6–10% of the population are affected by a rare disease


95% of rare diseases are estimated to have no treatment option


30% of rare disease patients die before the age of 5

What we 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.

‘No human life should be judged by a statistic. Every rare disease patient deserves treatment and support, no matter their disease or where they live.’ 

Prasanna Kumar B Shirol

Organisation for Rare Diseases India

Key partners and stakeholders

  • International Federation of Pharmaceutical Manufacturers and Associations (IFPMA)
  • International Rare Disease Research Consortium
  • Rare Diseases International
  • Findacure, UK

  • Tuberous Sclerosis Alliance, USA
  • Organisation for Rare Diseases India
  • Advocacy Service for Rare and Intractable Disease multi-stakeholders, Japan

The project was initiated and funded by the IFPMA.