Celebrated on 29 February 2024, the rarest day of the year, Rare Disease Day unites the global rare disease community to address challenges and promote equity in healthcare, diagnosis, and treatment accessibility. It's a day to come together to share knowledge and support, aiming to improve understanding of rare diseases.

The European Union defines a rare disease as one that affects fewer than 1 in 2,000 people (the USA 1 in 1,666). There are over 6,000 known rare diseases, and more diseases are being identified. It could reach 10,000.

Although they are considered rare, more of us than you may perceive are affected by rare diseases. Over 300 million people worldwide live with a rare disease, which is the size of the third largest country. This accounts for 5% of the global population.

Smartway proactively supports Rare Disease Day, in solidarity with those affected, advocating for increased awareness, research, and support for individuals and families living with rare diseases.

Navigating the challenges of rare diseases

The challenges faced by patients living with rare diseases are real and significant. Research indicates that in the UK 1 in 17 people are likely to encounter a rare disease during their lifetime.

Living with any disease presents its own set of challenges, but those with rare diseases face other obstacles, such as:

  • Delay in diagnosis: The lack of scientific knowledge, awareness and high-quality information about rare diseases often leads to delays in diagnosis. That means valuable time can be lost.
  • Misdiagnosis: Initial misdiagnosis is common due to the vast diversity of disorders and relatively common symptoms that can mask an underlying rare disease. Moreover, symptoms can vary not only from disease to disease but also from patient to patient with the same disease.
  • Inequality in healthcare access: Access to appropriate quality healthcare can be challenging, leading to inequalities and difficulties in accessing treatment and care. This can result in heavy social and financial burdens on patients.
  • Lack of awareness: Limited awareness about rare diseases can lead to misconceptions, less research funding, a lack of understanding, and inadequate support for affected individuals and their families.

How Smartway supports the rare disease community

At Smartway, our mission is to transform lives by empowering access to medicines globally. As a pharmaceutical wholesale supplier with an extensive global network and regulatory expertise, we take our role seriously in helping patients access medicines that are difficult-to-obtain. 

Accessing medicines to treat patients with rare diseases presents various challenges. Orphan drugs often come at a higher cost, they are usually launched in only a few countries, they have a limited supply, and entail exceptionally strict supply chain requirements. 

Working with healthcare professionals and hospitals

Smartway uses its global procurement and distribution network and its regulatory expertise to supply medicine where it’s needed. We respond to requests from Physicians and Pharmacists to source therapies not available in their country. By leveraging our partnerships with our network of global companies and distributors, we ensure that medicines, including orphan drugs and unlicensed medicines, are delivered safely and on time.

Partnering with manufacturers and other innovators - Early Access Programmes (EAP)

Smartway designs, implements, and manages global programmes that empower access to innovative therapies. We partner with pharmaceutical and biotech organisations to deliver customised programmes for patient treatment. These partnerships have positioned us as a leader in providing Expanded Access, Early Access, Compassionate Use, and Named Patient Programmes across 98 countries.

Our partnership with Ambrose Healthcare, a specialist pharmaceutical company addressing unmet medical needs in rare diseases, is one such example. Through our partnership, we serve as the exclusive supplier of end-to-end EAP services to Ambrose Healthcare. This includes regulatory design and submission, as well as global compliant distribution of their therapeutics under international EAPs.  

Our contribution to Rare Disease Day 2024

On 29 February 2024, we hosted internal events to raise awareness through our whole company and shared information about rare diseases, marking the importance of Rare Disease Day.

From warehouse to accounting, we encouraged our employees to participate in a quiz designed to enhance their knowledge and understanding. This has reinforced our commitment to spreading awareness and fostering a culture of continuous learning across all departments.

With over 90% participation, both online and in person, our employees initiated informative conversations about rare diseases and the importance of raising awareness. All of them said that their understanding of rare diseases increased.

Supporting Rare Disease Day is a vital step towards ensuring that no one with a rare disease feels alone or forgotten. Together, we can illuminate the path towards greater understanding and support for those in need. - Robert Donnell, EVP – Medicines Access 

Click below to discover more about how we support patients worldwide in accessing the therapies they need through our EAP programmes.

Smartway EAP


  1. 1. About - Genetic and Rare Diseases Information Center. (n.d.). https://rarediseases.info.nih.gov/about
  2. 2. Rare Disease Day 2024. (2023, November 17). What is a rare disease? - Rare Disease Day 2024. https://www.rarediseaseday.org/what-is-a-rare-disease/
  3. 3. Rare diseases. (n.d.). Research and Innovation. https://research-and-innovation.ec.europa.eu/research-area/health/rare-diseases_en#:~:text=In%20the%20European%20Union%2C%20a,million%20people%20in%20the%20EU
  4. 4. The UK Rare Diseases Framework. (2020, December 28). GOV.UK. https://www.gov.uk/government/publications/uk-rare-diseases-framework/the-uk-rare-diseases-framework