28th February 2022 is Rare Diseases Day, which is about raising awareness for the 300 million people worldwide living with a rare disease. A rare disease is one that affects less than 1 in every 2,000 people. The Rare Disease Day campaign is about awareness, promoting equitable access to diagnosis, treatment, health and social care and social opportunity for people affected by a rare disease.
Rare Disease Day is important to acknowledge at Smartway. Everything we do is focused on our key mission: transforming lives by empowering access to medicines globally. So for us, this event is really important because of its direct relevance to what we do. We want to help patients with rare diseases get access to medicines where there are significant barriers to obtaining them, through working closely with manufacturers, innovators and healthcare professionals. The impact of our work in ensuring that these medicines are available is at the centre of every decision we make. We will continue to work with manufacturers, regulators and healthcare professionals to meet the objectives of Rare Disease Day, including facilitating early access to medicines and setting up systems and programmes to collect Real World Data on the use of these medicines.
But today, we want to do our bit to raise awareness for Rare Disease Day. So this year, we want to celebrate the journey of the patients with rare diseases, the lives that have been transformed by physicians and carers, and the way communities come together to support every individual who suffers from rare diseases.
Recognising our privileged role in empowering access to medicines
We take our privileged role in empowering access to medicines seriously. There can be challenges to providing access to these medicines that can be simply because of the small patient population for a specific medicine, or that they’re in a difficult to reach part of the world. That is why we invest significant resources in finding new ways to support practitioners and, ultimately, patients, get access to these medicines. Sometimes, it is as simple as raising awareness about them. Other times, it is about getting them to the right person.
As part of Rare Diseases Day, we took some time to showcase the work we’re already doing to help others.
Access to medicines for low and middle-income countries
Rare diseases are global. We believe everyone deserves access to the treatments that they need. We work with charities, aid organisations and NGOs to find the medicines they need and supply them to patients in clinics or refugee camps across the world.
In just one example, we helped patients in Lebanon get access to otherwise unavailable medicines to manage a rare disease.
Partnerships with manufacturers
We work with manufacturers and innovators to enable access to orphan drugs before they’re commercially launched. Supply pre-launch is important because an effective treatment can take years to be launched in a particular country – if it is ever even launched there at all. Facilitating early access supports patients.
Empowering pharmacists and physicians across the globe
In August 2021, we received a request from a pharmacist in a South American country who had an urgent patient clinical need for an orphan drug. We worked urgently with a manufacturer to establish a legal supply chain and distributed the medicine across to the hospital. This is a day-to-day occurrence for our team: a crucial aspect of how we help patients around the world, often on an urgent basis.
Putting close relationships at the core of our mission
What these examples have in common is our culture of transparency and trust. At the core of our work in rare diseases is our ability to maintain close relationships with healthcare professionals, manufacturers, innovators and our wider supply chain.
It’s this transparent culture that will allow us to continue to transform lives for years to come, responding with care and commitment to new challenges, wherever in the world they arise.
To find out more about rare diseases, visit Rare Disease Day.