John Lagus
Executive Vice President of Business Development, Tanner Pharma
Living with a rare disease can be particularly challenging; from getting the right diagnosis to finding and accessing treatment that works.
It’s estimated there are around 7,000 rare diseases, and only 10% have treatments available. This means that most people living with a rare disease — defined by the NHS as a long-term debilitating condition affecting less than five or fewer people in 10,000 — may have no treatment options. Even if potential treatments exist somewhere in the world, they may not currently be accessible for patients in many countries, if ever at all.
Three main routes
Although it might seem overwhelming for patients who are used to getting a prescription from their doctor to seek out innovative treatments, there are processes in place to help people with rare diseases gain access to new, investigational medicines.
John Lagus, EVP of Business Development at Tanner Pharma explains: “There are three main routes available to access a medicine: you can become part of a clinical trial, or if you are lucky, that medicine may be available commercially where you live. If neither of these is an option, most countries have legislations in place that allow patients, under the care of their doctor, to have an unlicensed medicine imported for use when that patient has no other treatment options.”
The benefits to patients of being able to access investigational medicines in this way are obvious in terms of the lifesaving or life-enhancing impact.
Since accessing medicines for rare diseases is a physician-driven process, the more information the patient has about procedures in their country, the better the outcome — even if it means they have to start the conversation with their doctor.
Pathways in place
People living with a rare disease, their parents or carers, are often well-informed about their condition and what treatments are available, Lagus says. They are active advocates for their own rare conditions and are engaged with patient groups. This knowledge is increasing all the time as information about rare diseases becomes more widely available. Similarly, information about early access pathways is gaining awareness among rare disease patients and advocates.
Uncovering possibilities
Lagus adds that the best thing people with rare diseases can do is to engage with patient groups and their doctors. “Since accessing medicines for rare diseases is a physician-driven process, the more information the patient has about procedures in their country, the better the outcome — even if it means they have to start the conversation with their doctor.”
If you are a patient who needs access to unlicensed medicine, speak with your doctor to see if early access is an option for you.
