Marketing in the field of life science is complex, with very different practices, standards and tactics required depending on the size of the potential customer base.
A lot of this comes down to economies of scale; as most of the cost of a new medicine or medical device is in its research and development, there needs to be enough people who will use it to make it viable.
This has, for decades, meant that certain rare diseases lack an incentive for laboratories to explore potential treatments, as the costs for researching orphan drugs are often passed onto patients and health authorities.
One of the biggest examples of this in recent years is the story of Libmeldy, a drug described by the NHS as the “most expensive” in the world at a cost of £2.8m per course of treatment.
It is used to treat a rare childhood disease known as metachromatic leukodystrophy (MLD), which affects five babies per year in England and works as a personalised gene therapy treatment.
MLD is a genetic disorder with similar symptoms to multiple sclerosis caused by a deficiency in the enzyme arylsulfatase A. This leads to progressive loss of cognitive and motor function, and children diagnosed with it have a life expectancy of between five and eight years old.
The rare nature of the disease also led to complications with getting approval; most Phase III clinical trials require at least 1000 people to take part, but some diseases are so rare that there simply are not that many patients able to take part.
Ultimately, the process of getting Libmeldy approved was somewhat complex. Whilst it was approved for medical use in the EU in late 2020 and in the UK in early 2021, it took a full year of negotiation for NHS England to agree to provide the specialist treatment with a confidential discount agreed.
This, alongside FDA approval in the United States, was seen as a watershed moment where a particularly rare orphan drug can help patients, incentivising laboratories to follow the lead of Orchard Therapeutics and other orphan drug specialists.
