Cell and gene therapy companies should work to convince healthcare systems of the value of their therapies, as well as improving the efficiency of their manufacturing processes. That’s the view of Andrew Olaye, general manager, U.K. and Ireland and head of EMEA market access at Orchard Therapeutics.
Olaye, who will be speaking at the 21st Annual bioProcessUK conference this month, will focus his panel talk on widening the concept of increasing value in advanced therapies.
“On one hand, there’s a manufacturing cost perspective, but you can also look at it from the other side of the equation—how to quantify the value of treatment.”
According to Olaye, the reason he’s passionate about widening the concept of value is because Orchard’s lead product, Lenmeldy™, is designed to treat early-onset metachromatic leukodystrophy (MLD), an inherited, rapidly progressive and deadly disease affecting only a handful of patients.
“There’ll be globally very few patients identified who are eligible for treatment initially. After newborn screening, patient numbers may increase, but given the personalized nature of the therapy, you’re not going to get economics of scale just by driving down manufacturing costs,” he explains.
Most discussion of gene and cell therapy costs focuses on increasing manufacturing efficiency. For example, as Olaye explains, Orchard is reducing the cost of vector production by moving from adherent-based to suspension-based cell cultures.
They are also automating their drug manufacturing processes, he says.
However, as Lenmeldy can only be given to patients who aren’t yet displaying symptoms or have very early symptoms, reducing manufacturing costs can only go so far, he adds.
MLD affects around 1 in 40,000 live births, meaning only a few hundreds of babies are born with the condition worldwide each year, most of whom would not be diagnosed in time for treatment. Thus, he says, Orchard is also seeking to increase understanding of the value of the therapy to health systems.
The company have sponsored pilot newborn testing screening programs for MLD across the United States and in several countries in Europe. The results of one study was recently published, showing—in Germany—patients could not only be identified using a validated testing algorithm, but also entered into treatment, he says.
The company says this, and other work by researchers, has led to MLD being added to the National Newborn screening program in Norway.
“The process of getting a disease added to national screening programs is variable for each country, but the steps are the same. You need a validated assay and to convince the relevant authorities that you can both screen and have an actionable treatment available,” Olaye says.
Another aspect of improving value, he adds, is demonstrating the effects of treatment.
“It’s about the evidence package you bring to payers that the treatment will be durable and long-lasting,” Olaye explains. “[You] also need to show how treatment would reduce direct health care costs as well as the [long-term] socio-economic burden of the condition.”
