A new report has highlighted the changing face of drug development with new and emerging biopharmaceutical companies increasingly dominating late-stage clinical trial activity.
Historically, large global pharmaceutical companies have been the dominant players in the development and commercialisation of new medicines.
Yet a new report from IQVIA has found emerging biopharmaceutical companies are now increasingly dominating R&D output.
This report defines 'emerging' as companies with annual revenue of less than US$500 million or an annual R&D spend under US$200 million.
These companies accounted for almost two-thirds of new therapies patented in 2018 - companies like Bluebird bio and Alnylam Pharmaceuticals - compared to large global pharmaceutical companies with just one-quarter.
Bluebird bio is a US-based company with a market capitalisation of around $10 billion.
Alnylam Pharmaceuticals has a current market capitalisation of $14 billion and the first RNA-interference therapy approved in the US and Europe. The highly complex targeted therapy works by turning off genes that cause disease.
The emerging companies also dominate pipelines, accounting for 72 per cent of late-stage clinical trial activity, up from 61 per cent ten years ago. Large pharmaceutical companies accounted for just 31 per cent of therapies in late-stage development - down 20 per cent over the past decade.
Another development is the growing trend of these emerging companies retaining control over the commercialisation of their therapies - partnering selectively or not at all.
The dynamics of the global biopharmaceutical landscape appear to be changing - along with the technology.
According to the report, the number of cellular, gene and nucleotide therapies in late-stage development has more than doubled over the past three years. They now represent around 10 per cent of all therapies in late-stage development.
The highly targeted and personalised nature of these therapies pose a number of new challenges for regulatory and reimbursement decision-makers around the world, including Australia, with new requirements for manufacturing, distribution and administration.
Dr Amanda Ruth