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Drugs for rare diseases will help propel pharma sales to $1.2trn by 2024; report

Orphan drug sales growth will be 11%+ a year


The most valuable drug in the pharma industry’s pipeline isn’t a new medicine for diabetes or cancer, but Vertex’s triple therapy for rare hereditary disease cystic fibrosis, according to EvaluatePharma.

That assertion ties in with its new report on worldwide prescription drug sales, which says that drugs for orphan diseases will outperform the overall pharma market, almost doubling to reach a whopping $262bn in 2024 when they will account for a fifth of all drug sales.

“This highlights the industry’s continued move to address small groups of neglected patients with high unmet need and to benefit from traditionally reduced payer scrutiny on orphan drugs, as well as regulatory and financial incentives,” says EP.

Gene and cell therapies will also be a growth driver as prescription drug sales grow at more than 6% a year to reach $1.2trn in 2024 – a six-fold increase on the growth rate seen between 2011 and 2017 when the financial crisis was at its peak. Multiple new cell and gene therapy approvals are expected in the wake of the launches of CAR-T therapies for cancer and Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec) for vision loss.

Meanwhile, orphan drug sales growth will be above 11% a year over the period, predicts the report, and oncology overall will perform even better with annual growth of 12%.

There’ll be plenty of factors working to restrict growth, however, including payer scrutiny of prices and sales losses due to generic and biosimilar competition which will impact $251bn of sales over the next seven years. That will also be a factor for orphan drugs, with recent evidence pointing to a reduction in the price increases that can be supported. It suggests.

The report also suggests that the trend among big pharma companies to narrow down the focus of their businesses to a few core therapeutic areas will pay off, with these areas delivering the greatest value gain and cumulative sales of $324bn. Novo Nordisk’s laser focus on diabetes and other core areas such as haemophilia will give it the best return on R&D investment.


Among the other predictions in the EP report are that Novartis will be the biggest pharma company by 2024, swapping places with Pfizer which will be competing with Roche for the second spot. The planned merger of Takeda with Shire – assuming it goes ahead – would propel the combined company to ninth place based on 2024 sales.

Looking at specific products, EP says AbbVie’s immunology drug Humira (adalimumab) will remain the biggest-selling drug in the world in 2024 – despite the start of biosimilar competition in 2023. Merck & Co’s immuno-oncology drug Keytruda (pembrolizumab) will be in second place with sales of $12.7bn, overtaking Bristol-Myers Squibb rival Opdivo (nivolumab) which will be in fourth place behind Celgene’s cancer immunomodulator Revlimid (lenalidomide).

Article by
Andrew McConaghie

7th June 2018

From: Research



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