Orphan drugs face new pressures - European Medical Journal

Orphan drugs face new pressures

2 Mins
Words by EMJ GOLD newsdesk

The orphan drug market, which has flourished in recent years, faces a more complex landscape in the decade ahead, according to a new report from Evaluate Pharma, which asks whether orphans are “losing their sparkle”.

Despite healthy average growth over the past decade, forecasts suggest a more modest trajectory for the sector over the next 10 years. The industry’s compound annual growth rate averaged 11% over the past decade, but is expected to barely break into double digits in the next, with the gap between orphan and non-orphan markets narrowing from 5% to just 2% over the next four years.

So why is this? A new focus on more common diseases, such as obesity and Alzheimer’s disease, is cited as a key driver of the shift. The report details how “the blazing return of big drugs for big diseases” could attract attention and resources away from orphan drug development due to their expansive market size, promising big rewards for investors and patients alike.

The obesity market alone could be to be worth 100bn (USD) by 2030, according to the report, when it sat at just 8.4bn (USD) in 2022, with over 100 drugs currently in trials and eight on the market.

More demanding payers are highlighted as another reason for heightened pressure on orphan drug makers, with many moving to prioritise broader health challenges facing their populations. They’re also “balking” at the high prices of these therapies, says Evaluate, so are hoping to benefit from competitors moving into some corners of the orphan market in the next few years. This will be a new test for existing players for which market access is already a challenge.

Looming legislative measures such as the Inflation Reduction Act could also add a layer of complexity, finds the report. While orphan drugs approved for a single indication are exempt from controls, therapies with multiple indications are on the hit list. These include AbbVie’s B-cell cancer drug ibrutinib, the only orphan drug subject to the first round of IRA price negotiations, but others could be targeted later.

Despite these challenges, however, Daniel Chancellor, Director of Thought Leadership,  Consulting and Analytics, Evaluate, remains optimistic, describing the road ahead as “turbulent” rather than wholly negative. Notably, despite the headwinds, orphan drugs will generate 185bn (USD) this year, and close to 270bn (USD) by 2028.

“Growth rates may be moderating slightly, but this is an inevitable consequence of the success of orphan drug strategies,” Chancellor explains. “It is far easier to achieve high growth from a small base, but to sustain that despite commercial maturity shows that the orphan drug pipeline remains highly productive.”

While the orphan drug market is facing tougher times and sectors targeting less specialised diseases are looking forward to strong growth in the coming years, there is room in the market for both to succeed. As Chancellor identifies, the industry is poised to weather the storm and continue to thrive for the benefit of rare disease patients, albeit at a more measured pace than in previous years.

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