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The global orphan drug market is expected to account for 19% of all branded prescription drug sales by 2020, according to a new report.

EvaluatePharma estimates drugs for rare diseases could collectively account for $176bn in annual sales in just over five years – easily outstripping the current 5% growth rate seen in the overall prescription drug market.

Anthony Raeside, Evaluate’s head of research, said: “Although orphan populations are by definition the smallest, they represent big per-patient outlays and insurers will be looking carefully at new tools to arrest cost growth as more and more orphan drugs launch.”

Industry in rare disease has been increasing in recent years and 2013 saw 260 orphan drug designations granted in the US, a record number from the FDA.

A key driving force behind this is the higher prices orphan drugs can command, with the average orphan drug cost per patient per year standing at $137,782 compared to the $20,875 average price for a non-orphan drug.

Meanwhile, Evaluate found that, despite the inherent difficulty of recruiting patients for orphan drug trials, the impact of regulatory assistance measures means that phase III orphan drug development costs half that of non-orphan drugs. However, they said development time for orphans is not shorter than that of non-orphans.

Competition in the sector is fierce, with return on investment for a phase III or filed orphan drug standing at 1.89 times more than for a non-orphan drug.

At the top, Evaluate predicts Bristol-Myers Squibb will overtake Novartis as the leader in orphan drug sales by 2020 due to sales forecasts for its cancer immunotherapeutic Opdivo.

© Interactive Pharm 2022

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