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The opportunities and challenges of biosimilar orphans

Expert opinion

The opportunities and challenges of biosimilar orphans

Hoss A. Dowlat

The orphan class of biological products deserves special attention for biosimilar development as they provide a vital niche to the patient and society, and are currently minimally investigated.

Biosimilar orphans would afford the only opportunity for treatment of numerous new patients worldwide due to reduced price and better accessibility. Their development presents financial and logistic obstacles to industry. Therefore, new R&D and support initiatives are needed for biologics with solely orphan indications.

Although the existing market for orphans is small in comparison with blockbuster biosimilars, scope and markets at regional, national, and continental level will expand, as experience has shown with entrance of other biosimilars.

Eculizumab and palivizumab humanized mAbs exemplify two of the few orphans being developed.

The same principles as regulatory approval of current biosimilars can be applied to this class of orphans, but the restricted availability of originator reference medicine and difficult access to patients should be acknowledged and considered by regulators including the EMA or FDA through caseby- case Scientific Advice. In the long term, this R&D will provide hope for treatment for a larger sector of the population in Europe, United States, and worldwide.

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