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European Biopharmaceutical Review

Advancing Affordable Orphan Drug Development

EBR: What are the key challenges in access to, and affordability of, medicines for orphan diseases, and how can they be tackled?

Haishan Jang: One of the most difficult challenges of orphan drug development is the small number of patients available. Regulatory agencies allow for fewer patient participants in trials, but there are relatively high costs in patient recruitment, trial design, and manufacturing small batches of a drug. However, as more orphan drugs reach the clinic, data are starting to show that with the right team and regulatory strategy, orphan drug development is, on average, less expensive than traditional drugs.

Since around 90% of rare diseases have no available medicines, patients lack treatment options, and physicians don’t have experience in diagnosing and treating the diseases. With limited market size, it is understandable that many companies do not prioritise or allocate resources to developing new drugs for orphan disease indications. To offset development costs, treatment costs per patient can be exponential, and beyond the affordability of health agencies and insurers.

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With more than 30 years in the pharmaceutical industry, CEO of BRIM Biotechnology, Haishan Jang, was previously a member of senior management at Centocor, DuPont, and Sanofi, and was the former President at TWI Biotechnology, Taiwan. Haishan has contributed to the development of many leading drugs, including Uroxatral®, Tirazon®, Remicade®, Simponi®, and Stelara®. Her breadth of experience and translational science expertise has led BRIM to advance discoveries into the clinic, and successfully spin-out Ascendo Biotechnology, Inc. Haishan co-founded BRIM Biotechnology with Dr Frank Lee in 2013.
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Haishan Jang
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