Three Key Factors In Supporting Rare Disease Clinical And Commercial Drug Product Manufacturing

Despite the name, rare diseases are actually not that rare at all. According to the National Institutes of Health (NIH), 10% of the population have one of the nearly 7,000 known rare illnesses. And even with a diagnosis, more than 90% are still without an FDA approved treatment.

Biopharmaceutical researchers are making great progress in combating rare disease, yet there is still much more work to be done.

Developing medicines to treat rare diseases presents scientific and operational challenges. The complex biology creates unique hurdles for scientists, making it difficult to design and implement a drug development program. Within a particular rare disease, there can be many variations or subtypes resulting in numerous clinical manifestations and disease progressions.

In this article, explore the considerations for drug product manufacturing, specifically pricing, small batch filling, and risk to patients.