Accelerating the Developing of Orphan Drugs for Rare Diseases

A rare disease is defined as one affecting less than 200,000 people (in the US) or no more than five in 10,000 of the general EU population. 

There are approximately 7,000 rare diseases, affecting an estimated 30 million people in each of the US and EU. Worldwide, there are over 300 million people living with one or more identified rare diseases, representing 3.5% - 5.9% of the global population. 

The development of new treatments to address these unmet clinical needs clearly represents an important global health priority. Historically, commercial pressures meant the challenges and cost of developing such medicines were not cost-effective given the projected financial returns. In recent years, however, there has been a sea change in industry activity as evidenced by the increasing prevalence within drug development pipelines and the numbers of new molecules reaching the marketplace. Between 2016-2019, 82 of the 175 new FDA approvals were for rare diseases. Regulatory authorities have sought to encourage and motivate industry to develop drugs for these serious medical conditions through the creation of a supportive infrastructure.

This white paper will discuss four principal CMC challenges for the developers of orphan drugs and the potential solutions which are emerging:

• Development of patient-centric dosage forms based upon molecule properties and patient needs
• Rapid accelerated optimization and validation of product performance in humans
• Tailored manufacturing and supply of drug products into patient trials
• Rapid scale-up and commercial manufacturing of low-volume products