What we’re reading: Q&A on the ethics of ultra-rare disease drug development and expanded access

From time to time we share articles on topics of interest to us here at MedaSystems. Yesterday was Rare Disease Day, and there were a number of interesting articles and posts around rare disease research and avenues to obtain treatment. One of these was this conversation in Clinical Trials Arena between Alison Bateman-House and William Newton, Q&A: On the ethics of ultra-rare disease drug development and expanded access. It caught our attention because Dr. Bateman-House mentions the issue of drug companies who are out of compliance with the 21st Century Cures Act.

She observes:

“Still, the FDA should enforce compliance to the basic transparency requirements that are currently in existence but oftentimes are not followed. Drug companies are supposed to put in a public space, typically interpreted as their website, the contact person for filing an expanded access request, what information is needed for a request, and how long it will take for a response.”

We know that small life science companies are often overwhelmed and may not be aware of these requirements, which is why we offer a solution that enables companies, even those who are not offering expanded access, to quickly and inexpensively ensure they are in compliance, as well as receive, triage and process other inquiries, such as requests for information about clinical trials. If they later decide to consider expanded access or investigator-initiated studies requests, they can easily add that functionality to the platform, ensuring they have a central record of all contact they have had with external parties, including a complete audit trail.

Visit Q&A: On the ethics of ultra-rare disease drug development and expanded access to read more of the interview.