In the U.S., a rare disease or disorder is defined as one that affects fewer than 200,000 persons. Cumulatively, more than 7,000 rare diseases affect more than 30 million Americans. [2] “As many as 95% of rare diseases have no specific treatment or curative options,”...
Product Development
Regulatory Guidance for Your Rare Disease Therapy
February is Rare Disease Month because the 29th is the rarest of days. This month prompts a crucial exploration into the challenges of developing therapies for rare diseases. These conditions, often affecting children and encompassing serious or life-threatening...
Preparing for Strategic Investor and Partner Meetings
This post explores the art of building investor and partner relationships at conferences like JPM Healthcare. We share insightful strategies for small companies and startups to use these platforms for growth effectively. From aligning your yearly plan with strategic...
Do Value-Based Agreements for New Medicines Work?
In February 2020, I posted about a value-based agreement (VBA) for Alnylam Pharmaceuticals RNAi treatment for acute hepatic porphyria, a group of rare genetic diseases due to one or more deficiencies in the heme biosynthesis pathway in the liver. [1] I re-shared the...
Making a Generic Drug
This article explores the complex world of making generic drugs, cheaper versions of brand-name medicines. Creating these generics comes with many challenges. It starts with not having all the information about the original drug you're trying to copy. You need to do a...
Tools for Transforming Drug Development
Note: I used the AI Assist Drafting in Notion to generate some topics and content blocks. Photo by National Cancer Institute on Unsplash Introduction The FDA issued final guidance on the qualification process for Drug Development Tools (DDTS) in November 2020 after...
The CDERDirect NextGen Collaboration Portal
Rapid and secure communication with the FDA is an essential task within the regulated industry, and CDER makes some communications easier through the NextGen Collaboration portal. This tool is part of the FDA's efforts to streamline and modernize the drug development...
Make or Buy: Outsourcing to Meet Your Strategy
Aseptic culturePart 1 - Why Outsource?Very few pharmaceutical companies conduct all parts of discovery, development, and manufacturing in house, and startups or small companies discover the majority of new medicines. New medical devices are frequently developed by...