The discovery and development of a new medicine is a complex and lengthy process. Many founders begin their search for a new therapy without understanding these details. In my experience, most of the public views drug development as “the news reports that scientists...
Product Development
Making a New Medicine Part 2: Introducing Clinical Trials
The discovery and development of new medicines is a complex and lengthy process. Many founders begin their search for a new therapy without an understanding of these details. In my experience, most of the public views drug development as “the news reports that...
Making a New Medicine Part 1: Discovery and Development
The discovery and development of a new medicine is a complex and lengthy process. Many founders begin searching for a new therapy without understanding these details. In my experience, most of the public views drug development as “the news reports that scientists show...
The Importance of Rare Disease Research
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,”...
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...