Today is Rare Disease Day.
In a way, every day for CDD and the CDD community is Rare Disease Day. Why?
Effective collaborations are the key to efficient drug discovery on a tight budget. And by definition, all rare disease drug discovery must be done on a tight budget leveraging a small number of highly motivated, inspired researchers. People who are working for more than “just” money.
Rare diseases, paradoxically, may be the best way to do commercial drug discovery given the current challenges with the core business model. Rare diseases may be more tractable – they are rare by definition and may be due even to a single mutation. Rare diseases, from a biological perspective, are generally simpler than complex diseases like Alzheimer’s, Cancer, and CV. And given the complex, interrelated nature of many biological processes, insights gleaned from rare disease R&D efforts will illuminate commercial disease R&D efforts – and vice versa (analogous to the piggybacking of neglected disease R&D efforts on the coat-tails of commercial drug discovery budgets).
Rare diseases may be better targets even from a Machiavellian economic perspective. Wait…really. Yes. The largest expense for drug discovery are the phase 3 clinical trials. Rare diseases by definition have small patient populations which generally will correspond to less expensive clinical trials with faster timelines. Once a drug is approved by the FDA or similar regulatory bodies, it is relatively easier (and cheaper) to expand from the beachhead market. Gleevec was initially approved for a niche market and now provides over a billion dollars annual revenue for Novartis. When everyone is zigging, sometimes it is smart to zag.
So please think about developing new therapies for rare diseases, not just as a good thing to do for the people (and families) with rare diseases, but as a holistic strategy for doing drug discovery, understanding biological diversity…and well, collaborating.
CDD has a long history supporting neglected disease drug discovery and for similar reasons we are passionate about rare disease drug discovery. CDD has a number of technologies for collaboration and drug repositioning which may be helpful for certain projects. Please contact us directly (Barry Bunin: [email protected]) if you have an interesting scientific approach or collaboration (or are in need of them) for a potential therapy for a rare disease. If we can help or make the connection that can help, we will. Even if we can’t help directly, we may know the person who can help or provide some hope or progress towards the ultimate goals.
Of course it is not just about CDD. There are many others are working on rare disease drug discovery and collaborations. For two quick links, see:
- The Global Genes Project: (Contact Nicole Boice – President & Founder, [email protected])
- And the NIH office of rare diseases: http://rarediseases.info.nih.gov/Default.aspx
This blog is authored by members of the CDD Vault community. CDD Vault is a hosted drug discovery informatics platform that securely manages both private and external biological and chemical data. It provides core functionality including chemical registration, structure activity relationship, chemical inventory, and electronic lab notebook capabilities!
CDD Vault: Drug Discovery Informatics your whole project team will embrace!