“That will never be a drug!”

How tempting it is to say that, because it’s almost always true.  In this paper that I recently published, I tried to dispel some of the dogma and preconceptions of what an oral drug looks like. 

https://pubs.acs.org/doi/10.1021/acs.jmedchem.5c02863

The exercise of putting together these examples was enlightening to me of how many times I said myself “wow, that’s a drug…no kidding?”.  There are multiple examples in this paper, and I hope you will find it useful in your own work.  However, this is not meant to say that “Anything Goes!” (queue up the Cole Porter tap dance music…) because that’s also not true.   

Putting a compound in development is a big, risky venture…maybe it’s better to think about it if it was your money, would you do it?

I watched over time sometimes scientists talk themselves out of a molecule, but then a couple of years later, say, “you know, that old thing is not as bad as we thought. Maybe it’s not a Mercedes, but its a fine used car with low mileage and can get us from Boston to New York without breaking down. Why not?” Maybe you don’t like the car analogy, perhaps you prefer another analogy.

The feedback on this has been useful. Perhaps one of the most interesting pieces of feedback is that it does not take into account the commercial success of each individual drug. That is true, and I do understand the idea behind it, which is to say “OK, you can get drugs approved that have less than ideal properties compared to ideal standards, but can they compete on the market with other drugs that do not?” Although conceptually this is easy to understand, in truth it’s hard to compare since the sales of any drug is also highly contingent on investments in commercial sales force across organizations. The point in the article that I hope came through was that for orphan drugs and FIC drugs, it is more common to see higher doses and BID doses compared to drugs that are not in this category. To me this speaks to the value proposition that every program must weigh as they go through the journey of discovery and development. Does the profile I have provide a meaningful difference to patients compared to current standard of care? If so, will it be safe and well-tolerated?