22  Conclusions

I truly hope this text has provided you with further insights into how and why we need to better quantify D-E-R at both the Population and Individual level, and why we need to refocus drug development on individual patient outcomes.

If it has led to you using an improved design for your program and/or dose-ranging trials, please let me know. It would be nice to know it had a positive effect somewhere.

Equally, if you disagree with anything written herein, please also let me know. On many topics in drug development my views have evolved (changed!) over time through further learning and insights provided by friends/colleagues. For example, I initially believed that our goal was to find “the” optimal dose regimen; thus to determine the Population D-E-R relationships for both efficacy and safety/tolerability, and then select the dose which yielded the best “trade off” between these benefits and harms. It didn’t occur to me that this is only the best strategy if we must only pick a single dose for approval. I am now much more aware that different patients need different doses, and simply forcing all patients to take the same dose will inevitably lead to unnecessary and avoidable under-dosing and over-dosing for many patients. I also now view safety data more through the lens of the patient, and see their suffering as a failure of the sponsor/team/myself to adequately tailor the dose for them.

If we are to make real progress in changing drug development for the better, we all need to be willing to “change our minds” as we transition from our old ways to a new, patient focused, drug development paradigm. I see this as a measure of the strength of character of an individual, and am very fond of this (mis) quote attributed to John Maynard Keynes:

“When the facts change, I change my mind. What do you do?”

Although it is doubtful that Keynes actually said this, the quote is both excellent and clear. I would like to think my views on drug development have changed as I have acquired more “facts”, and feel no shame in saying so.

I will leave you with two provocative options:

Are you a part of the problem, continuing to accept fixed-dose regimens because you are very familiar with “simple”, even though this leads to worse patient outcomes?

Or are you part of the solution, prepared to embrace Personalised Dosing because you truly care and want the best possible outcomes for our patients (even if the path looks a little “scary” and unfamiliar)?

Our path to Personalised Dosing may not always be straightforward, and we will all need to better learn how to best implement it across all therapeutic areas. However fundamentally it is the right thing to do; be an advocate, speak up, and collectively our scientific arguments will prevail and convince the “simple” crowd to join us.

I am sure patients will appreciate our (imperfect) efforts to truly learn how best to deliver Personalised Dosing and achieve better patient outcomes; they are also entitled to be very angry with us when they suffer terribly because we persist with “simple” fixed-dose regimens. Remember, patients are not fields!

So let us move forward, and truly implement Drug Development For Patients.