Introduction

For therapeutics developers, there is no more important question than, “Who is the Patient?” Spend the time to understand as much as you can about the patient and their caregivers. How is the patient diagnosed? What are their symptoms? What complications are common? How are they treated today? What do they care about? What are their options? Where do they live? Can you (ethically) reach out and speak with patient groups? An investment in collecting this information first, has myriad benefits. First and foremost, you and your organization begin from a patient-centric perspective. It forces you to check your assumptions and yields invaluable context. Without a patient-centric plan, you will almost certainly make significant development errors, resulting in lost money, lost time, and lost impact. And if you aren’t sure which indication is right, or if you want to develop your therapy to treat multiple indications, multiply this effort by the number of indications you wish to investigate. No matter your decision, understanding your patient today is always a good use of time and resources.

For platform developers, i.e. companies that have broadly useful tools which may be applicable across indications, this can be complex. We still recommend selecting the “most likely to succeed” patient(s) and going through the patient definition process. A rigorous patient definition process provides knowledge for development, helps align investors to the mission, and creates a powerful empathic culture rooted in delivering better patient outcomes. The powerful connection to patients usually results in an empathic, caring, and highly motivated organization.