Precision/Personalization, Segmentation, and Incidence/Prevalence

Introduction

We’ve all heard that cancer is not one disease, but many. Most diseases have subtypes. Taken to the logical extreme, each patient is different. A few decades ago, the paradigm was, “treat all patients the same, and work on highly prevalent diseases.” Today, we understand the power of precision medicine. Determining just how personalized a treatment should be, however, is non-trivial.

What

The first step in answering “Who is the Patient” is to define an indication, and investigate the known subtypes of the disease, comparing your therapeutic concept to its likelihood of being safe and effective in each subtype. Often the nature of a therapy demands new ways to segment patients, which will likely benefit from coincident development of companion diagnostics, or at least biomarkers. Understand the incidence and prevalence of the applicable subtype(s), and trends over time (and ideally the drivers of those trends) for each.

When

This is the first step in conceptualization of a development plan. If you don’t know where you are going, any road will get you there.

Why

Knowing – in detail – for whom you’re developing your therapy allows you to understand the actual unmet medical need, how a therapy will change the standard of care, what aspects of the current (and future) standard of care are inviolate, what your patient(s) care about, how they and their caregivers make decisions, how easy it is to find 3 patients, how easy it is to enroll them in clinical trials, and many other crucial contextual learnings.

Learning Goals

Understand how to develop and execute a precision medicine strategy, if, when, and how to engage with patients and their caregivers, how to segment/subtype general patient groups, and how to determine accurate incidence and prevalence data and trends.