Once there is a drug that has been developed, it is very clear why talking with patients and collaborating with them is useful for pharmaceutical companies. What is less obvious to both companies and patient organizations is how patients can be active in research before there is any drug in development, during the preclinical phase, and how this might lead to medicines being developed. Some patient organizations might start by funding some academic group, but without a clear vision and a longer-term strategy the return on those efforts will be compromised, and medicines will take longer to come. Here I want to outline the broader strategy that patient organizations can follow to advance research towards new medicines even before companies are working on it.

In the previous entry we discussed the many types of companies that get interested in rare diseases, each of them because of different reasons. If your disease field matches one of those business propositions you will get at the top of the list of interesting diseases for that company, but another part of the decision equation is time, or field maturity. Is this field ready for us to start working on it already The following are the questions that a company will often need to answer to be able to judge if the field is ready for them, too early for them, or maybe even too mature for them to get in.

Most patient organizations, no matter how small, start with the same mission: to raise awareness and funds for research in their disease. But what research are they doing? are they all starting in the same place? and do they all evolve to run the same type of research? In my experience there are many types of strategy and approaches that patient groups take, in particular when they are starting. What a patient organization means when they say “we are doing research”, therefore, hides many different possibilities. The article describes some of these types, and discusses their advantages and disadvantages.

INTRODUCTION TO THE SERIES - The field of rare diseases, where each disease affects only hundreds or thousands of patients world-wide, is leading this revolution. Their diseases were once orphan to medicine, too rare to receive sufficient attention and investment to move the needle. Today patients take ownership of their research fields, and move the needle themselves. They have become the center of the network and the expert in the room. They are impatient, not wanting to wait for pharma to take an interest in their disease. They are impatient patients and are leading an impatient revolution.