The way that medicines are discovered starts in a lab, either at a university or at a pharmaceutical company, and when the discovery seems promising a pharma company picks it up and spends hundreds of millions of dollars to get it through clinical trials and into your nearest pharmacy shelf.
Until recently, patients only showed up in this process as volunteers for the clinical trials and as the final customer once the drug reached the market.
I’ve trained as a scientist, one of those that first discover things in the lab, but for the last 7 years I have been working with patient organizations. I have been able to witness (and join!) a true revolution in the way we discover medicines, with patients and patient groups taking a leading role from the very beginning. 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.
A keyword that has become mainstream in these recent years is “patient engagement”, with companies wanting to reach out to patient groups but not sure of what patient engagement would actually involve, what best practices apply, or how they can measure the value of those interactions. The industry is following the patients in this revolution, and learning from them.
I published a free eBook in February of 2017 called the #ImpatientRevolution that summarizes much of my learnings as a scientist working with rare disease patient organizations. It is written with the impatient patients in mind, and talks about how drug discovery works, what they can do to advance research from a patient organization, and how they can engage with the pharmaceutical industry. That´s right, there is much to say about “corporate engagement” as well.
Over the past two years I’ve found myself in many conversations with groups of patients and caregivers that were just starting an organization and wanted to know the best way to do that. The eBook has been a very useful reference in all of these cases, but there is more content and more topics that are not necessarily fully covered in #ImpatientRevolution.
To give me the flexibility to keep adding new content that will serve as a reference for many future conversations, I have decided to start a series of articles called the Impatient Series, and publish them here under Dracaena Report.
Some of the topics I want to include are:
What patient organizations mean when they say they are doing research (different formats that I’ve seen, when to choose one or another)
What patient organizations can do to advance preclinical research, even before the pharma industry gets interested in their disease.
How to make research funding decisions (different formats, when to choose one or another)
How to evaluate and track projects to make sure your money is well-spent.
What makes pharma companies choose one disease vs another one.
What are the types of drugs that could treat rare diseases and what research is needed for each of them.
Working with the pharma industry: NDAs, trust and more.
I also want to address a tricky topic that often comes up when a new patient group finds me and asks me for advice. Their questions are usually followed by “can we hire you to guide our research efforts?” and my answer is invariable no, I can give you some strategic advice but you need to find someone else (I´m only one person with 24 hours in each day). This is usually followed by “where can I find another scientist that could work with us?” and I don’t have an easy answer for that. We are not talking about your usual scientific advisor that remains comfortably seated in their academic chair, but about finding a scientist with the knowledge and entrepreneurial instinct that patient groups need and who is willing to roll up their sleeves and step inside the world of patient organizations.
But I think there is a solution for this because in the last 12 months I’ve started to also come across the reverse situation, scientists who approach me to ask if I think working for a patient group would be good for them and how to find a good patient group. So I am very excited to be adding some new topics to these series that were never covered in the original #ImpatientRevolution Book:
How do I find a scientist that could work with our patient organization.
For scientists: what to consider if you think you might want to work with a patient organization.
…and more related questions to come
As a final note, it goes without saying that this is all personal advice and insights based on my very own experience. I look at the evolving landscape of social entrepreneurship in the patient community, followed by adaptation in the pharma industry worried to be left behind, and try to understand the model that explains it and the rules that it follows. Your experience might be different, and I would love to hear from you. Overall I hope the series will become both a useful resource and a door to dialogue.
Let’s start an #ImpatientRevolution!
Ana Mingorance, PhD
IMPATIENT SERIES ARTICLES:
#1 – WHAT PATIENT ORGANIZATIONS MEAN WHEN THEY SAY THEY ARE DOING RESEARCH
#2 – WHAT ARE COMPANIES LOOKING FOR WHEN CHOOSING A RARE DISEASE