by Dr. Femida Gwadry-Sridhar
Have you ever considered the connection between drug development for rare diseases and olive trees in Italy? I assure you, there is one.
In September, I had the honor of presenting with a panel of peers at a Rare Revolution webinar on early access programs (EAPs). These programs allow patients with severe illness early access to drugs still in development.
At the webinar, I was speaking about real-world data and the value of EAPs. The topic reminded me of a very specific situation concerning olive trees in a very specific region in the world.
The olive trees in Puglia, Italy, were infected about eight years ago with a bacteria, Xylella fastidiosa pauca, that ultimately kills the trees. It is believed to have begun to spread in 2008 when a single infected coffee plant from Costa Rica was introduced in Italy. Today more than 30% of the 60 million trees are affected.
I thought about the parallels with human experience. We may have isolated the gene variation or pathogen and therefore know the cause of a disease, but this is not enough to develop a cure. It takes years if not decades to develop new drugs, and they do not all make it through the clinical trials.
However, for the drugs that do make it to clinical trials, the requirements for patients to participate are often stringent, which will not reflect the drug’s use in the real world. This is where EAPs come in: they provide drug companies real-world data about a drug’s effectiveness and outcomes from a broader population than a clinical trial can.
Not unlike the researchers seeking answers for the olive trees that have had no cure for most of the last decade, we cannot lose hope in our search for cures for the over 7,500 rare diseases out there. The trees are receiving transplants from healthy tress and healthy roots are trying to get hold. Likewise, new therapies for rare diseases are testing innovations in the hopes that one will work.
EAPs can help test those innovations precisely because they allow a broader range of participants to experience a drug than a clinical trial can. At the root of these branches of science—botany and medicine—are data. Collecting data to help better understand how to deal with the problem and the natural history and evolution of a disease in both humans and plants offers us more detail than we would have from doing nothing.
In the webinar, I pointed out that some of the failures we have seen in treatments have happened because we have not really understood the natural history of the disease. By collecting the appropriate data, we can build the proper natural history. EAPs help with this.
I am not a botanist, but I am a scientist. As such, I have much in common with the botanists studying the Xylella fastidiosa pauca in olive trees in Puglia, Italy. Perhaps the most important commonality is this: as critical as it is to study bacteria in laboratories, it is imperative to study them in the real world. EAPs give us this opportunity early on. Innovators in any field should take advantage of them wherever possible.
So, while the solutions to treating disease may seem far away, we have to be persistent and continue to look for answers in the science and continue to focus on elimination or control of the problem so that the next generation has something more to look forward to. Science is nothing if not a test of persistence.