As we look back to 2015 it has been a good year for pulmonary hypertension patients. We have made steps forward. We have an expanding number of medications to treat PAH. Patients are living longer and feeling better. We have taken further steps toward understanding the genes involved in the disease.
Despite our progress, we remain ineffective in answering some very basic questions about PAH, and we lack a comprehensive strategy for research and development. All of our approved medications work to either improve exercise capacity or delay clinical worsening. However, no one medication is effective for all patients. Some patients respond very well to one therapy and not well to others. We have made no progress in understanding how to match our available therapies with individual patient characteristics.
Much has been made about personalized medicine. This is the concept of making patient level decisions that optimally match treatments to each patient. In order to do this we need much better quality research. The community of PH doctors has been clamoring with increasing fervor for drug companies to collect blood samples and analyze the information in a way that allows us to choose the optimal medication regimen.
Unlike many other diseases where research studies involve many thousands of patients, most PH studies have a few hundred patients up to a thousand patients. Furthermore, as more community doctors are comfortable prescribing oral therapies for PAH patients, fewer patients are being referred to research centers. This has complicated the process of efficiently enrolling research studies.
Here is my Pulmonary Hypertension wish list for 2016
1. Clinical research studies will standardize the collection of blood and information to allow scientists to begin exploring how to choose the best treatments for each patient
2. The Pulmonary Hypertension Association should continue to expand their leadership in advocating for federal funding for PAH research
3. The expansion of treatment options that target new pathways
4. Improved diagnostic tests that allow us to monitor our patients’ progress
Last but not least, I would like nothing else than to be out of a job when we find a cure for PAH. Until then, we have much to do and many patients to help.