Rare Patient Voice President Wes Michael was interviewed recently by PharmaVOICE’s Kim Ribbink in the article, “Rare Patient Voice’s President is Striving to Create a Five-star Experience for Patients,” which was published on March 23, 2022. When asked why the patient voice is so important in the development and patient recruitment process, Wes provided a thought-provoking answer:
“Without hearing from patients, researchers can make wrong decisions. I remember being at a pulmonary fibrosis conference with patients, physicians and researchers. The researchers were developing treatments to save patients’ lives. The patients were grateful, but they said the cough was making their quality of life suffer. The researchers said the cough wasn’t what was killing them, but the patients pushed back and said they didn’t want to live with that cough. Ultimately, the researchers heard the voice of the patient and learned how important that facet of their quality of life was to them.
Another example is treatment for erythropoietic protoporphyria (EPP), a condition that causes patients severe pain on exposure to sunlight and some artificial light. One patient, who is also a researcher, noted that a treatment under investigation lengthened the time before the burning sensation began to happen. While regulators were saying this time duration wasn’t enough to help anybody, the feedback from patients led regulators to look at the data again and the product was approved.
The industry is learning to listen more and regulatory bodies are also asking for the patient perspective. It’s no longer enough just to consider expert opinion; increasingly it’s become important to engage patients and advocacy groups.”