Thursday, October 10, 2013
New Drug Approved for PAH and CTEPH Patients in U.S.
PHA President Rino Aldrighetti says, “Treatment opportunities for patients are growing rapidly. Eighteen years ago there were no treatments for pulmonary arterial hypertension. Twelve years ago there was one. Today there are ten, with a possible eleventh later this year (Actelion’s macitentan). Of the 7,000 rare diseases, only three have as many or more treatments available. We are fortunate to have a strong and growing research community with great communication and cooperation. That has helped foster industry interest in exploring new options for patients.”
Better Prognosis for PH Patients
The strong progress in the field of pulmonary hypertension over the past two decades has led to a better prognosis for PH patients. Whereas physicians treating PH once delivered terminal diagnoses, they now expect their patients to live with their condition. Many patients survive for years -- even 10-20 years or more -- with proper PH treatment.
Patients Not Receiving Treatments in a Timely Manner
“The problem we now face is getting these treatments to patients sooner. While we have made huge strides over the past 20 years, the time it takes to reach a correct PH diagnosis has not changed,” explains PHA Vice President for Community Engagement Mollie Katz. “With missed or late diagnosis, the disease continues to get worse, affecting patients’ survival and quality of life. Many patients will see three or more different physicians over a three year period before they are properly diagnosed with PH, and almost three-fourths have advanced PH by the time they are diagnosed. Because PHA sees early diagnosis as a huge priority, second only to a cure, we are working with medical professionals and patients to improve diagnosis through our early diagnosis campaign, Sometimes it’s PH.
Not There Yet
While there are 10 treatments for pulmonary arterial hypertension (PAH) and now one for CTEPH, there are none approved for pediatric patients. PHA has launched the only pediatric PH research program in the United States, the Robyn Barst Pediatric Research and Mentoring Fund.
And, says Rino, “Ultimately, our goal is a cure. With increasing numbers of researchers engaged in more studies every year, we are hopeful and encouraged as we see that hope turning into action.”
Learn more: FDA decision | riociguat study findings | PH treatments in the U.S. | PH treatments worldwide | Sometimes it’s PH website