Stephanie Fox-Rawlings, PhD, National Center for Health Research, July 25, 2019
Thank you for the opportunity to speak today on behalf of the National Center for Health Research. I am Dr. Stephanie Fox-Rawlings. Our Center analyzes scientific and medical data to provide objective health information to patients, health professionals, and policy makers. We do not accept funding from drug and medical device companies, so I have no conflicts of interest.
There is a critical need for new treatments for SSc-ILD [systemic sclerosis-associated interstitial lung disease]. We all hope that Nintedanib [brand name OFEV] will help slow the rate of decline, but the data are not yet sufficient.
There is a statistically significant reduction in the decline in FVC [forced vital capacity; a measure of lung function] after 1 year for patients randomized to the drug. However, there are questions about whether it is clinically meaningful for patients. The questions about the impact on patients’ health is reinforced by the lack of improvements in secondary endpoints that measured patient-centered outcomes such as quality of life. If those modest changes in FVC were meaningful, we expect that the quality of life measures and other patient-centered outcomes should have also improved. They didn’t.
In addition, the evidence of efficacy regarding FVC comes from a single clinical trial. While the trial seems well designed to answer this question, randomizing over 570 patients to drug and placebo and following them for 52 to 100 weeks, it is still a single trial. Replication is the key to scientific evidence. Independent trials could have a smaller or larger effect due to differences in the demographic or treatment profiles of patients or other factors.
It is important to have clear evidence that this drug slows decline before approval. That will take additional studies. It may be that studies need to be longer or that this drug is only beneficial for certain patients. It is essential to have that information before approval, because once a drug goes on the market, it is often impossible to compare to placebo. Clear evidence of efficacy is especially needed because this drug has risks, and patients and their doctors should have enough information to weigh the benefits and risks in order to decide whether or not to try it.
We understand the desire to approve treatments with more uncertainty for conditions without good treatment options. However, approving drugs with questionable efficacy raises the cost of health care, potentially exposes patients to risks without the possibility of benefit, discourage the development and scientific evaluation of new and more effective treatments, and can prevent patients from seeking treatments that do work. To continue to be the “gold standard” for approval, FDA needs to maintain high standards of evidence for approval.
Thank you for considering our analysis of the data for this product.
The committee vote on whether nintedanib should be approved to treat SSc-ILD was split with 10 voting for approval and 7 voting against.
FDA approved the new indication for nintedanib to slow the rate of decline in pulmonary function in patients with SSc-ILD in September 2019. You can read FDA’s news release here.