Thank you for the opportunity to speak today. My name is Dr. Megan Polanin. I am a Senior Fellow at the National Center for Health Research, and I previously trained at Johns Hopkins University School of Medicine. Our research center analyzes scientific and medical data and provides objective health information to patients, providers, and policymakers. We do not accept funding from pharmaceutical companies, and therefore I have no conflicts of interest.
We realize that the goal of all five of the drugs discussed during this meeting are for treating rare pediatric cancers and that these patients desperately need new treatments. In some recent approval decisions, the FDA has been criticized for approving treatments for rare diseases based on inadequate data and wishful thinking. As a result, most insurance companies were not willing to pay for those approved drugs. Instead of getting free drugs in clinical trials, those patients are left with no affordable options. So, it is essential that the studies conducted are designed to be able to prove whether or not any of these 5 drugs have benefits that outweigh the risks.
We strongly support FDA Advisory Committee meetings like this one to garner input from experts on how best to design and conduct clinical trials for pediatric patients. This committee has been and will continue to be thorough in asking specific questions of the drug sponsors on their trial design while also offering helpful critiques and suggestions when needed.
Despite the challenge of studying these drugs for extraordinarily rare populations of patients, it is critical to uphold the scientific integrity of the proposed trials. For example, when possible, researchers should use randomized or well-matched control group/comparison samples for new drugs because it is the most methodologically sound design in order to demonstrate whether a drug is safe and effective. When a proper control group is not available, researchers should observe a robust single agent response rate in order to support a drug’s efficacy.
By law, FDA decisions are NOT based on cost, but you know that the cost of these drugs can be a huge issue for children and their families. In the last year, two drugs used to manage — not to cure — rare diseases were priced at $300,000 and $750,000 per patient, per year. As I noted, insurance companies are making sure these drugs are proven to work before they are willing to pay for them. You can help the FDA make sure that the evidence is clear.
When analyzing the proposed clinical trials, we urge this committee to keep in mind the possible pitfalls associated with using biomarkers and surrogate endpoints in lieu of outcomes that are most meaningful for patients such as overall survival and quality of life. A study published in JAMA found that only 14% of cancer drugs approved using surrogate endpoints were later determined to improve patients’ overall survival. Our Center found that only one of these drugs was proven to improve quality of life, and yet all were still on the market — costing an average of almost $100,000 per year. Let’s make sure that cancer drugs for children are proven to provide meaningful improvement in patients’ health or quality of life, if not both.
Additionally, there are clearly subpopulations of patients who respond better to treatment than others. We encourage the sponsors to further characterize the positive responders in order to target the population of patients who are most likely to benefit and least likely to be harmed by adverse events from their treatment.
Drug efficacy is a complex issue for children with chronic and/or rare diseases like the cancers discussed here. We commend the FDA and this committee for providing an open discussion focused on the best ways to test these five new drugs in pediatric populations. This marks a positive effort to help ensure that drugs are safe and effective for everyone for whom they are prescribed, particularly when the cost of these drugs can be so high.
Thank you for the opportunity to express our views.