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In my previous role as the associate director of rare diseases at the FDA’s Center for Drug Evaluation and Research (CDER), I saw first-hand how companies that slowed down long enough to conduct robust natural history (NH) studies early and collect data that not only meet regulatory standards but help them generate evidence for payers get to market faster in the long run. Here’s why:
Why invest in a high-quality natural history study?
1. You’ll get more precise data on the target patient population and healthcare burden. Collecting detailed data on how a disease progresses will enable you to stratify patients in clinical trials. Then you can include patients at differing stages of disease in your clinical trial to seek a broader marketing label. Potential partners and buyers will place a higher value on your asset if the market is precisely defined.
2. You’ll start a valuable conversation among key opinion leaders (KOLs). When I was at the FDA, KOLs from around the world shared their experiences treating rare diseases in our Patient-Focused Drug Development (PFDD) public meetings. They were eager to learn from each other because there are so few patients with these conditions. Formal treatment guidelines don’t exist for many rare diseases and the process to create them is long and complicated.
These conversations alone helped improve and harmonize the standard of care. By gathering KOLs together in person or remotely to plan an NH study, you can build consensus around a standard of care (SOC) for the condition.
3. You’ll quantify the standard of care and how it differs between regions. To make regional arguments to payers, you need to understand the standard of care in every locality. If you don’t know how they differ, regional SOC variance can create statistical noise that makes it hard to interpret the trial results.
Getting the SOC right is essential because you must demonstrate an incremental improvement in the current SOC to make your payer argument. It’s difficult and expensive to go back and get the data after Health Technology Assessment (HTA) agencies ask for it. It is much easier to gather it in NH studies prior to clinical trials.
4. You train investigators and sites while fostering a clinical trial network. The KOLs in your NH study will likely be the future principal investigators (PIs) for your clinical trials. Centers of excellence for rare diseases often collaborate and share information during the study, creating relationships that will later help your clinical trial run more smoothly.
The bottom line: A good, early natural history study will help get your medicine to patients faster.
Prior to joining Parexel, I held several positions at the FDA, including Associate Director of Rare Diseases in CDER, Senior Medical Reviewer for Rare Diseases and Medical Genetics Division, and Team Leader and Senior Medical Reviewer within OND’s Division of Psychiatric Products. During my tenure, I managed an international regulatory program with EMA, Health Canada, and other regulatory bodies. I also co-authored inter-center and intra-center draft and final Guidances to Industry on drug development for common issues for rare diseases drug development, natural history studies, pre-IND meetings, rare pediatric priority review voucher programs, and eligibility criteria, among other specific rare disease guidances. Before joining the FDA, I held several positions both in the intramural and extramural program at the National Institute of Mental Health (NIMH).