The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are complex, we want our community to be knowledgeable about the burden of proof required to support an FDA-approved treatment, and how that approval process works. As we get closer to approved treatments, we understand and expect that the frustration in our community will increase.
To help address this need, this year’s Annual SMA Conference included several presentations on clinical trials, the NDA process, and issues that will impact access to a drug after it is approved, including insurance coverage and drug labeling.
Use the links below to download two of the keynote presentations. In the coming weeks, we will share additional presentations, videos, and other resources from the conference on these important topics.
In the United States, the Food and Drug Administration (FDA) is responsible for granting marketing approval for drugs. If a drug developer has evidence from clinical trials that a drug is safe and effective for its intended use, the company can file a new drug application (NDA). The FDA review team thoroughly examines all submitted data on the drug and makes a decision to approve or not to approve it.
When the FDA evaluates a drug for approval, they must weigh many different factors, including the quantity and quality of evidence of safety and effectiveness, potential benefits of a treatment versus the potential risks, and the impact the treatment will have on the patient community.