Finding a Roadmap to Approval – Hint: You Can’t Pick One Up at the FDA


  • January 31, 2018

Earning FDA approval for your drug program is a journey. A misstep at any point of that journey could jeopardize your chance at getting your drug approved. That’s why it’s so important to have a dependable roadmap that will get you where you want to go.

A misconception many sponsors have is that the FDA is a consulting organization to the pharmaceutical industry. Those sponsors think the Agency will provide them with a roadmap to approval. They couldn’t be more wrong.

In reality, the FDA is a science-based regulatory agency, operating with the expectation that each sponsor will come with a well thought out and justifiable plan that supports the safety and efficacy of their product. With a roadmap like this in place, the product has a healthy chance of getting approved.

What the Agency doesn’t want is for sponsors to be on a fishing expedition during meetings, hoping that the Agency will identify problems with their program and provide solutions. The FDA’s role is to protect the American consumer, not to guide sponsors through the drug development process.

Don’t Expect the FDA to Point Out Problems with Your Program

Some sponsors assume that, because the FDA didn’t raise an issue with their program during a meeting, they are pursuing the right path to obtaining approval. This is an assumption that should never be made. The Agency’s silence on an issue doesn’t mean that everything is okay. Things may be far from okay.

It’s up to the sponsor to think through their program, understand where the weak spots are, and decide what they are going to do about those weak spots. Contrary to what some sponsors may believe, the FDA has no obligation to point out problems within their program.

It’s not the Agency’s job to perform a gap analysis of the sponsor’s drug development program. It is also not their obligation to ensure that a sponsor’s program is successfully approved. Sponsors should not put themselves in the position of relying on the Agency to be the chief development officer of their program.

For sponsors, drawing an effective roadmap involves more than just identifying weak spots in their development program. During meetings, sponsors shouldn’t be afraid to address those weak spots head on, in a frank and open manner.

Because the objective is to get a clear understanding of the Agency’s position on matters relating to the drug being developed, it is crucial to ask questions that can be answered with a “yes” or a “no.” Binary questions such as “Do the Agency agree?” or “Is this acceptable?” are most likely to yield a response from the FDA that will be most helpful to drawing or revising a roadmap, and then following that roadmap to approval.

Path to Drug Development & FDA Approval in the US

FDA Guidance Documents are Not a Roadmap

It’s true that sometimes the FDA provides guidance on the development of drug products. This guidance is typically a short document that may recommend the type of studies that should be conducted and the analytics to measure.

Take, for example, a pain medication product. Having approved many applications for pain medications, the FDA knows exactly what sponsors need to do to get their pain medication approved. Because this is such a well-beaten path, the Agency can communicate precisely what data the sponsor needs to provide. This guidance only expresses the Agency’s thinking on the topic at the time of publication. Therefore, it is not a roadmap. Among other things, the sponsor still has the responsibility of putting data into the appropriate context of what purpose the data is intended to serve.

However, for lesser understood conditions such as autism spectrum disorder, the roadmap to approval is untested. That’s because autism, a disorder consisting of a group of conditions and symptoms, has not been as relatively studied and characterized. In this case, there is no published guidance on the topic. It is entirely up to the sponsor to draw their own roadmap.

Drawing Your Own Roadmap

Successful sponsors understand that the FDA is a science-based, data-driven organization. This means that sponsors must draw a roadmap that enables them to provide the Agency with the data necessary to get approval. Essentially, the Agency is conducting a benefit analysis when deciding whether to approve a drug. When this analysis is complete, there must be more checks in the “benefit” column than the “risk” column.

While you can’t get your roadmap to approval from the FDA, The Weinberg Group can help you establish your own. Our services help you escape the echo chamber of your organization by providing you with an independent, third-party view of your program.

We’ve organized our company as a drug development unit. This means we have all the major disciplines in place to take a holistic, 360-degree perspective on your program. So, we are able to speak scientist to scientist regarding every aspect of your program.

Getting approval from the FDA is a moving target. Because we interact with the Agency on such a regular basis, we have a finger on the pulse of what the Agency’s thoughts are. This is important because the Agency is not a static organization. It is influenced by such things as the latest health crisis and the priorities of the current FDA commissioner.

Let us use our knowledge, experience, and positioning to help you draw your successful roadmap to approval.