FDA approval programs

The U.S. Food and Drug Administration is the agency that approves medicines for people to use in the United States. Companies must put new medicines through a series of studies before they can be made available to the public. The results of these studies are given to the FDA, which approves the medicine if they find the medicine works as the company said and it is safe for people to use.

Studies can take years to prove the effects of a medicine. For example, researchers may need to watch how well a new medicine for breast cancer works over 5 or 10 years before the findings are thought to be proven. The FDA’s review adds more time to the process, but the agency does have programs to help important medicines move through the process – and become available to people who need them – more quickly.

To qualify for one of these programs, a medicine has to do one of three things:

• Provide help for a condition that has no or few other treatments
• Get much better results than available medicines
• Cause less serious side effects than current medicines

The FDA may work closely with the companies that make medicines that fit these requirements, or offer ways to get fast responses from the FDA to questions and applications so the medicines can be made available to the public sooner than they usually would.

Fast track

The fast track process was designed for medicines that treat serious conditions for which there are no treatments or very few treatments available now. The company making the medicine must apply to the FDA to be put on the fast track. If approved, the researchers get:

• More meetings with the FDA for advice on study plans and collecting the right information
• Written advice from the FDA about what the agency would like to see in the design of clinical trials and other plans
• A rolling review process to get parts of the new drug application reviewed as the information is ready, rather than waiting for the whole application to be completed
• Ability to get accelerated approval and priority review, if the medicine meets the other requirements for these programs

The fast track encourages researchers to speak with the FDA often as they study the medicine so the agency can answer any questions right away. The goal is to have the study go through approval more quickly by taking care of any issues before the final application is submitted.

Breakthrough therapy

The FDA uses the breakthrough therapy designation to help medicines that may do better than the available options for treating a serious disease or condition. Like the fast track, the breakthrough therapy designation is meant to give a smoother and faster way to approval than usual for medicines that could help people with serious diseases like cancer.

A medicine recognized as a breakthrough therapy will get all the benefits of the fast track program plus:

• The chance to work closely with the FDA on a development program
• The promise from senior management at the FDA to help the process along

The FDA can ask the company developing the medicine to apply for a breakthrough therapy designation if early results suggest it will meet their requirements and the designation will help it through the rest of the approval process.

Priority review

Priority review directs the FDA’s attention to medicines that may offer “significant improvements” in treating serious conditions. A significant improvement may mean:

• Better outcomes than current treatments
• Fewer or less serious side effects
• More people staying on the medicine, which leads to better outcomes
• The ability to treat a group that can’t take current treatments

Where fast track and breakthrough therapy designation can help researchers in the planning and trial parts of the process, priority review focuses on getting the medicine through the approval line faster once the studies are done. The FDA normally starts its review within 10 months of receiving an application. But for priority review, the FDA will start its review within 6 months.

Accelerated approval

The FDA can grant accelerated approval to medicines that treat a serious condition when there is no current treatment available. The usual approval process can take years to study the effects of medicines on survival times or how often cancer returns.

Accelerated approval allows some medicines to be approved based on “surrogate endpoints.” These are not the outcomes the studies are designed to see but signs that the medicine is working toward those outcomes.

For example, researchers may want to see if a medicine will make someone with a cancer that has no effective treatments live longer. The study may look at how many people have lived without cancer spreading at 5 or 10 years. But the FDA may give accelerated approval after 2 years based on findings that the medicine is safe and it causes tumors to shrink, a sign that the medicine may work to extend people’s lives.

Using these outcomes can get medicines into the hands of people who need them years before they might have otherwise and may save or extend lives. Researchers will still have to hold trials to confirm the clinical benefits of the medicine after an accelerated approval and the FDA can take back approval if the final results do not show a benefit.

Real-time oncology review pilot program

The FDA created the Real-Time Oncology Review Pilot Program to approve new treatments faster while ensuring the treatments are safe, as well.

The pilot program is similar to FDA’s existing breakthrough therapy designation, which helps medicines move through the approval process and get to the people who need them more quickly. The agency hopes it will help them find ways to streamline the review and approval process even more.