Author: Matthew Reiss, MSE, PhD, Manager, Precision Medicine & Navigation, GO2 for Lung Cancer 

The first anti-cancer therapy, mechlorethamine, was approved in 1949. Since then, the U.S. Food and Drug Administration (FDA) has worked to ensure that new lifesaving therapies reach those who need them most. 

But what does it mean to be “FDA-approved?” Do all drugs have the same approval process? What is the FDA doing to get the newest therapies to market as quickly as possible?  

The following information explains the basics of the FDA’s approval process to understand how new cancer drugs become available to the public. 

Note: This information refers to the drug approval process for the United States only. 

FDA Approval Pathways 

Standard approval 

The ultimate goal for all new therapies is full, standard, or traditional FDA approval. These types of approval mean that based on data and research, a drug’s potential benefits for the intended treatment group outweigh any possible risks the drug may have.  

When assessing a new drug, the FDA will often evaluate it compared to a currently available “standard of care,” which means a treatment that is generally considered to be the best treatment for a specific disease. The benefit-risk analysis for a new drug may differ from expert to expert, but the FDA uses the best available scientific information to make a final decision on a drug’s approval. A drug given standard approval for a specific use can be used for the disease without any restrictions. 

The process of receiving standard approval is time-consuming and expensive. The average drug takes about 10 years and hundreds of millions of dollars to reach full approval. Only 10 percent (1 out of every 10) of drugs will make it through the process. For people living with serious diseases such as lung cancer, this makes it challenging to get new therapies to people. Fortunately, the FDA has developed several additional ways to help bring promising new treatments to people sooner—without sacrificing safety and effectiveness. 

Accelerated approval 

There are times when the FDA can speed up the approval of a new drug. The “accelerated approval” pathway often allows promising new therapies for serious conditions to get to the public sooner, especially if they show a significant benefit over existing treatments.  

The FDA will sometimes grant accelerated approval based on early trial data that shows the drug has a good chance of working, even though it may be less certain than if given a full approval. Accelerated approval is particularly helpful in developing cancer therapies that look at treatment over long periods of time. This allows treatments that seem promising to become available sooner.  

Drugs granted accelerated approval can be given to the public but are under increased monitoring to verify and describe the drug’s benefit in the real world. Many of these drugs receive standard approval once more data supporting their use is collected. The FDA may also withdraw a drug’s approval if the data fails to show that the drug has a beneficial effect or if the company fails to meet FDA guidelines. Over the past few years, several targeted therapies for non-small cell lung cancer (NSCLC) have received approval through the accelerated approval pathway.  

Drug development designations 

In addition to the standard and accelerated approval pathways, the FDA has also developed some special designations or approaches to encourage the development of drugs for diseases with unmet needs. They tend to focus on diseases that don’t have suitable treatments or on therapies that have significant improvements compared with currently available therapies. A drug may have one or more special designations and should support the evaluation of the drug’s risks and benefits as quickly as possible. 

Breakthrough therapy 

A breakthrough therapy designation identifies drugs intended to treat the most serious health conditions (such as cancer) and drugs where early clinical evidence shows a significant advantage of the new therapy over the current standard of care. Examples may include: 

  • A substantial increase in the drug’s response duration (how long it works),
  • Significant improvements in a drug’s safety,
  • OR a clear improvement of the drug’s clinical benefit compared to existing options.

The drug company must request a breakthrough therapy designation from the FDA to help expedite the development and review process. The FDA may also suggest that a company apply for breakthrough designation if the initial results look promising and the drug meets the FDA’s criteria. When granted, this designation allows for increased FDA guidance to help collect evidence to support approval and bring the drug to market as quickly as possible. 

Fast-track 

The fast-track designation identifies drugs that fulfill an unmet clinical need for a broad range of serious diseases with promising pre-human data. This data may show that a new drug could prevent the disease from becoming a more serious condition (improved effectiveness) or that the drug can significantly improve quality of life. Drugs developed for diseases without an available treatment can also receive fast-track designation.

Like breakthrough therapy, the drug company must request fast-track designation from the FDA to receive guidance in expediting the development and review process of new treatments. When granted, the company participates in additional meetings with the FDA to help design studies that could support approval. Fast track also removes some of the requirements put in place by the FDA to start reviewing an application earlier in the process. 

Priority review 

The priority review designation identifies drugs that the FDA intends to give increased attention and resources to when evaluating the drug for approval. Once a company has submitted all necessary materials for approval to the FDA, drugs with priority review receive a decision within 6 months. Priority review is generally granted to therapies with evidence of significant safety improvement and increased effectiveness for the diagnosis, prevention, or management of serious conditions, including cancer.  

Unlike other designations, the FDA determines if a drug receives priority review status based on its outlined criteria for each application. Companies may also request priority review. A drug designated as a “priority” does not change the standards used to evaluate the drug for approval. 

Regenerative medicine advanced therapy (RMAT) 

The newest designation a therapy may receive, RMAT, is a special designation that grants accelerated approval based on early trial data for treating serious medical conditions.  

To qualify for RMAT designation, a therapy must meet a strict set of qualifying criteria, including: 

  1. The treatment must meet the definition of a regenerative medicine therapy,
  2. The treatment must be intended to treat or cure a serious disease,
  3. AND the treatment must have supporting early data that the therapy addresses an unmet need. 

Only specific types of lung cancer therapies are given for RMAT designation, including CAR-T cell therapy. Therapies that qualify for this designation benefit from all the advantages of accelerated approval and breakthrough therapy, with increased FDA cooperation to support expedited FDA approval. To date, no lung cancer therapies have received FDA approval through this pathway.