Gary Lyman, MD, MPH, is professor of medicine and director of Comparative Effectiveness and Outcomes Research at Duke University School of Medicine and the Duke Cancer Institute.

He is also a senior fellow in the Duke Center for Clinical Health Policy Research. Lyman has served on the Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee.

What is the Oncologic Drugs Advisory Committee?

The Oncologic Drugs Advisory Committee or “ODAC” reviews and evaluates data concerning the safety and effectiveness of drugs used to treat cancer. The committee makes recommendations to the FDA about whether or not to approve a drug for use by patients.

This includes new drugs as well as drugs already approved by the FDA for treating other conditions.

Can you explain the process for a new drug to be developed and then approved by the FDA for patients?

By the time a pharmaceutical company submits a drug application for approval from the FDA, it has often spent 10 years or more and millions of dollars to develop and test the drug. During that time, the company has conducted clinical trials and also has often met with the FDA, sometimes numerous times, to discuss the drug and the data.

Once submitted to the FDA, the drug application is reviewed. At that point, the FDA has the authority to make the final decision to approve a drug or not. The FDA can do so without asking for review and recommendation by ODAC. However, the FDA can choose to send the information about the drug to ODAC for its additional review, and it often does.

If a drug application is sent to ODAC, then members of that committee review the data. A hearing is then held, during which the FDA and the company make presentations and members of the public can ask questions.

After extensive review and discussion, members of ODAC vote on recommendations to the FDA anonymously to avoid any perceived influence. The FDA usually follows the advice of ODAC on new drug approval, any change in approved indications and any requirement for further safety monitoring.

Who serves on ODAC?

The committee consists of 13 voting members including the chair. Members and the chair are selected by the commissioner of the FDA and are considered authorities knowledgeable in the fields of general oncology, pediatric oncology, hematologic oncology, immunologic oncology, biostatistics, and other related professions.

The committee includes a patient advocate and also a non-voting member associated with the pharmaceutical industry.

How long does it normally take a drug to go through this final approval process with the FDA and ODAC?

It usually takes a minimum of six months, but it can be closer to nine to 12 months for some drugs to go through the review process once a final application is sent to the FDA.

Why does it take so long for a new drug to be approved by the FDA?

The drug approval process is lengthy and the rules and regulations change frequently. The FDA not only reviews hundreds of pages of documents but also conducts their own independent analysis of the data.

Many members of the public believe the drug approval process takes too long while others argue that there is not enough attention to safety. The FDA is committed to ensuring that the science behind the development of the drug is solid both in terms of efficacy and safety.

What types of things are considered when reviewing a drug for approval?

As a member of ODAC, I consider many things. For example, I consider side effects of the drug. I review the study design to make sure it is methodologically sound. Considerations include whether there are currently available alternatives to the drug under consideration.

ODAC members are specifically instructed not to consider the cost of the drug in our recommendations. This is different from many other countries, where the cost of the drug is considered part of the approval process.

The role of an ODAC member is difficult because you don’t want to hold drugs from any patient who may benefit, but you also don’t want to recommend approval of an unsafe drug. It’s a balancing act -- need and benefit versus safety and harm.

What is accelerated approval of a drug and when does that occur?

This is a conditional approval. Essentially, accelerated approval can occur when there is enough evidence to approve the drug; however more information is desired to be certain of the level of benefit and safety.

This often occurs when clinical trials show very good results for the patients, and there are few alternatives for that patient population that might benefit from the drug. The pharmaceutical company is required to provide additional information to the FDA even after the drug is approved and prior to granting the agent full approval.

Do you see more drugs being approved faster in the future? Why or why not?

Since we want and need to ensure the safety and effectiveness of all drugs, we can only shorten the process so much.

Certainly, we will continue to see more and more cancer treatments submitted to the FDA for approval. Many of these drugs will be targeted therapies which have potential advantages but also present additional challenges.

Nonetheless, the ultimate goal of the FDA and ODAC is to get safe and effective drugs to patients with cancer as quickly as possible.