Most Physicians Do Not Fully Understand FDA Approval – What About Patients?

A study found that only 41% of physicians in the United States reported a moderate or good understanding of the drug approval processes of the FDA (Food and Drug Administration). For medical device approval processes, the figure was only 17%.

The FDA is a leading institution worldwide in the development, testing, and approval of new treatments. Although its decisions are binding only within the United States, they serve as an important reference and guide for other countries.

The FDA is one of the world's most influential health authorities, running a comprehensive approval process to assess the safety and effectiveness of new treatments, drugs, and medical devices. The process is designed to protect public health and provide patients with effective treatment options. FDA approval indicates that a product must undergo rigorous clinical testing and evaluation before entering the market, providing a safety guarantee for both healthcare professionals and patients.

FDA approval processes generally fall into three main categories:

• New Drug Application (NDA) for drugs,
• Biologics License Application (BLA) for biological products, and
• Premarket Approval (PMA) or 510(k) notification for medical devices.

For a new cancer drug, the FDA approval process would involve a “New Drug Application (NDA).” The NDA process requires comprehensive information on the drug's safety and effectiveness, generally in a detailed dossier containing results from preclinical testing (animal experiments) and clinical testing (human studies). 

Each process has different requirements depending on the product type and intended use. For example, drugs generally require extensive clinical trials, whereas the process required for medical device approval varies according to the device's risk level.

The variety of FDA approval processes helps healthcare professionals and patients access appropriate treatments while encouraging the safe, effective development of new therapies. Their complexity may also prevent physicians and patients from fully understanding them. A proper understanding supports informed healthcare decisions and helps improve treatment outcomes.

How Does a Cancer Drug Obtain Approval?

Cancer drug development is a lengthy, costly journey. Each step aims to provide safe, effective treatments by carefully assessing the drug's potential benefits and risks. Some brief, anecdotal information about this process follows:

• How Long Does It Take? The journey from discovery of a cancer drug to FDA approval generally takes 7 to 15 years. It includes discovery, preclinical studies, clinical trials, and the final approval stages.
• Cost: Developing a cancer drug can cost billions of dollars. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), the average cost of discovering and bringing a drug to market is approximately 2.6 billion dollars. This includes failed trials and high research and development expenses.
• Success Rates in the Approval Process: The FDA approval rate for cancer drugs is low. Approximately 5-7% of drugs entering clinical trials receive FDA approval. This reflects the challenges of cancer research and the need to demonstrate the safety and effectiveness of new treatments.

• Expedited Approval Pathways: The FDA offers various expedited programs to accelerate treatments, particularly for serious or life-threatening diseases. Programs such as Breakthrough Therapy Designation and Accelerated Approval allow faster evaluation of drugs meeting specific criteria. However, follow-up studies are still required after marketing to monitor effectiveness and safety.
• Clinical Trials and Efficacy Endpoints: Cancer drug development generally uses outcomes such as overall survival rates or disease-free survival to assess effectiveness. In some cases, however, surrogate endpoints (such as tumor shrinkage) can allow potential benefits to be assessed earlier.

These anecdotal details highlight both the challenges of cancer drug development and why it must be conducted rigorously. As each new cancer drug undergoes approval, the difficulties encountered in development and evaluation offer important learning opportunities for healthcare professionals, researchers, and patients.

How Well Do Physicians Understand FDA Processes?

A study published in Health Affairs on 8 January 2024 found that many physicians lacked a sound understanding of FDA approval processes. Researchers surveyed oncologists, internists, and cardiologists in the United States to assess their understanding of drug and medical device approvals.

Of the 478 physicians who completed the survey, 41% reported a moderate or good understanding of drug approval and 17% of medical device approval. Oncologists understood drug approval better, whereas cardiologists better understood device approval.

What Do Physicians with Limited Understanding Think?

Despite limited understanding of approval processes, most survey respondents said the FDA's current approval criteria were “about right” for both drugs (59%) and devices (66%).

Most respondents said the FDA should require 2 or more randomized controlled trials for most new drugs and devices (57% for drugs and 60% for devices). However, 68% of oncologists thought a single randomized controlled trial was sufficient for most new drugs.

Almost all respondents (98%) said the FDA should require follow-up studies when drugs or devices are approved based on surrogate endpoints. However, only 63% said they knew what a surrogate endpoint was.

Surrogate endpoint: using an easier-to-measure indicator instead of directly measuring a treatment's effect. For example, a cancer treatment's effectiveness can be assessed through objective response rate (a surrogate endpoint) rather than overall survival. Overall survival is considered the most reliable endpoint for assessing cancer treatment effectiveness. However, obtaining survival data can take a long time and delay evaluation of new treatments. Surrogate endpoints are therefore used to observe results over a shorter period and assess potential effectiveness earlier.

The researchers concluded: “In this national survey, we found that many physicians did not understand the level of evidence required for FDA approval and expected or believed that it should be more stringent, particularly for drugs and medical devices marketed through expedited pathways.”

What Do These Findings Mean?

This research reveals gaps in physicians' understanding of FDA approval processes. These gaps may affect their knowledge of drug and device safety and effectiveness when making treatment decisions for patients.

Further research should identify the best ways to improve physicians' understanding of FDA approval processes. This may help patients receive safer, more effective treatments.

What Cancer Patients in Turkey Should Take from This Article

• The FDA is a leading institution worldwide in developing, testing, and approving new treatments. Its decisions are binding only in the United States, but serve as an important reference and guide for other countries.
• The FDA has a rigorous approval process to ensure that drugs and medical devices are safe and effective.
• The FDA approval process is not always perfect, and some drugs or devices may later be withdrawn from the market.
• It is important to make treatment decisions with your physicians, weighing the risks and benefits of each drug or device.

Additional Information

• In Turkey, drugs and medical devices are approved by the Turkish Medicines and Medical Devices Agency (TİTCK).
• TİTCK has an approval process similar to that of the FDA.
• Patients can visit TİTCK's website for information about drugs and medical devices.