Pharmaceuticals

 Pharmaceutical harm is more than just adverse reactions; it is a system-wide problem. A lack of transparency and questionable protocol adds to patient harm. Patients need to understand how medications are approved, regulated, and marketed.

The FDA’s Center for Drug Evaluation and Research (CDER) breaks down the approval process into the following phases:

1. Preclinical: After a drug company discovers a new compound, the FDA approval process starts. The drug must first be tested on animals to determine its safety, effectiveness, and toxicity levels. After compiling initial data, the company submits an Investigational New Drug (IND) to the FDA. The IND includes animal testing results and a human testing plan. At this point in the process, the FDA decides on risk vs. benefit.

2. Clinical Study: The drug company conducts testing on humans, and clinical trials consist of 3 phases.

a.) Phase 1- To discover the drug’s most frequent side effects.

b.) Phase 2- Researchers gather preliminary data on how the drug works in people with specific diseases or conditions. They compare the drug against a placebo.

c.) Phase 3: Researchers test the safety and effectiveness of more people on different doses and combinations of other drugs.

3. New Drug Application (NDA) review: Once clinical trials end, the drug company submits a New Drug Application (NDA). The FDA has  60 days to review the NDA before filing it. Once that happens, it reviews the research submitted by the drug company for safety and effectiveness and the proposed label. Lastly, the FDA inspects the facility where the drug is manufactured. It is at this point that the FDA either approves the drug or rejects it.

Prescription Drug User Fee Act (PDUFA)

During the New Drug Application review process, the drug companies may apply for Accelerated Approval, also known as  Fast-tracking. This is allowed under the Prescription Drug User Fee Act  (PDUFA). PDUFA was a law passed by the  United States Congress in 1992 that allowed the FDA to collect fees from drug manufacturers to fund the new drug approval process. It is passed every five years, and Congress can add things. PDUFA  allows pharmaceutical companies to pay fees to fast-track a medication through approval. It is likened to “approve now and ask questions later .”The fast-tracking considers what are called “surrogate endpoints” to determine effectiveness.

A surrogate endpoint is an indicator, such as medical testing (blood testing, scans), used to tell if a treatment works but does not guarantee its effectiveness. For example, if a cancer drug reduces a tumor, the FDA considers it effective even if there is no clinical trial data showing that it prolongs life.

The FDA’s funding comes from taxpayers, but nearly 50%  of drug approval funding comes from the drug companies themselves—the industry it should be regulating. PDUFA was passed to provide a budget for hiring more scientists to review the extra load of drug approvals. This raises many red flags for consumer groups and patient advocates, as there is a greater chance of adverse events post-approval when a drug is fast-tracked. Adverse reactions are not discovered promptly, which puts patients at risk. Faster does not always equal better.

Patient Safety Concerns About Pre-Approval Process

• Clinical trials can be too small to estimate adverse events correctly.
• Clinical trials can be too short and do not account for dangerous adverse events several years later.
• Some clinical trials can use healthy clinical trial participants and may not include a sicker population who would benefit from the drug.
• Physicians may prescribe a drug off-label, but its uses are not studied or approved by the FDA.

Patient Safety Concerns About Post-approval Process

• Adverse events are not always reported; they are voluntary.
• While the FDA can request a post-market study to follow up on safety concerns, the drug makers do not have to do them.
• Drug makers do not always publish unfavorable clinical trials.
• There is no system to analyze large patient databases for adverse events.

MedWatch: FDA Adverse Event Reporting System 

An investigation into the Food and Drug Administration’s (FDA)  adverse event reporting system reveals serious flaws that may delay the dissemination of timely information about risks and dangers associated with some prescription medications. Unless drastic changes to the system are implemented, patients may unknowingly put themselves at risk when taking medicines prescribed by their physicians that they assume to be safe.

The investigation revealed that reports over one year between 2013 and 2014 were either sent directly to the FDA through the agency’s MedWatch program or to drug manufacturers that, in turn, sent the information to the FDA. The report concluded that events reported through drug manufacturers were much less likely to contain complete information.

In addition, less than 10% of adverse reactions are reported to MedWatch. Many patients don’t know they could report adverse events or don’t connect the side effects to the medication they are prescribed. The reporting system is voluntary and relies on patient reporting; doctors and healthcare systems are not required to report.

Many drugs that are suspected to have serious side effects cannot be accurately monitored without a consistent and accurate reporting system. The only way to change that is to overhaul the system completely.

Suppose you experience any adverse reaction or side effects. In that case, reporting your adverse reactions to the FDA’s MedWatch system is essential. You can find information here on how to report.