Pharmaceuticals

 Pharmaceutical Harm is more than just adverse reactions; it is a system-wide problem. There is a lack of transparency and questionable protocol that add 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. Pre-clinical: After a drug company discovers a new  compound, the FDA-approval process is started. The drug must first be  tested on animals to determine safety, effectiveness, and toxicity  levels of the drug. After compiling initial data, the company then  submits an Investigational New Drug (IND) to the FDA. The IND includes  results from the animal testing and a plan for human testing. It is at  this point in the process that the FDA makes a decision on the 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 certain diseases or conditions. They compare the drug against a placebo.

c.) Phase 3: Researchers test the safety and effectiveness in a  larger number of people on different doses and varying combinations of  other drugs.

3. New Drug Application (NDA) review: Once clinical  trials end, the drug company submits a New Drug Application. 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 it reviews 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 which allowed the FDA to collect fees  from the drug manufacturers to fund the new drug approval process. It is  passed every five years, and Congress can add things to it. PDUFA  allows pharmaceutical companies to pay fees in order to fast-track a  medication through the approval process. It is likened to “approve now  and ask questions later”. The fast-tracking takes into account 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. Using the example of a cancer  drug, if a drug reduces a tumor, the FDA considers it effective even if  there is no clinical trial data showing that it prolongs your life.

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

Patient Safety Concerns About Pre-Approval Process

• Clinical trials can be too small to give proper estimation of adverse events.
• Clinical trials can be too short and do not account for dangerous adverse events that occur 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; uses not studied or approved by the FDA.
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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 in place to analyze large databases of patients 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 continue to unknowingly put themselves at  risk when taking medications prescribed by their physicians that they  assume to be safe.

The investigation revealed that reports over a one-year period  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 even  reported to MedWatch. Many patients don’t know they could report adverse  events or they 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 completely overhaul the system.

If you experience any adverse reaction or side effects, it is important to report your adverse reactions to the FDA’s MedWatch system. You can find information here on how to report.