What is the definition of a drug for humans?

What is the definition of a drug for humans

When is something a medicine, and when is it a medicine for humans? What are the risks?

Medicines are remedies for diseases that are based on an active substance, which are administered in a correct way, with the aim of having a specific effect on the physical body of the patient or on his soul. Since these chemicals are not part of our diet, an important phrase from history is important: “sola dosis facit venenum”. (Paracelsus)In this post, I give my own opinion, not that of any organization
Author: Manu Steens

Definition of drug for human use:

The European Directive (2001/83/EC) gives the following definition:

‘Any substance or combination of substances presented for treating or preventing disease in human beings. Any substance or combination of substances which may be administered to human beings with a view to making a medical diagnosis or to restoring, correcting or modifying physiological functions in human beings is likewise considered a medicinal product.

When is something a medicine for humans? And is a placebo a drug?

For a substance to be legally classified as a drug, it must undergo scientific validation and be approved for its intended purposes of diagnosing, treating, preventing, or relieving symptoms of a disease. It is important to follow all safety and efficacy guidelines and regulations before using any kind of drug.

Placebos, lacking active ingredients, aren’t drugs. In clinical trials, researchers use placebos as controls to differentiate the effects of actual drugs from the psychological benefits induced by belief in treatment. This helps to showcase the mind-body interaction in healing.

Determining factors:

Purpose: a medicinal product is a chemical substance or complex of chemical substances with an intended pharmacological, immunological, or metabolic effect on the (animal or human) body. (https://nl.wikipedia.org/wiki/Geneesmiddel)

The active ingredient is the chemical active substance or substances with the intended effect. It undergoes clinical tests, and the effect is shown to be more effective than that of a placebo. The effect may be symptomatic or curative.

Safety: medication safety is defined as minimizing drug-related problems, of which medication errors are an essential part. (Medication Safety).

Authorization: in the West, authorization through a series of tests is required to market a product as a medicine.

Rigorous testing for FDA and EMA:

To serve as a medicine for humans, the product must undergo a series of rigorous tests to demonstrate efficacy, dosage and safety, and quality. These tests are performed after other tests (on animals and sometimes organ baths) in so-called clinical trials in which the product is tested on human subjects. When found positive, these tests are intended to convince regulatory agencies such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) of the product’s effectiveness and safety at that dose, form, and route of administration. Then, it can be approved for human use, provided sufficient information is provided in a package leaflet.

In addition, there are manufacturing, labeling, distribution, and marketing guidelines to help ensure safe use.

Is a placebo a drug?

No, in the traditional sense, a placebo is not a drug because it contains no active ingredients. However, in some cases, it does work as a medicine because of the belief that the patient attaches to the drug’s effect. This is called the placebo effect.

Researchers often use placebos in comparative studies versus the drug candidate.

What types of medicines are there?

There are different types of medicines. The most commonly used classifications are those according to actual operation, mode of action, based on the application, and dosage form. I give a limited number of examples here for each of these.

  1. Classification based on operation:
    • Analgesics: Pain medications, such as paracetamol.
    • Antibiotics: Medications for bacterial infections, such as penicillin.
    • Antivirals: Medications for viral infections, such as acyclovir.
  2. Mode of action:
    • Medicines that treat symptoms do not cure the condition, such as painkillers.
    • Medicines that treat the cause of a condition, such as antibiotics, antidepressants, and chemotherapy drugs.
  3. Classification based on application:
    • Cardiovascular agents: Medications for the heart and circulatory system.
    • Diabetes medications: To regulate blood sugar levels, such as insulin.
    • Psychotropic drugs: Medications for mental illness, such as antidepressants.
  4. Classification by dosage form:
    • Oral medications: Tablets, capsules, drinks, and powders.
    • Topical medications: Creams, ointments, lotions, and sprays to apply to the skin.
    • Injectable medications: Injections into the muscle, vein, or under the skin.
    • Inhalation medications: Medications administered through an inhaler.
  5. Classification based on prescription:
    • Medicines over the counter: available without a prescription.
    • Prescription-only medicines.

What types of risks are associated with the different types of medicines?

The risks associated with various medications, a critical aspect of understanding drugs for humans, vary depending on factors such as the specific drug, dosage, length of use, the patient’s overall health condition, and potential interactions with other medications or substances. Though not an exhaustive analysis, the following discussion aims to outline a range of general risks that can arise from the use of drugs intended for human consumption.

Side effects are unwanted effects that can occur while taking a drug. They can be mild or severe.

Some medications can be toxic at high doses or with long-term use, which can cause damage to organs.

An overdose can cause serious health problems or even death.

Interactions with other substances, such as alcohol, food, or other medications, can affect the effectiveness of the drug or give rise to health problems.

Withdrawal symptoms when you suddenly stop taking a medication, resulting in multiple possible symptoms.

Some medications, such as opioids, can lead to addiction. This means that you may become physically or mentally dependent on the drug.

Development of resistance in microorganisms due to long-term use of certain medications, such as antibiotics or antivirals.

Sometimes, medications can trigger allergic reactions. This can happen with mild but also severe symptoms.

Crises Involving Drugs for Humans: Historical Overview

The Thalidomide Crisis:

Unknown individuals distributed thalidomide samples to doctors, who were referred to as “clinical investigators,” without obtaining permission or verification from Canadian authorities.

The U.S. Food and Drug Administration (FDA) at the time refused to approve thalidomide due to the lack of sufficient research. The FDA asked Richardson-Merrell to demonstrate that the drug was safe for pregnant women. The company has never provided this proof.

“The physicians who prescribed thalidomide, the pharmacists who dispensed it, and the patients who took it were entitled to the assumption that reasonable precautions had been taken by the appropriate federal government officials to ensure that the drug would not harm an unborn child.” (Report of the Thalidomide Task Force of Canada’s War Amputations, p.2.)

Around the world, in the late 1960s and into the early 1970s, the victims of the drug thalidomide and their families filed class action lawsuits. They ended up getting settlements.

Sometimes, large-scale scandals have to take place to gain insight and change.

The Vioxx Incident:

Shortly before the FDA approved Vioxx in 1999, drugmaker Merck launched a study that it hoped would prove Vioxx was superior to older painkillers. Instead, the study ultimately showed that Vioxx can be fatal and cause heart attacks and strokes.

In September 2004, Merck withdrew Vioxx after a study on the prevention of intestinal polyps showed that the drug increased the risk of heart attacks after 18 months. When the manufacturers withdrew Vioxx from the market, they had already sold the drug to around 20 million Americans. According to The Lancet, around 88,000 Americans experienced a heart attack, and of those, 38,000 individuals passed away.

The Opioid Crisis:

The opioid crisis refers to the mortality of patients in the United States who have died as a result of overdose or overuse of opioids. More than drugs such as heroin, the drugs involved are legal painkillers such as oxycodone, hydrocodone, fentanyl and tramadol.

The opioid crisis is a result of the overuse of these drugs. Patients could take these drugs more and more over time because they could obtain them outside of regular medicine circuit.

Addressing Unseen Side Effects in Drugs for Humans Post-Clinical Trials

If new side effects occur, you can take the following steps to address the situation and in that order:

  1. Stop using it.
  2. Contact your treating physician or the pharmacist from whom you purchased the medicine.
  3. You can report medication side effects directly to the drug manufacturer. They are required to investigate all reported side effects, even if they have already been reported to the healthcare provider.

Post-marketing surveillance programs collect data on the drug’s safety and efficacy through adverse event reports from healthcare providers and patients. It is important to keep monitoring for any new side effects that may occur and assess them for further action if necessary. This helps ensure the continued safety and effectiveness of the treatment.

Regulatory authorities like the FDA in the United States and the EMA in Europe play a pivotal role in ensuring the safety of drugs for humans. They accomplish this by issuing safety notices and warnings to healthcare providers and the general public about the potential risks associated with pharmaceutical products. These actions include modifying drug labeling and package inserts to highlight possible side effects, specifying precautions for particular demographic groups, and outlining recommendations for monitoring patients undergoing treatment with these drugs for humans.

Occasionally, severe or unexpected side effects may occur that are of such a nature that a reassessment of the approval is necessary. If the risk of and from the side effects exceeds the drug’s benefits, the regulatory authority may withdraw the approval.

Conducting further research is essential to gain a better understanding of the causes and risk factors. This can facilitate improvements to the drug by making adjustments to dosages, treatment guidelines, or administration methods, or even by developing new drugs with fewer side effects.

An example of side effects that would not have emerged during the tests was birth defects in the product marketed in the Netherlands under Softenon. (See also the thalidomide crisis mentioned above)

For some medicines, a side effect sometimes occurs very late or only in the next generation.

Conclusion: Understanding Drugs for Humans

The pharmaceutical sector, focusing on drugs for humans, operates at the intersection of immense potential and significant risks. Consequently, we have put in place a rigorous array of feedback mechanisms to verify the efficacy and safety of active ingredients before approving them for use.Despite these precautions, incidents and adverse reactions can still occur, underscoring the importance of cautious medication use. It is important to treat drugs for humans with caution and not take them casually like candy. There are numerous risks involved, including fatal outcomes, that should not be overlooked.

In addition to the risks mentioned above, malicious intent may also exist. An example of a mystery that was never fully solved was that of Johnson & Johnson with the painkiller Tylenol. In my opinion, the producer solved this setback for Johnson & Johnson as a textbook example.

Manu Steens

Manu works at the Flemish Government in risk management and Business Continuity Management. On this website, he shares his own opinions regarding these and related fields.

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