1.2: Drug Sources, Forms, and Names
By the end of this section, you should be able to:
- 1.2.1 Discuss drug sources and forms.
- 1.2.2 Explain drug standards.
- 1.2.3 Discuss the U.S. Food and Drug Administration’s drug approval process.
- 1.2.4 Define the chemical name, generic name, and trade name of drugs.
- 1.2.5 Explain the difference between a drug’s generic and brand name equivalents.
- 1.2.6 Differentiate between prescription and over-the-counter drugs.
- 1.2.7 Compare and contrast traditional drugs, biologics, and alternative and complementary drug therapies.
Drug Sources and Forms
Drugs are substances or compounds that prevent, treat, diagnose, or cure various conditions or diseases. As mentioned previously, drugs come from a variety of resources—plants, animal products, and inorganic substances. Ideally, these chemicals have desirable therapeutic effects without harmful properties, although many derivatives may have poisonous effects, depending upon the dosage used. Some plant-based products in use today include digitalis from the foxglove plant, vincristine from the periwinkle, and morphine from the poppy plant. The Amazon Basin is home to countless numbers of plants with medicinal properties. Though not in use today, one of the first paralytic agents used in anesthesia was curare, made from a vine known as Chondrodendron tomentosum , found near the Amazon River in South America. The Indigenous population in the area placed a curare mixture on the tip of blow darts and used it for hunting (see Figure 1.3). The prey is paralyzed once curare enters the bloodstream; however, the meat is safe to eat because it is not toxic when eaten and digested.
Vy states that she has been taking 1000 mg of acetaminophen every 4 hours per day for the last 48 hours. What is the priority nursing intervention for the client?
- Draw liver enzymes immediately.
- Educate the client about dosage recommendations for acetaminophen.
- Notify the health care provider to recommend initiation of hydrocodone.
- Immediately assess the client’s bowel function.
- Answer
-
Educate the client about dosage recommendations for acetaminophen.
The nurse is discussing the use of over-the-counter medications with Vy. What advantages of using over-the-counter drugs will the nurse discuss?
- Over-the-counter drugs do not need to be reported to the provider.
- Insurance companies will reimburse the cost of over-the-counter medications.
- Minor ailments can be treated by the client rather than the prescriber.
- Over-the-counter drugs are always safe.
- Answer
-
Minor ailments can be treated by the client rather than the prescriber.
Drug Standards
The FDA is the government agency responsible for the regulation of the development, production, and sale of drugs. The FDA was given much closer control over the production of drugs after the drug thalidomide was prescribed to pregnant clients for the treatment of morning sickness and for sedation in the 1950s and 1960s. This drug was found to be highly teratogenic to fetuses and caused many fetal malformations, especially limb defects. The Kefauver-Harris Amendments in 1962 required drug manufacturers to establish the efficacy of drugs and gave the FDA more control over the testing of drugs before they were placed on the market.
The United States Pharmacopeia National Formulary (USP-NF) has a rich history, dating back to the inaugural publication of the United States Pharmacopeia in 1820. In 1888, the National Formulary (NF) joined its ranks. Then, in 1975, these two entities merged to form the formidable USP-NF. This institution operates as an independent, nonprofit organization dedicated to establishing vital standards for compounding medications, biologics, drug development, and manufacturing, both within the United States and globally. It produces the only official book of drug standards in the United States annually, and drugs referenced in this book have met very high standards for quality, strength or potency, and purity (U.S. Pharmacopeia, n.d.). The USP-NF is a nongovernment organization and is not associated with the FDA; however, the standards that the USP-NF puts forth are enforced by the FDA as the official standards for the production and quality control of drugs and dietary supplements in the United States. Complementary medications and supplements are not required to meet these same standards, though some do, and they carry a USP seal guaranteeing safe manufacturing and quality. The Canadian Food and Drugs Act also recognizes the USP-NF as a reliable authority of drug standards in Canada for health care providers.
There are many reliable sources of drug information for health care providers, including the American Society of Health System Pharmacists’ AHFS Drug Information book and Drug Facts and Comparisons. Many online resources are available to nurses, such as Medscape, Skyscape , DailyMed , and UpToDate . Applications can be downloaded to personal electronic devices or devices in the clinical setting (though not all are free). Often hospitals/health care systems and clinics have institutional access to these resources. It is helpful for the nurse to be able to access a wide variety of resources. A useful and free app for herbal supplements is About Herbs , which is produced by Memorial Sloan Kettering Cancer Institute. It is particularly helpful in the discussion of the mechanisms of action, herb–drug interactions, and adverse effects.
Drug Approval Process
The Center for Drug Evaluation and Research (CDER) is a branch of the FDA that evaluates new drugs before they can be sold in the United States. It provides health care providers and consumers with the information needed to use drugs appropriately. One of the tasks of CDER is to ensure that both brand-name and generic drugs work as they should (FDA, 2022b). Before any drug is sold in the United States, it must be tested. Once a chemical that may have therapeutic effects is isolated, it will undergo scientific tests and clinical trials to prove its efficacy and safety. Drugs must pass through several stages of development before receiving approval from the FDA to be marketed to consumers. Table 1.2 briefly describes the process of obtaining FDA approval for a new drug.
| Stage of Development | Description |
|---|---|
| Preclinical trials | Drugs are tested on animal subjects to evaluate the compound in living tissues and to evaluate for adverse effects. |
| Phase I studies | A very small sample of human volunteers is used to test the drugs. Usually, the individuals are healthy subjects. |
| Phase II studies | Clinical investigators test the drug on clients with the disease that the drug has been developed to treat. Subjects are monitored very closely to evaluate the intended effects and for adverse reactions. |
| Phase III studies | This is a large-scale clinical trial. Prescribers assist in observing the client taking the drug. The goal of this phase is to gather data about the effectiveness and safety of the drug. |
| FDA approval | Once drugs make it through Phase III studies, they are evaluated by the FDA following the submission of a New Drug Application. If the FDA approves the drug, it can then be marketed. |
|
Phase IV studies
(post-marketing surveillance) |
Continual evaluations of drugs following approval by the FDA |
Some estimate the cost of developing just one new drug as ranging from less than $1 billion to over $2 billion (Wouters et al., 2020). The development of new drugs requires 10–15 years before the testing and drug studies are complete. Thousands of compounds are tested yearly, but only a few make it to clinical trials. Once a drug enters clinical trials, only a few are approved. CDER does not perform the clinical trials—this is up to the drug company; however, CDER reviews the pharmaceutical company’s data and the proposed drug labeling. At the heart of the approval process is establishing the health benefits of the drug and its safety profile. Does the benefit of the drug outweigh its risk?
There also are limitations to testing new drugs. Historically, there has been limited testing of drugs in the populations of females (particularly those of childbearing age), children (anyone under the age of 18 years old), and people of color (POC) . This has recently changed, but before 2000, minimal testing was performed on females. No clinical trials were allowed for females of childbearing age, even if they were not pregnant and were taking effective birth control. This greatly limited knowledge about how females would respond to many drugs. This also means that there is limited data about drug safety during pregnancy.
Children were also excluded from clinical trials in the past, though some exceptions have been made more recently. According to the FDA (2016), only about 20% of drugs are approved for pediatric use. For that reason, physicians have had to prescribe drugs “off-label” for children. The problem is that well-controlled clinical trials for pediatric dosing have not been established for many drugs, meaning there is no safety data for most drugs. The reasons for this lack of data are somewhat surprising. Some believe that pharmaceutical companies saw little profit in medications for children. Finding adequate numbers of children for robust testing could be more of a challenge than with adults, especially in trials where blood would need to be drawn.
There are also ethical reasons to exclude children from trials, especially because children are unable to give consent. According to the FDA (2016, para. 7), “Parents are involved in the decision to enroll children in a study, and children ages 7 or older can ‘assent’ or ‘dissent,’ meaning they can agree or disagree to participate in a study.” For more information about children’s assent and parental permission, review this article by the National Cancer Institute . More testing has been conducted in the last decade, and the information coming out of those studies has shown much more accurate methods for dosing and prescribing. The FDA web page on drug research and children has more information.
Link to Learning
In this video from the National Heart, Lung, and Blood Institute, pediatric researchers, doctors, and nurses explain why children should be included in clinical drug trials and answer common questions from parents and caregivers.
Even as late as 2019, only 15%–19% of participants in U.S. clinical drug trials were Black. When broken down further, only 3% of participants in clinical trials for cardiovascular disease were Black males (6% were Black females), and less than 5% of oncology trial participants were Black males (2% were Black females) (Whyte, 2022). When specific populations are under-represented in a clinical trial, the efficacy and safety are unknown for that particular subset. The efficacy and safety of some drugs may differ for various ethnicities and genetic backgrounds (Clark et al., 2019). Due to a variety of barriers, racial and ethnic minorities are often underrepresented in clinical trials (FDA, 2022a). When clinical trials test therapies only within a homogenous group, the findings are likely to be skewed. One of the problems with this is that minority groups may have less benefit from those therapies. Some of the barriers to increasing minority involvement in clinical trials include a lack of understanding of the value of the process; mistrust of research; lack of information, time, and resources; and a lack of knowledge about the existence of various trials (Clark et al., 2019). Many differences in health outcomes have been documented based on race and ethnicity, underscoring the importance of including a variety of racial and ethnic groups in clinical trials.
Trending Today
Off-label prescription drug use pertains to the utilization of a medication in ways that deviate from the specifications provided on the drug’s label or within its FDA-approved package insert. Such usage encompasses employing the medication for divergent medical conditions, adjusting dosages, targeting varying client groups, or employing alternative routes of administration, all of which may contrast with the medication’s initial FDA approval. For example, after the COVID-19 pandemic started, a research institute focused its efforts on repurposing existing drugs (see the Wyss Institute website and the video above).
The safety of off-label drug use varies based on factors such as the specific medication and condition, the health care provider’s judgment and knowledge, and the available evidence. Providers should weigh potential benefits against risks (as discussed in the Wall Street Journal video above) because off-label use may be valuable when approved alternatives are lacking. This article provides 2023 statistics on the drug classes in which off-label use is typical.
When administering off-label drugs, nurses should ensure informed consent from the client or their representative, discussing potential risks and benefits of the proposed medication. The nurse should also collaborate with the health care team for guidance and to determine the appropriateness of the drug. Other duties of the nurse include documenting the rationale for the use of the drug, educating the client on its proper use, monitoring the client closely for unexpected effects, and staying updated on the latest research. Above all, it is important for the nurse to uphold ethical principles and keep the client’s well-being at the forefront.
Canadian Drug Regulation
The United States’ and Canada’s drug laws have evolved in a similar manner. Any drug manufacturer must provide scientific evidence of the drug’s safety, efficacy, and quality to Health Canada before the sale of that product is authorized. The federal review process by Health Canada was empowered by the Food and Drugs Act of 1927 as well as additional regulations (in 1953, 1954, and 1979) intended to protect consumers from risks associated with the production and sale of drugs, cosmetics, food, and medical devices (Health Canada, n.d.). It regulates the manufacturing, packaging, labeling, storage, and sale of food and drugs in Canada. It also determines whether a drug is a prescription or nonprescription medication. Canada requires prescriptions for narcotics and strict guidelines for record-keeping for prescribing and dispensing those drugs. Drug use in Canada is regulated and enforced by many different agencies, including Health Canada, the Royal Canadian Mounted Police, and agencies within various provinces.
Although the schedule of drugs is different from that of the United States (see Drug Classifications and Prototypes for U.S. drug schedules), Canada has tried to align the schedules throughout each province so that the sale of medications is consistent throughout the country. The governing body for Canadian national drug schedules is the National Association of Pharmacy Regulatory Authorities (NAPRA) , though each province does regulate how drugs are sold or dispensed. In Canada, drugs are assigned to one of four categories (Drug & Alcohol Testing Association of Canada [DATAC], 2017; National Association of Pharmacy Regulatory Authorities, 2023):
- Schedule I: All prescription drugs. These drugs must be provided to the consumer by a pharmacist through a controlled, regulated environment. Drugs in this category include heroin, cocaine, morphine, and methadone.
- Schedule II: Restricted-access nonprescription drugs. These drugs may require professional intervention from a pharmacist when sold to the consumer. Must be stored with no public access or chance for the public to choose or select the drug (in other words, “behind the counter”). Schedule II drugs include insulin, pseudoephedrine, and sublingual nitroglycerin.
- Schedule III: Available without a prescription and sold on store shelves under the supervision of a pharmacist. Examples in this category are ibuprofen or naproxen.
- Unscheduled: Can be sold without supervision from any retail outlet. Emergency contraception, such as norgestrel, is considered an unscheduled drug.
Drug Counterfeiting
The United States has one of the world’s safest drug supplies, partly due to the FDA and USP-NF. Unfortunately, counterfeit drugs threaten that safety. Counterfeit drugs are products that are illegally manufactured or mislabeled with regard to their identity or source so that they appear to be a genuine product. They are fake drugs and may harm the recipient's health. Illegal online sales expose consumers to potential counterfeit drugs. Counterfeit drugs are not the same thing as generic medications. As mentioned earlier in this chapter, a generic drug is manufactured with the intent that it is identical in the active chemical ingredients, safety, strength, quality, dosage form, and intended use as its brand name counterpart (FDA, 2021a). These are drugs approved by the FDA. Counterfeit drugs are not.
Consumers can protect themselves by buying medications from state-licensed pharmacies in the United States. If using an online pharmacy, the consumer should check for the online pharmacy’s license through the state Board of Pharmacy. If it is not listed, that pharmacy should not be used. Reputable online pharmacies should have a seal of approval from the Verified Internet Pharmacy Practice Site (VIPPS) . An online pharmacy should always require a health care provider’s prescription and have a physical address and phone number in the United States. Another strategy for the consumer is to be alert to changes or variations in the packaging of medications—for example, the color or lettering might be different. Consumers should be alert to any unusual taste or side effects from the drug and report it immediately. Concerns about potential counterfeit drugs should be reported to the FDA through its MedWatch website or by calling 1-800-FDA-1088. The 2013 Drug Supply Chain Security Act was passed as an initiative to combat the production and use of counterfeit drugs. The FDA’s Office of Criminal Investigations investigates counterfeit drugs, their producers, and their sellers and attempts to bring them to justice.