PHARMACOTHERAPEUTICS: RATIONAL USE OF MEDICINES, EVIDENCE BASED MEDICINE, ESSENTIAL MEDICINES LIST, AND STANDARD TREATMENT GUIDELINES: A TEACHER’S COMPREHENSIVE GUIDE
Welcome, future pharmacists and healthcare professionals!
Rational use of medicines is one of the most critical concepts in healthcare delivery. It ensures that patients receive the right drug, in the right dose, for the right duration, at the right cost. As a pharmacy educator with years of experience teaching pharmacotherapeutics, I have observed that students often underestimate the importance of rational drug use and evidence-based practice. Let me tell you: Rational use of medicines is the cornerstone of quality healthcare.
In this comprehensive guide, I will walk you through the fundamentals of rational drug use—its definition, reasons for irrational use, types of irrational use, and WHO interventions. I will also explain Evidence-Based Medicine (EBM), the Essential Medicines List (EML), and Standard Treatment Guidelines (STGs). By the end of this article, you will have a thorough understanding of how to ensure rational and evidence-based prescribing. Let us begin our journey!
Dpharmguru’s exam insights:
Rational use of medicines is frequently tested in pharmacy exams. Remember: Rational use means the right drug, right dose, right duration, and right cost. Pay special attention to the reasons for irrational use and WHO interventions to promote rational use. The EBM triad (Clinical Expertise + Best External Evidence + Patient Values) is a classic exam question. Also, remember the five-step model of EBM—Ask, Acquire, Appraise, Apply, Assess!
1. RATIONAL USE OF MEDICINES
Rational use of medicines refers to prescribing the right drug, in adequate dose for sufficient duration, and suitable to the clinical requirements of the patient at minimum cost.
It includes:
- Patients receive medications as per their clinical needs
- In doses that meet individual requirements
- For an adequate time period
- At the lowest cost to them and their community
A. Reasons for Irrational Use of Drugs
- Lack of adequate information
- Defective, insufficient training and education of healthcare providers
- Under-privileged communication between healthcare providers and patient
- Not having diagnostic services
- Insistence from the patient
- Sub-standard drug supply system and bad drug regulation
- Promotional performance of pharmaceutical industries
B. Types of Irrational Use of Medicines
- Poly-pharmacy: Use of multiple medications unnecessarily
- Inappropriate use of antibiotics: Overuse or misuse of antibiotics leading to resistance
- Excessive use of injections: Overuse of injectable formulations when oral forms are adequate
- Non-accordance with clinical guidelines: Not following evidence-based protocols
- Self-medication: Use of medicines without professional supervision
Dpharmguru’s exam insights:
Types of irrational use are frequently tested. Remember the mnemonic “P-I-E-N-S”: Poly-pharmacy, Inappropriate antibiotics, Excessive injections, Non-accordance with guidelines, Self-medication. A common exam question is: “What is the most common type of irrational drug use?” (Answer: Poly-pharmacy or Inappropriate use of antibiotics).
C. WHO Interventions to Promote Rational Use of Medicines
- Establishing a multidisciplinary national body to coordinate policies on medicine use
- Using clinical guidelines
- Developing and using national essential medicines list
- Establishing drug and therapeutics committees in districts and hospitals
- Including problem-based pharmacotherapy training in undergraduate curricula
- Continuing in-service medical education as a licensure requirement
- Supervising, auditing, and feedback
- Using independent information on medicines
- Providing public education about medicines
- Avoiding perverse financial incentives
- Using appropriate and enforced regulation
- Providing government expenditure to ensure availability of medicines and staff
2. EVIDENCE-BASED MEDICINE (EBM)
Evidence-Based Medicine (EBM) refers to an interdisciplinary approach that uses science, engineering, biostatistics, and epidemiology techniques—like meta-analysis, decision analysis, risk-benefit analysis, and randomised controlled trials—to deliver the right care at the right time to the right patient.
EBM is a systematic approach to clinical problem-solving through which the best available research evidence can be integrated with clinical expertise and patient values.
The EBM Triad
- Individual Clinical Expertise – The clinician’s experience and skills
- Best External Evidence – The best available research evidence
- Patient Values and Expectations – The patient’s preferences and circumstances
Dpharmguru’s exam insights:
The EBM Triad is a classic exam question. Remember: Evidence-Based Medicine is the integration of clinical expertise, best external evidence, and patient values. A common exam question is: “What are the three components of the EBM Triad?” (Answer: Clinical Expertise, Best External Evidence, Patient Values).
A. Five-Step Model of EBM
STEP 1: ASK a Clinical Question to Identify a Key Problem
The need for information (about prevention, diagnosis, prognosis, therapy, causation, etc.) is converted into an answerable question. A detailed and clearly worded question is formulated because these words will be used to perform a literature research.
STEP 2: ACQUIRE the Best Evidence
The best evidence with which to answer that question is tracked down. The EBM pyramid was designed to let clinicians compare the quality between various sources of evidence. Each rising level denotes a different study design and corresponds to increasing rigor, quality, and reliability.
STEP 3: APPRAISE the Evidence
The evidence should be critically appraised for its validity (closeness to the truth), impact (effect size), and applicability (usefulness in clinical practice). Clinicians should determine whether or not the evidence is valid and applicable for the situation or population considered.
STEP 4: APPLY the Evidence to Daily Clinical Practice
The critical appraisal is integrated with clinical expertise and with the patient’s biology, values, and circumstances. Evidence-based medicine does not replace a clinician’s expertise or judgment; but enhances their ability to make better care decisions based on the patient’s needs and preferences.
STEP 5: ASSESS the Performance
The effectiveness and efficiency in executing the above four steps is evaluated and ways to improve them are sorted by establishing a baseline and measuring the improvements. It is an ongoing process that reassesses and re-measures any gains or losses as part of an ongoing cycle to ensure the best outcomes.
B. Importance of EBM
- Helps clinicians to remain updated on standardised, evidence-based protocols
- Improved access to data and knowledge through Electronic Medical Records (EMRs), decision support systems, and data warehouses
- Improves transparency, accountability, and value
- Improves quality of care
- The most important reason—EBM works—better outcomes in every way
3. ESSENTIAL MEDICINES LIST
According to WHO, essential drugs (medicines) are “drugs that satisfy the priority healthcare needs of the population”.
Essential drugs are selected on the basis of:
- Relevance to the health of the community
- Confirmation of efficacy and safety
- Relative cost-effectiveness
Essential drugs must be available at all times, in suitable quantities, in correct dosage forms, with guaranteed quality, at an economical rate affordable by individuals and the community.
The WHO brought out its first Model List of Essential Drugs in 1977, for the guidance of member countries. The WHO Model Lists of Essential Medicines are updated every two years by the Expert Committee on Selection and Use of Essential Medicines.
Selection of Essential Medicine List
- Availability of satisfactory information about effectiveness and safety
- Availability of the drug with assured quality and stability
- Influence of disease prevalence, facilities, skilled personnel, financial resources, genetic, demographic, and environmental factors
- Comparative efficiency, safety, quality, cost, and availability—cost-benefit ratio is the chief concern
- Essential drugs should generally be single compounds. Fixed ratio combination products should be used only when proven advantages exist.
Current Essential Medicine List (India)
The Indian Government released the National List of Essential Medicines (NLEM) 2021, in which 39 drugs, including anti-cancer, anti-diabetes, and antiretrovirals, have been added. 16 drugs from NLEM 2015 have been removed.
Newly Added Drugs
- Amikacin (antibiotic)
- Azacitidine (anti-cancer)
- Bedaquiline (anti-TB)
- Bendamustine Hydrochloride (anti-cancer)
- Buprenorphine (opioid to treat opioid use disorder)
- Buprenorphine + Naloxone (to treat opioid addiction)
- Cefuroxime (antibiotic)
- Dabigatran (anticoagulant)
- Daclatasvir (hepatitis C drug)
- Darunavir + Ritonavir (antiretroviral)
- Delamanid (anti-TB)
- Dolutegravir (antiretroviral)
- Fludarabine (anti-cancer)
- Fludrocortisone (corticosteroid)
- Fomepizole (antidote to certain poisoning)
- Fulvestrant (anti-cancer)
- Insulin Glargine (long-acting insulin)
- Irinotecan HCl Trihydrate (anti-cancer)
- Itraconazole (antifungal)
- Ivermectin (anti-parasitic)
- Lamivudine (antiretroviral)
- Latanoprost (ophthalmic)
- Lenalidomide (anti-cancer)
- Leuprolide acetate (anti-cancer)
- Montelukast (anti-asthmatic)
- Mupirocin (antibiotic)
- Nicotine replacement therapy (to treat tobacco use disorder)
- Nitazoxanide (anti-parasitic and antiviral)
- Ormeloxifene (Centchroman) (non-steroidal oral contraceptive)
- Phenoxymethyl Penicillin (antibiotic)
- Procaine Benzyl Penicillin (antibiotic)
- Rotavirus vaccine
- Secnidazole (anti-infective)
- Tenecteplase (thrombolytic drug)
- Teneligliptin (antidiabetic)
- Tenofovir + Lamivudine + Dolutegravir (antiretroviral)
- Tenofovir Alafenamide Fumarate (TAF) (Hepatitis B drug)
- Terbinafine (antifungal)
- Valganciclovir (antiviral)
Drugs Removed from List
- Alteplase
- Atenolol
- Bleaching powder
- Cetrimide
- Erythromycin
- Ethinylestradiol + Norethisterone
- Ganciclovir
- Lamivudine + Nevirapine + Stavudine
- Leflunomide
- Nicotinamide
- Pegylated interferon Alfa 2a
- Pegylated interferon Alfa 2b
- Pentamidine
- Prilocaine + Lignocaine
- Rifabutin
- Stavudine + Lamivudine
- Sucralfate
Advantages of Essential Medicines List
- Better management of medicines: Easier procurement, storage, distribution, manageable stock, better quality assurance, easier dispensing, less fragmentation of budget, and enhanced drug availability
- Improved prescribing: Focused training and drug information, better recognition of adverse drug reactions and drug interactions
- Relates to treatment guidelines for clinical diseases
- Flexible and adaptable to different situations
- Ensures better availability, more economical and balanced use of drugs
4. STANDARD TREATMENT GUIDELINES (STGs)
Standard Treatment Guidelines (STGs) (or treatment protocols or prescribing policies) are systematically developed statements designed to assist prescribers in making decisions about appropriate treatment and healthcare for specific clinical problems.
Key Features of a Successful STGs Manual
- Simplicity: Limited to commonly observed conditions; clear and precise information
- Credibility: Developed by esteemed and experienced clinicians
- Treatment Standards for all Levels: Same standard treatment; referral criteria differ
- Medicine Supply Based on Standards: Medicine supply must match STGs and essential medicines list
- Regular Updating: Revised versions reflect current recommendations
- User-friendly: Small, durable pocket manual, easy to carry and use
STGs Development Process
1) STGs Writing Group
- Total 10 clinical specialty sub-groups were formed
- Groups recruited members from diverse backgrounds
- Senior most expert designated as facilitator
- Members declared conflicts of interest
- Writing group members were trained for guideline development methodology
2) Topic Selection Process
- Diseases prioritised based on burden of disease, non-availability of Indian guidelines, and policy importance
- Diseases with existing MoHFW guidelines were excluded
- Total 14 diseases selected for the first phase
3) Guideline Development
- Guidelines developed using agreed “Hybrid” methodology
- Scope of guidelines defined before searching for guidelines
- Reputed repositories searched for existing guidelines
- Guidelines screened against AGREE II tool before adaptation
4) Documentation
- Full Background Document: Detailed recommendations, source guidelines, development process
- Quick Reference Guide: Brief version with key recommendations and clinical pathways
- Implementation tools: Quality Standards, Patient Information Sheet, Formulary
5) Review Process
- Three-tier review process
- Internal review by designated harmonisation group
- External review by DGHS-appointed experts
- Open consultation and comments from public
- Final approval by MoHFW
Advantages of STGs
- For Patients: Cost-effective therapy, better compliance, improved availability of drugs, better quality of care
- For Healthcare Providers: Standardized guidance, standard quality of care, basis for monitoring
- For Supply Managers: Identifies medicines to be available, facilitates pre-packaging
- For Health Care Policy Makers: Focused therapeutic integration, cost control
Disadvantages of STGs
- Establishment, development, and implementation are difficult, lengthy, and time-taking
- Needs regular updation to reflect changes in treatment strategies
- If not updated regularly, loses reliability and becomes outdated
- Extent of information to be provided is a difficult decision
Dpharmguru’s exam insights:
Standard Treatment Guidelines are frequently tested. Remember the key features: Simplicity, Credibility, Treatment Standards for all levels, Medicine Supply Based on Standards, Regular Updating, and User-friendly. A common exam question is: “What are the advantages of STGs for patients?” (Answer: Cost-effective therapy, better compliance, improved drug availability, better quality of care).
COMPARISON: EBM, EML, AND STGs
| Concept | Purpose | Key Feature |
|---|---|---|
| Evidence-Based Medicine (EBM) | Integrate best evidence with clinical expertise and patient values | Five-step model: Ask, Acquire, Appraise, Apply, Assess |
| Essential Medicines List (EML) | Identify drugs that satisfy priority healthcare needs | Updated every 2 years by WHO |
| Standard Treatment Guidelines (STGs) | Assist prescribers in making treatment decisions | Systematically developed, evidence-based statements |
FREQUENTLY ASKED QUESTIONS (FAQs)
1. What is rational use of medicines?
Rational use of medicines means prescribing the right drug, in adequate dose, for sufficient duration, at minimum cost, suitable to the clinical requirements of the patient.
2. What are the five steps of Evidence-Based Medicine?
The five steps are: ASK a clinical question, ACQUIRE the best evidence, APPRAISE the evidence, APPLY the evidence to clinical practice, and ASSESS the performance.
3. What is the EBM Triad?
The EBM Triad consists of Individual Clinical Expertise, Best External Evidence, and Patient Values and Expectations.
4. What is the Essential Medicines List?
The Essential Medicines List is a list of drugs that satisfy the priority healthcare needs of the population. It is updated every two years by WHO. The Indian NLEM 2021 is the current list.
5. What are Standard Treatment Guidelines?
Standard Treatment Guidelines (STGs) are systematically developed statements designed to assist prescribers in making decisions about appropriate treatment for specific clinical problems.
6. What are the key features of successful STGs?
The key features are: Simplicity, Credibility, Treatment Standards for all levels, Medicine Supply Based on Standards, Regular Updating, and User-friendly.
SUMMARY
Rational use of medicines is the cornerstone of quality healthcare. This guide covered:
- Rational Use of Medicines: Definition, reasons for irrational use, types of irrational use, and WHO interventions
- Evidence-Based Medicine (EBM): Definition, EBM Triad, Five-Step Model, and Importance
- Essential Medicines List (EML): Definition, selection criteria, NLEM 2021 updates, and advantages
- Standard Treatment Guidelines (STGs): Definition, key features, development process, advantages, and disadvantages
As I always tell my students: “Rational use of medicines is not just about prescribing—it is about ensuring patient safety, quality care, and optimal use of healthcare resources.”
REFERENCES AND FURTHER READING
- Pharmacy Council of India (PCI). (2022). Pharmacotherapeutics Syllabus. New Delhi: PCI.
- World Health Organization (WHO). (2022). Rational Use of Medicines. Retrieved from https://www.who.int.
- World Health Organization (WHO). (2021). WHO Model List of Essential Medicines (22nd ed.). Geneva: WHO.
- Ministry of Health and Family Welfare. (2021). National List of Essential Medicines (NLEM). Government of India.
- Sackett, D. L., et al. (2000). Evidence-Based Medicine: How to Practice and Teach EBM (2nd ed.). Churchill Livingstone.
- National Health Systems Resource Centre (NHSRC). (2022). Standard Treatment Guidelines. Government of India.
Disclaimer: This article is for educational purposes only and does not constitute medical advice. Always consult qualified healthcare professionals for medical concerns. Pharmaceutical regulations and guidelines may vary by region—always refer to your local regulatory authorities for specific requirements.
written by:
Dr. N. Sujith Kumar
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