Optimising Drug Use: Essential Medicines, Guidelines & Formulary Management

Essential Medicines

Definition of Essential Medicines

• WHO Definition: Medicines that satisfy the priority healthcare needs of a population.

Selection Criteria

• Selected based on:
  • Public health relevance: Disease prevalence in the region/country.
  • Scientific evidence: Efficacy, safety, and cost-effectiveness.
  • Healthcare system factors: Treatment facilities available, knowledge/skills/training of healthcare personnel, ease of administration, storage facilities, and patient acceptability.
• A mixture of evidence-based criteria and healthcare system-related factors.

Importance and Concept of Essential Medicines

• Availability: Should be available at all times, in adequate quantities, appropriate dosage forms, with assured quality, appropriate information, and at an affordable price.
• Coverage: Expected to cover 80-90% of a population’s healthcare needs.
• EML Concept: A limited range of carefully selected core essential medicines leads to better healthcare, improved drug management, and lower costs.
• Dynamic Nature: Regularly updated based on current evidence, changes in resistance patterns, emerging diseases, newer molecules, and improved formulations.

Essential Medicines List (EML) vs. Essential Drugs List (EDL)

• No Difference: Both terms refer to the same concept.
• Nomenclature Change: ‘Drug’ was replaced by ‘medicine’ after 2000 in many documents to avoid association with narcotics and substances of abuse.

Origins and Evolution of the WHO Model EML

• Initiation: Started in response to requests from developing countries to the WHO in 1975.
• First Publication: The first list of essential medicines was published in 1977.
• Alma Ata Declaration (1978): Essential medicines were outlined as one of the eight components of primary healthcare.
• Regular Updates: WHO regularly updates the list every two years (17th list published in 2011).
• EML for Children (EMLc): A model list for children was first published in 2007 and updated in 2009 and 2011.
• Guide, Not Global List: The WHO model list serves as a guide for national and institutional lists; its selection method can be applied by all countries.
• Evidence-Based Approach: Shifted from expert experience to an evidence-based approach in 2003.
• Transparent Process: To add or delete a drug, an application form with 15 questions is submitted and made available for public comment on the WHO website. An expert international committee evaluates the evidence and declares conflicts of interest.

Advantages of Having an EML

• Preventing Deaths: Availability of essential medicines can prevent a large proportion of deaths from treatable causes (e.g., diarrhoea, pneumonia, neonatal sepsis) in developing countries.
• Easier Procurement: A limited list simplifies procurement of correct formulations in appropriate quantities.
• Manageable Storage: Easier to track medicines and prevent stock-outs or pile-ups leading to expiry.
• Cost-Effective Quality Assurance: Testing quality is cheaper with a limited list.
• In-depth Knowledge: Doctors and pharmacists can acquire in-depth knowledge of clinical pharmacology, leading to appropriate use and better patient counselling.
• Increased Efficiency: Prescribers often use a maximum of about 50 drugs; if these are from the EML, it increases healthcare system efficiency.
• Adherence to Guidelines: Ensures patients are treated according to accepted Standard Treatment Guidelines (STGs) and national health programmes.

Disadvantages and Controversies of Having an EML

• Perception of being for the poor: The EML is often perceived as a list for the poor, despite its universal applicability.
• Prescriber Resentment: Doctors may resent restrictions on prescribing, viewing it as a loss of autonomy. However, doctors in high-income countries often already prescribe from approved lists.
• Pharmaceutical Industry Opposition: The industry has a strong commercial interest in many medicines being on the market and often opposes limited lists, highlighting the conflict between health and commerce.

Number of EMLs

• Typically one national list per country, reflecting population healthcare needs.
• In large, diverse countries like India, there may be State Lists, which specify which medicines are available at primary, secondary, and tertiary levels of care.

Rational Drug Use (RDU)

Definition of Rational Use of Drugs

• WHO (1985): Patients receive medications appropriate to their clinical needs, in doses that meet their individual requirements for an adequate period, and at the lowest cost to them and their community.
• Involves choosing the correct drug based on efficacy, safety, suitability, and cost for the appropriate indication, correctly dispensing with understandable information, and patient adherence.
• Irrational use occurs when one or more of these conditions are not met.

Common Types of Irrational Use

• Polypharmacy: Use of too many medicines in a single patient.
• Inappropriate Antibiotic Use: Inadequate dosage (strength or duration), or use for non-bacterial (e.g., viral) infections.
• Over-use of Injections: When oral route would be more appropriate.
• Inappropriate Self-medication: Mostly with prescription-only drugs.
• Failure to Prescribe in Accordance with STGs.

Why is Irrational Use a Problem?

• Can lead to increased costs of medical care, antimicrobial resistance, adverse effects, and patient mortality.
• In the private sector in India, irrational use can lead to a conflict of interest, as doctors and pharmacists may gain financially from prescribing expensive, unnecessary, branded drugs.

Monitoring Rational Drug Use

• Aggregate Medicine Consumption Data: Compares actual vs. expected consumption (based on morbidity), identifies use of costlier/less effective medicines. Publicising data can reduce consumption.
• WHO Drug Use Indicators: Used to describe prescribing patterns and quality of care in primary healthcare facilities.
  • Prescribing Indicators: Average number of medicines per patient, % prescribed by generic name, % with an antibiotic/injection, % from EML/formulary.
  • Patient Care Indicators: Average consultation/dispensing time, % medicines dispensed/labelled adequately, % patients with knowledge of correct doses.
  • Facility Indicators: Availability of EML/formulary, clinical guidelines, % key medicines available.
  • Complementary Indicators: Average medicine cost per encounter, % prescriptions in accordance with clinical guidelines.
• Anatomical Therapeutic Chemical (ATC) Classification and Defined Daily Dose (DDD) Methodology: Compares drug consumption patterns across institutions, regions, and countries.
  • ATC System: Classifies drugs by Anatomic system, Therapeutic use, and Chemical class. Each drug has a unique identifying number (e.g., omeprazole A02BC01).
  • DDD: A uniform concept, unit of measurement for comparing utilisation. Disadvantages: not for all drugs/paediatric use, multiple indications for a drug.
• Drug Utilisation Review (DUR) or Focused Drug Use Evaluation (DUE): Useful in hospitals for specific medicines/diseases to identify problems.
• Qualitative Methods: In-depth interviews, structured questionnaires, or focus group discussions to identify causes of irrational use.

Strategies to Promote Rational Drug Use

• Managerial: Supervising and guiding clinical practice, persuading doctors to use STGs and EML, prescription audits for feedback.
• Economic: Incentives for institutions, patients, and providers. Insurance agencies may only reimburse for medicines from restricted lists. Pharmaceutical companies compete on price for formulary inclusion.
• Regulatory: Restricting prescribing choices. Laws like allopathic doctors not prescribing other systems’ medicines. Licensing, drug scheduling, banning unsafe medicines, and regulating pharmaceutical promotion (a serious lacuna in India).
• Educational: Regular education/training for health providers, use of formularies, clinical supervision. Patient counselling by pharmacists. Education alone has limited effect without monitoring and feedback.
• Funding: A small percentage of the medicines budget can be allocated to these measures, potentially leading to significant savings.

National Strategies to Promote Rational Drug Use (WHO Recommendations)

• Establish a mandated national body to coordinate medicine policies and monitor them.
• Prepare and use evidence-based clinical guidelines or STGs for training and decision-making.
• Prepare an EML based on treatment of choice, for procurement and insurance reimbursement.
• Set up Drug and Therapeutics Committees (DTCs) at institutional and district levels.
• Introduce problem-based pharmacotherapy training in undergraduate medical courses.
• Make continuing medical education a mandatory requirement for licensing.
• Provide independent, unbiased drug information (formularies, bulletins).
• Encourage supervision of prescribing, drug audit, and feedback.
• Educate the public about medicines.
• Eliminate perverse financial incentives that lead to irrational prescribing.
• Enforce appropriate drug regulations and WHO ethical criteria for promotion.
• Ensure adequate government spending for equitable availability of medicines and health providers.

Role of the Pharmacist in Promoting RDU

• Pharmacists play a vital role in RDU promotion, from pharmaceutical care and unbiased drug information to selection, procurement, and dispensing of essential medicines.
• DTC Member: Provides vital input in selection of essential medicines, EML preparation/implementation, and other activities.
• Drug Procurement: Ensures procurement of good-quality, reasonably priced medicines. Well-versed in inventory control and forecasting. Procures EML medicines from reliable suppliers.
• Drug Storage: Responsible for indenting, receiving, and storing medicines, following good storage practices, and preventing stock-outs or pile-ups leading to expiry. Deals with expired, narcotic, and costly medicines using SOPs.
• Dispensing: Ensures accurate dispensing for inpatients and outpatients. Restricted lists limit dispensing errors. Vigilance against pilferage.
• Drug Information Service: Provides unbiased, up-to-date information to prescribers, patients, public, and other healthcare workers.
• Pharmaceutical Care: A patient-centred practice where the pharmacist assumes responsibility for a patient’s drug-related needs and collaborates with other healthcare providers to achieve health goals. Improves rational use.

Development of Therapeutic Guidelines (TGs)

Meaning of Therapeutic Guidelines

• Definition: Systematically developed statements designed to assist practitioners and patients in making decisions about appropriate healthcare for specific clinical circumstances.
• Clinical treatment protocols prepared by experts using scientific evidence.
• List preferred drug and non-drug treatments for common health problems, considering demographic, epidemiological, cultural, socioeconomic factors, and resource availability.
• Can be prepared for different levels of healthcare providers.

Need for Therapeutic Guidelines

• Quality of Care: Provides consistent and high-quality care.
• Cost Containment: Reduces therapy costs by using substantiated means. Important in countries with limited resources and conflicts of interest.
• Adverse Events: Limits the number of adverse events and therapeutic misadventures.
• International Research: Allows practitioners to incorporate international research data into general practice.
• Standard of Care: Ensures a consistent standard of care without the costs of individual investigation, clinical trials, bias, and error.

Locating Published Therapeutic Guidelines

• Many guidelines are available in medical literature (e.g., NCEP for hypercholesterolaemia, available on NIH website).
• Online resources like the US Agency for Healthcare Research and Quality (www.guideline.gov/).

Steps in the Development, Review, and Implementation of TGs

1. Identify Group Members for Guideline Development: Multidisciplinary group (physicians, nurses, pharmacists, epidemiologists, health economists, guideline methodology specialists). Ideal size: 6-15 members. Pharmacists should play an integral role.
2. Identify and Refine the Problem: Prioritise subjects/topics based on morbidity/mortality, uncertainty in therapy, or resource conservation. Analyse drug utilisation trends.
3. Literature Review: Exhaustive search using multiple mechanisms (Medline, Micromedex, Cochrane, Embase). Critically evaluate relevant original papers, summarising key findings and derivable drug use criteria. Criteria must be scientifically based, valid, unambiguous, realistic, easily measured, and outcome-oriented, developed with specialist input.
4. Consensus and Recommendations: (Implicit in the process of expert group review)
5. Dissemination: Guidelines must be officially introduced with proper training and wide dissemination. Sufficient copies printed and distributed.
6. Implementation: Factors affecting implementation include: participatory approach in formulation, ease of use, training, dissemination, and integration with audit and feedback.
7. Audit and Feedback: Essential part of STG use.
8. Regular Updates: Must be regularly updated to maintain relevance and favourability among prescribers.
9. Funding: Adequate funds needed for formulation, printing, training, auditing, and disseminating feedback.

Potential Limitations of Therapeutic Guidelines

• Outdated Information: Unless regularly updated, they may not be viewed favourably or used by prescribers.
• Lack of Specificity: May not apply to all individual patient circumstances due to unique factors.

Pharmacy and Therapeutics (P&T) Committee

Organisation

• Also known as Medicine and Therapeutics Committees or Drug and Therapeutics Committees (DTCs).
• Formed by an institution or hospital.
• Comprises clinicians from different specialties, pharmacists, and a representative of hospital administration.

Functions

• Facilitates the rational use of medicines by supporting healthcare providers and hospital administrators.
• Developing, adapting, or adopting clinical guidelines for the health institution or district.
• Selecting cost-effective and safe medicines (for the hospital/district drug formulary).
• Implementing and evaluating strategies to improve medicine use (including drug use evaluation, liaison with antibiotic and infection control committees).
• Providing on-going staff education (training and printed materials).
• Controlling access to staff by the pharmaceutical industry with its promotional activities.
• Monitoring and taking action to prevent adverse drug reactions and medication errors.
• Providing advice about other drug management issues such as quality and expenditure.
• Pharmacist input is vital in selection of essential medicines, and preparation and implementation of EML.

Policies of the P&T Committee

• Including Drugs into Formulary:
  • Criteria for inclusion should consider efficacy, safety, and cost-effectiveness (as seen in EML selection).
  • New drugs must pass rigorous screening to be listed (e.g., PBS in Australia).
• Inpatient and Outpatient Prescription: Policies govern what can be prescribed and for whom.
• Automatic Stop Order: For certain medications, an automatic stop date may be set to prevent prolonged, unnecessary use.
• Emergency Drug List Preparation: Ensures critical medications are readily available for emergencies.

Hospital Formulary

Definition

• A listing of drugs that a physician may prescribe within a hospital.
• Physicians are requested or required to prescribe only formulary drugs unless there is a valid medical reason for non-formulary drugs.

Contents of Hospital Formulary

• List of accepted medicines. These are typically the cost-effective and safe medicines selected by the P&T Committee.
• Includes essential medicines relevant to the institution.

Differentiation of Hospital Formulary and Drug List

• The syllabus states this as a point of differentiation. The provided text does not explicitly define a “Drug List” in contrast to a “Formulary” but implies a formulary is a specific, approved list for a facility. The PBS in Australia acts as a de facto national formulary, listing subsidised essential medicines.

Preparation and Revision

• Preparation: The P&T Committee is responsible for selecting cost-effective and safe medicines for the hospital formulary.
• Revision: The concept of the EML (which heavily influences formularies) is dynamic and requires regular updating based on current evidence, resistance patterns, new molecules, and improved formulations.

Addition and Deletion of Drugs from Hospital Formulary

• Based on rigorous screening for cost-effectiveness relative to other listed therapies for the same condition.
• The P&T Committee establishes policies for this.

Investigational Use of Drugs

• Description: This involves using drugs that are still under investigation or not yet fully approved for general marketing or specific indications.
• Principles Involved: Principles of clinical research apply, including ethical considerations and rigorous study design to determine efficacy, safety, interactions, and cost-effectiveness.
• Classification: Can involve different phases of clinical trials (Phase I-III for pre-marketing development).
• Control: Strict control is necessary, typically through protocols and regulatory oversight (e.g., Ethics Committees).
• Identification: Identifying such drugs and their use is crucial for monitoring.
• Role of Hospital Pharmacist: Hospital pharmacists are involved in the control of investigational drugs. They may also support clinical research activities.
• Advisory Committee: An advisory committee, likely the P&T Committee or an Ethics Committee, would be involved in overseeing the investigational use of drugs. (The source does not explicitly detail an “advisory committee” for investigational drugs but the functions of the EC and P&T Committee would cover this oversight).