Pharmaceutical Classification Systems PDF

Classification systems for pharmaceuticals Dietary supplements Foods for special groups Orphan drugs Medical devices PharmDr. Tereza Toušková, PhD. Department of Social and Clinical Pharmacy Social Pharmacy 2024/2025 Lecture outline Names of medicines and their classification...

Classification systems for pharmaceuticals Dietary supplements Foods for special groups Orphan drugs Medical devices PharmDr. Tereza Toušková, PhD. Department of Social and Clinical Pharmacy Social Pharmacy 2024/2025 Lecture outline Names of medicines and their classification systems Food supplements Foods for special groups Orphan drugs Medical devices Goals of the lecture Know the different names of medicines - when and where to use which ones and look for them Look in detail at classification systems and get a practical understanding of how to use them Learn what a defined daily dose is and how it is used Understand the issues and legal aspects surrounding dietary supplements and health claims Define and classify foods for special purposes Map the issue of orphan drugs and medical devices Classification systems for pharmaceuticals  Classification of pharmaceuticals According to the chemical structure: Chemical drugs Biological drugs By chemical structure: Benzodiazepines, sulfonamides, phenanthrenes... By origin: Drugs of natural origin (digoxin, cocaine, colchicine...) Drugs of polysynthetic origin (oxycodone, hydromorphone, irinotecan...) Synthetic drugs (enalapril, acebutolol, cisplatin...)  Classification of pharmaceuticals According to the registration statute: Registered Unregistered By method of preparation: mass-produced medicinal product individually prepared medicinal product By pharmacopoeial status: Official, pharmacopoeial (medicines listed in the current Pharmacopoeia) Unofficial, non-pharmacopoeial (medicines not included in the current Pharmacopoeia)  Classification of pharmaceuticals According to the method of reimbursement from public health insurance: Fully covered Partially covered Not covered Based on market availability: Marketed, available (products with active presence on the Czech or other national market according to regular supply reporting by distributors) Not traded, unavailable By method of dispensing the medicinal product (MP): Medicinal products dispensed by prescription only (prescription, request form) MP dispensed by prescription only with blue stripe (prescription for addictive substances) MP dispensed by prescription only with restriction MP dispensed without prescription with restriction MP dispensed without prescription (over-the-counter products) Restricted MP (OTC out of pharmacy)  Classification of pharmaceuticals By origin: Original (innovative) drugs (Lead compound and Me-too drugs) Generic drugs and biosimilars By Expiry: Non-expired (until expiry date) Expired (after expiry date) By shelf life: Usable (original packaging, not expired...) Unusable (deteriorated, damaged, not used, expired...)  Classification of pharmaceuticals According to their necessity: Essential medicines (essential for the general population in developed countries) Orphan drugs (= orphan drugs) Obsolent drugs ( no longer used in therapy in developed countries) Further classification: According to similar groups in chemical structure (enzymes, hormones, salicylates...) According to their action Inducing (hypnotics, diuretics, nootropics, obstipants...) Avoiding (antiarrhythmics, antibiotics, antidiabetics, contraceptives, antidiarhotics) Combined designation (beta-blockers, hormonal contraceptives)  Drug name Unambiguous Unique Simple  Trivial names Code names, code names Chemical names Name of Generic names drugs INN names Brand names Pharmacopoeial names Trivial name It is simple, generally accepted, specific to a particular chemical compound. It relates to the discovery of a drug, its preparation, its origin, or the characteristic properties of the drug. Trivial names - no part has a systematic meaning Semitrivial names - from a part formed systematically and from a part not formed systematically.  Code name This is the first working designation for a drug granted during its research. Usually, the code name is used until the substance is given an INN name. Medicinal products may also have two designations if several entities are jointly involved in its research and development There are more than one way to use it: Alphanumeric combination: The alphabetic part refers to the company, the laboratory, or it is the manufacturer's own code. The numerical component may be determined arbitrarily. Alphabetic combination.  Chemical name = systematic name It is a systematic name according to the current IUPAC rules Can be defined by unique international codes It is unique and unambiguous, but usually complicated and therefore often unusable in routine pharmacy practice. Applicable (Aluminii hydroxidum, Kalii chloridum, Magnesii oxidum) Unusable (2RS)-2-[bis(2-chloroethyl)amino]tetrahydro-2H-1,3,2- oxazaphosphin-2-oxide)  Generic name Simply put - INN adapted to linguistic conventions, or the older equivalent of the INN name Generic names were created by research teams without fixed rules, which brought the risk of duplication or different names for the same substance International Nonproprietary Names (INN) INN (International Nonproprietary Name) Used in the scientific literature and on the packaging of medicines. The internationally used designation of a substance. To avoid duplication, the new name is subject to a comment procedure. Name assigned by WHO, not part of patent protection, i.e. not commercially protected. It is in the public domain. The main INN name is given in Latin, but also in English, French, Spanish, Russian, German Arabic and Chinese.  International Nonproprietary Names (INN) The cumulative list of INN now stands at some 7000 names designated since that time, and this number is growing every year by some 120-150 new INN. The aim: to provide health professionals with a unique and universally available designated name to identify each pharmaceutical substance It is important for the clear identification, safe prescription and dispensing of medicines to patients, and for communication and exchange of information among health professionals and scientists worldwide  INN Cumulative list All names selected as proposed and recommended INNs are published in a Cumulative list, which is updated periodically. Each entry includes: equivalent nonproprietary names: : in Latin, English, French, Spanish, Arabic, Chinese and Russian, as well as reference to other common names; a reference to the INN list in which the name was originally proposed or recommended, or last amended; reference to names of substances that have been abandoned or never been marketed; reference to national nonproprietary names; reference to pharmacopeial monographs or similar official references; reference to names issued by the International Organization for Standardization (ISO); reference to the Convention of Psychotropic Substances, if applicable; reference to the List of Narcotic Drugs under International Control, if applicable; the molecular formula; its Chemical Abstracts Service (CAS) number.  INN Cumulative list INN name formation - Stems There are relatively simple, but non-binding, rules for creating a name: the chemical structure, the intended use or the natural origin, one-word names are preferred, morphemes are recommended for some indication groups. Common Stems for International Nonproprietary Names (INNs)  Modified International Nonproprietary Names (INNM) INN are selected only for the active part of the molecule which is usually the base, acid or alcohol. In some cases, however, the active molecules need to be expanded for various reasons, such as formulation purposes, bioavailability or absorption rate. In 1975 the experts designated for the selection of INN decided to adopt a new policy for naming such molecules. In future, names for different salts or esters of the same active substance should differ only with regard to the inactive moiety of the molecule. For example, oxacillin and ibufenac are INN and their salts are named oxacillin sodium and ibufenac sodium. The latter are called modified INN (INNM).  Brand name = protected, corporate, commercial, original, manufacturing A commercially protected name designated by a manufacturing pharmaceutical company. Often a trade name is created from a generic or INN name associated with the manufacturer's name. INN Ibuprofen can be found, for example, under the following trade names: Brufen®, Ibalgin®, Nurofen®, Ibumax®, or combinations: Apo- Ibuprofen®...  Pharmacopoeial name Pharmacopoeial (official = listed in the pharmacopoeia, from Latin officina - workshop, pharmacy) names of drugs are the names under which individual drugs are listed in the pharmacopoeia. They always begin with a capital letter. Pharmacopoeial names are not a special kind of name - the title of a pharmacopoeial article (called a monograph) is an application of, for example, a chemical, INN...  Classification Systems of Medicines Ordering of terms (drugs, chemicals,...) into classes -> unambiguous identification of classified entities and their properties Classification is typically handled by national or international health organisations These classification systems can also be used as selection languages in the environment of some professional electronic information resources Classification systems ATC classification NDC code DIN code  Anatomical Therapeutic Chemical (ATC) Classification System ATC: anatomical-therapeutic-chemical ("Anatomical therapeutic classification"). It represents the most widely used classification system for drugs. Used on the basis of recommendations from the WHO, which is also the body that defines the conditions for registration of drugs in the system. Classifies drugs into groups according to their action on a given organ or system within the body, and also according to their chemical, pharmacological or therapeutic properties. In the ATC classification system, active substances are classified in a hierarchy with five different levels.  ATC classification of medicines - structure The active substances are sequentially classified according to the organ system they affect, their clinical effect on the organism and their chemical structure. 1. the most general division is into 14 classes - each class is assigned one letter  ATC classification of medicines - code format It typically consists of 7 alphanumeric characters that are assigned based on group membership within the hierarchical levels described  1 letter anatomical group - e.g. C Cardiovascular system, N Nervous system ATC 2 digits main therapeutic groupe - e.g. C03 Diuretics, N01 Anaesthetics, N02 Analgesics classification 1 letter indication group - e.g. N02A Opioid analgesics, N02C Antimigraines of medicines 1 letter chemotherapeutic group - e.g. N02AA Natural opioid alkaloids, N02CA Narcotic alkaloids 2 digits active substance - e.g. N02AA01 Morphine, N02AA59 Codeine Veterinary ATC = ATC vet Concerns medical products subject to ATC classification in the veterinary sector The ATC vet code comprises a standard string of 8 characters, always beginning with the letter Q  Herbal ATC = HATC ATC classification of herbal substances; it differs from the regular ATC system by using 4 digits instead of 2 at the 5th level group The Uppsala Monitoring Centre is responsible for the Herbal ATC classification, and it is part of the WHO Drug Global portfolio available by subscription.  Defined Daily Dose (DDD) "Defined daily dose" This is the estimated average daily maintenance dose of a medicine used for its main indication in adults. DDDs are only assigned to medicines with an ATC code. It is an international compromise determined according to the available documentation - !not intended to select the dosage of medicines! Only one DDD value is specified for each drug, which is problematic e.g. for drugs that have different types of effect depending on the dose, therefore this value cannot be universally binding. DDDs for individual substances are usually based on monotherapy. The basic principle is to assign only one DDD for each route of administration within an ATC code. Different DDDs may be set if the bioavailability differs substantially for different routes of administration. Defined Daily Dose (DDD) The DDD is expressed in units: of weight (mg, g, ml...) international units (IU) units of a particular dosage form (1 tablet, 1 sachet, 1 ampoule, 1 aerosol dose...) Defined Daily Dose (DDD) In problematic cases, DDD may not be indicated. (for rare disorders with individual dosing...) DDDs are often the same for different dosage forms of the same drug. Different DDDs may be established if the bioavailability differs substantially for different routes of administration The basic principle is to assign only one DDD for each route of administration within an ATC code. The recommended maintenance dose (long-term therapeutic dose) is usually preferred when setting the DDD. The starting dose may differ from the maintenance dose, but this is not reflected in the DDD. Drug groups without DDDs topical products (most products in ATC group D) sera (ATC group J06) vaccines (ATC group J07) antineoplastic agents (ATC group L01) general and local anesthetics (ATC group N01) ophthalmologicals and otologicals (most products in ATC group S) allergen extracts (ATC group V01) contrast media (ATC group V08). Defined Daily Dose (DDD) The average adult dose recommended for the main indication as reflected by the ATC code. Where the recommended dose relates to body weight, an adult is considered to be 70 kg. It should be emphasised that even special pharmaceutical forms intended primarily for children (e.g. mixtures, suppositories) are assigned the adult ADI. Exceptions are some preparations used only by children, e.g. growth hormones and fluoride tablets.  The most important quantitative unit used for the purpose of studying the consumption of medicinal products Use of DDD Allows comparisons of the consumption of products of the same medicinal substance, different substances and therapeutic groups. Relative units for comparison purposes are derived from the DDD, e.g. number of DDD/1000 inhabitants per day (basic unit of comparison, comparing consumption of medicinal products in different regions) or number of DDD per 100 treatment days, number of DDD/1000 insured persons per day.  Purpose of the ATC/DDD system The purpose of the ATC/DDD system is to serve as a tool for monitoring the use of medicines and research to improve the quality of medicines. One of its components is the presentation and comparison of drug consumption statistics at international and other levels. It is essential that the drug consumption monitoring tool is able to cover most of the medicines available on the market. An important objective of drug utilization is to monitor both rational and irrational drug use, which is an important step towards improving the quality of drug use. It is widely used in our country in the compilation of positive lists. Therefore, the inclusion of a substance in the ATC/DDD system is not a recommendation for use and does not imply any judgement about the efficacy or relative efficacy of drugs and drug groups. The ATC/DDD system has been expanding outside Europe in recent years with the support of the World Health Organisation and has the prospect of becoming a universal tool for classifying medicines and quantifying need for healthcare purposes. NDC code The most widely used drug sorting system in the US environment The administrator and editor of this classification is the U.S. Food and Drug Administration (FDA), which also defines the conditions for registering medical products into the system It is therefore used, for example, by the FDA's medical product database  NDC code - format Is a unique 10 or 11 digit three segment numeric identifier, segments are separated by a hyphen Section 1 - represents the manufacturer or distributor - is 4-5 characters long Section 2 - called the 'product code' - 3-4 characters long Section 3 - 1-2 characters in length - refers to the packaging of the medical product  DIN code „Drug Identification Number“ A major classification system for medical products found in the Canadian market Established and administered by Health Canada, Canada's national institution DIN code is assigned automatically at the time of LP approval Both prescription and over-the-counter LPs are subject to registration Consists of 8 numeric characters  Essential medicines For the most important diseases. The list of essential medicines can be adapted in each country according to specific conditions The aim of national medicines policy is to ensure fair, equitable and affordable access to medicines. Essential medicines meet the priority health care needs of the population. Their model list is produced every two years by the WHO. It produces separate lists of medicines for adults (since 1977) and for children (since 2007). The latest ones were published on 07/2023. It is formed taking into account the importance and relevance to population health, knowledge of efficacy and safety. The WHO list is available at: https://www.who.int/publications/i/item/WHO-MHP-HPS-EML-2023.02 According to this list, more than 150 countries already create their own lists of indispensable medicines. Individual national lists consist of 300-600 items.  Dietary supplements are foods which are distinguished from foods for normal consumption by their high content of vitamins, minerals or other substances having a nutritional or physiological effect and which have been produced to supplement the normal diet of the consumer to a level which favourably affects his state of health. They are therefore not intended for the treatment or prevention of disease. Dietary They are not approved, the manufacturer or importer has only an information supplements obligation towards the Ministry of Agriculture of the Czech Republic, where the text of the Czech label of the food supplement must be sent before it is first placed on the 1/2 market; the manufacturer is not obliged to verify the effectiveness of the food supplement before it is placed on the market, it must only be harmless (long-term use should not lead to damage to health).  As long as the claimed effects of the product do not contravene the Dietary legislation on the labelling of food and food supplements, it is allowed to state them on the packaging and in other materials supplements 2/2 accompanying the product, regardless of their truthfulness.  Dietary supplements - legislation Under Directive No. 2002/46/EC ( on the approximation of the laws of the Member States relating to food supplements), food supplements are foods intended to supplement the normal diet and which are concentrated sources of nutrients or other substances having a nutritional or physiological effect, either alone or in combination, are placed on the market in the form of doses, in the form of capsules, lozenges, tablets, pills and other similar forms, as well as in bulk form, as liquid in ampoules, in dropper bottles and in other similar forms of liquid or bulk products intended to be taken in small measured quantities. Dietary supplements shall be marketed only in prepackaged form. Legislation on dietary supplements is not harmonised within the EU.  Dietary supplements - legislation Dietary supplements within the Czech Republic constitute a separate category of food, which is regulated by Decree No. 58/2018 Coll. (Decree on Dietary Supplements and Food Composition), as amended. This Decree was created by transposing Directive 2002/46/EC of the European Parliament and of the Council. Labelling of food supplements shall not attribute to food supplements properties relating to the prevention, treatment or cure of human diseases or refer to such properties. shall not contain any claim stating or implying that a balanced and varied diet cannot provide sufficient vitamins or minerals in general. nutrition and health claims may be made under the conditions laid down in Regulation (EC) No 1924/2006. Advertisement - 'food supplement'  Dietary supplements - legislation When placing dietary supplements on the market, it is necessary to consider whether the dietary supplement contains novel ingredients (i.e. ingredients that were not used for human consumption to a significant extent in the territory of the European Communities before 15 May 1997) that can only be placed on the market in accordance with Regulation (EU) 2015/2283 of the European Parliament and of the Council on novel foods, amending Regulation (EU) No 1169/2011 of the European Parliament and of the Council and repealing Regulation (EC) No 258/97 of the European Parliament and of the Council and Commission Regulation (EC) No 1852/2001. Dietary supplements are foods, not medicines, and are therefore subject to the relevant provisions of food law. In practice, this means that the entity that manufactures, imports or places a food supplement on the market (in whatever form - e.g. mail order, online shop, etc.) is considered to be a food business operator, with all the resulting consequences. Act No 378/2007 Coll., on medicinal products (Czech republic) Decree No. 228/2008 Coll., on the registration of medicinal products The boundary between food supplements and certain other product categories is very narrow and the categorisation of a particular product has to be done on a case-by-case basis.  Dietary supplements on the borderline Products for which there may be doubt as to their classification. Borderline products are considered to be either. A) medicinal substances or medicinal products (including homeopathic medicines) B) other products not regulated under the medicinal products regime (e.g. food supplements, cosmetics, medical devices or other categories of products with a special regulatory regime) Basic distribution of claims Claim: Health (Regulation (EC) No 1924/2006) Nutrition (Regulation (EC) No 1924/2006) Therapeutic (Regulation (EU) No 1169/2011) Other claims (covered by other or no legislation)  Nutrition and Health claims A claim is optional information that states that a food has a property. Nutrition claim - attributes beneficial properties to a food based on the energy, nutrients or other substances contained or not contained in the food → related to the content Health claim - indicates a beneficial relationship between a food category, food or food ingredient and health → related to the effect  Therapeutic claims Dietary supplements are allowed to make health claims but not therapeutic claims – which are only applicable to medicines. As such, companies should refrain from the naming of diseases and medical conditions in the advertising of dietary supplements. Therapeutic claims are completely prohibited for foods (dietary supplements). What can be considered therapeutic claims 1) Names of diseases 2) Names of symptoms 3) Words otherwise associated with (pain, shortness of breath, diseases or health problems (acute, (flu, diarrhea, acne...) vomiting...) chronic, prevention...)  Therapeutic claims It is forbidden to put a therapeutic claim on a food supplement verbally, but also to give the impression of it by other means. 1) Graphic 2) As part of the name of the product 3) By placing it in categories (on shelves and sections in pharmacies or individual tabs of online pharmacies...)  "Other claims" Some other claims are: regulated by other regulations - e.g. on flavourings (no flavourings), production labelling (organic, organic), claims for consumers with disorders (gluten-free, lactose-free) - must comply with the relevant legislation unregulated - slogans (for happy children, gives wings), lifestyle claims (active life, wellness, fitness), beauty claims (for shiny hair, for glowing skin) For some claims it is not clear whether they are health claims, nutrition claims or other claims. These claims are being discussed by various inter- departmental working groups of the respective departments (superfood, no side effects, non-toxic, alkalizes the body...).  Health claims - definition A health claim is any claim that states, implies or suggests a link between a food category, a food or one of its constituents and health. Health claims are always specific - e.g. Calcium is needed to maintain normal bone health. A health claim is not, for example: Avocados are filling. Or Fruit is healthy. Health claims - classification according to Regulation 1924/2006 01 02 03 04 Claims of disease Claims related to Claims supported Claims based on risk children's by generally new scientific reduction (Accor development and accepted knowledge and health (According scientific data patent-protected ding to Articles (According to claims (Under 15, 16, 17 and 19) to Articles 15, 16, Article 13.1) Art.18) 17 and 19)  Health claims In practice, health claims can be used that are. Approved by the European Food Safety Authority (EFSA). EFSA has already confirmed the efficacy on the basis of scientific evidence, (according to Article 13 or 14) Non-specific On hold - provisionally valid for which EFSA has not yet issued a scientific opinion. The obligation to comply with health claims in accordance with Regulation (EC) No 1924/2006 applies to so-called commercial communications - communications of claims relating to foods in terms of their nutritional value and health effects.  Health Claims Health claims are defined in Regulation (EC) No 1924/2006 of the European Parliament and of the Council of 20 December 2006 on nutrition and health claims made on foods. Purpose: to increase consumer protection and to level the playing field for food manufacturers, in particular for food supplements, -Health claims - scientifically substantiated and the active ingredient is present in the amount that produces the claimed effect. Stating, implying or suggesting that there is a link between a food category, a food or one of its ingredients and health. Claims of disease risk reduction which state, imply or suggest that consumption of a food category, food or one of its ingredients significantly reduces the risk of a particular human disease.  Health Claims The Health Claims Regulation applies to both the labelling of food (including food supplements) and their advertising. Recommendations from doctors and other health professionals are not admissible. Nor may food be attributed with disease curing properties. Health claims may be used after approval on the basis of scientific justification. A health claim shall not include a claim on vitality, life energy, beauty, etc. Health claims are FORBIDDEN for foods.  Verbal flexibility in health claims All health claims included in the list of approved claims may be used, except those that are patent protected. The approved health claims also allow PPPs to use the claim to refer to the more general, non-specific beneficial effects of a nutrient or food on overall good health and mental and physical well-being. The claim does not have to be used verbatim as approved; some flexibility in the wording of the approved claim is allowed. However, it is necessary that the modified wording of the health claim expresses the same relationship between the food category, the food or one of its components and health. EU Register on nutrition and health claims EU Register on nutrition and health claims Dietary supplements vs. pharmaceuticals  Dietary supplements vs. pharmaceuticals The packaging of the medicinal product and the package leaflet shall bear the so- called registration number in the prescribed format (e.g. 95/312/99-C) (Annex 4 and 5 to Decree No. 228/2008 Coll.). The packaging of the food supplement shall bear the designation 'food supplement' as part of the name (Article 3(1)(a) of Decree No 225/2008 Coll.). For some food supplements, it is possible to encounter the so-called HEM number, which some manufacturers still indicate on the packaging of these products. This is the number of the decision under which the product was previously approved by the Ministry of Health of the Czech Republic to be placed on the market as a food.  Medicinal products - SmPC SmPC = Summary of Product Characteristics The basic information in the SmPC includes. 1.the name of the product 2.qualitative and quantitative composition 3.pharmaceutical form 4.clinical data 5.pharmacological properties divided into f-dynamic and f-kinetic 6.pharmaceutical data, i.e. list of excipients, incompatibilities, expiry date, special storage precautions... 7.the marketing authorisation holder, i.e. the manufacturer or distributor 8.marketing authorisation number 9.date of marketing authorisation 10.date of revision of the text Examples from the pharmacy -When dispensing, the pharmacist must follow the SmPC (in the case of dispensing a medicinal product), in the case of selling a dietary supplement he must follow the approved health claim. Activated charcoal -Registered medicinal products (Carbosorb, Carbocit) are dispensed in accordance with the marketing authorisation decision, in particular the summary of product characteristics. The dispensing shall include the provision of information necessary for the correct and safe use of the medicinal products dispensed and for their storage. x -Dispatch of a food supplement (Carbo medicinalis PharmaSwiss) in accordance with an approved health claim.  Health claims vs. SmPC Glucosamine sulphate Chondroitin sulfate Medicinal products have in the SPmC for example. 4.1 Therapeutic indications Dietary supplements containing: CONDROSULF is indicated for the treatment of degenerative joint diseases, in particular gonarthrosis, May benefit from this approved coxarthrosis and arthrosis of the joints of the fingers of health claim. the hand. Contribute to the normal function The product is indicated for adult patients in Kellgren (flexibility, mobility) of the joints. stages II and III of the disease; it is less suitable for patients with very advanced disease.  REGULATION (EU) No 609/2013 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL - on food intended for infants and young children, food for special medical purposes and total dietary allowance for weight management Food for special groups This Regulation lays down compositional and information requirements for these categories of foods for particular purposes: a) initial and b) cereal-based d) replacement of a c) foods for special continued infant snacks and baby full-day diet for medical purposes; nutrition; foods; weight management. https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:02013R0609-20170711&from=EN  Infant formula and follow-on formula Infant - child 0-12 months ‚Infant formula' means food intended for infants during the first months of life and which in itself meets the nutritional requirements of those infants until the introduction of appropriate complementary foods ‚Follow-on formula' means foods which are intended for infants after the introduction of appropriate complementary foods and which form the basic liquid portion of a progressively expanding mixed diet for those infants Regulation in advertising: must contain only scientific and factually correct information may only appear in infant care and scientific publications must not lead to the conclusion that infant formula is equivalent to or superior to breast milk Food for special medical purposes (FSMPs) are evidence-based nutritional solutions for the dietary management of specific disorders or disease-related medical conditions. are highly regulated and should be used under medical supervision across a wide range of settings such as hospitals, care homes, clinics and patients’ homes. can partially, or entirely, replace a normal diet to provide patients with the essential nutrients they need to survive, recover and thrive. these foods include highly specialised foods, oral nutritional supplements (ONS) or metabolic products and can be taken orally or given as tube feeds. They can be a lifeline for patients of all ages. Food for special medical purposes (FSMPs) in the Czech Republic The types of FSMPs, requirements for their composition and the method of their use are determined by a decree of the Ministry of Health of the Czech Republic. Health insurance companies pay for FSMPs from public health insurance in accordance with the applicable legislation and the contract on the provision and reimbursement of health services. The amount and conditions of reimbursement of FSMPs are determined by SÚKL in accordance with Act No. 48/1997 Coll., on Public Health Insurance and on Amendments and Supplements to Certain Related Acts, as amended. Medicines sold outside of a pharmacy Vary widely depending on the country's regulations and local laws In many countries, certain over-the-counter (OTC) medications can be sold in places other than pharmacies 1.Pain relievers 2.Allergy medications 3.Cough and cold remedies 4.Antacids and digestive aids 5.First-aid supplies 6.Sleep aids 7.Herbal tea 8.Multivitain preparations  Rare diseases Rare diseases are clinically heterogeneous, predominantly inherited (or congenital) multisystemic diseases with a very low prevalence in the population, which impact on the quality of life and social inclusion of the patient, or threaten their life. A disease is defined as rare in the EU if it affects less than 5 people in every 10 000. Rare diseases are most often manifested early after birth and affect 4-5% of newborns and infants, but can also manifest later in childhood or adulthood. About 80% of rare diseases are genetic in origin, but for most patients the cause of their disease remains undiscovered. For example, cystic fibrosis, Epidermolysis bullosa congenita or butterfly wing disease, but also hemophilia or spinal muscular atrophy. The National Coordinating Centre for Rare Diseases was established in 2012. Its mission is to provide expertise and coordinate activities in the field of rare diseases.  Rare diseases in numbers 6000-8000 known rare diseases worldwide 30 million people suffer from a rare disease in the EU ( in the Czech Republic about 600 thousand) Only 5% of rare diseases have a known cure 75 % of rare diseases manifest themselves in childhood 30% of children with rare diseases do not live to the age of 5  Ultra-rare and undiagnosable diseases In Europe, an ultra-rare disease is one that affects one person in 50 000 (or less than 20 people in a million). According to Eurordis, 65,000 babies are born in Europe every year with an as yet undescribed disease. Also in the Czech Republic there are many people with ultra-rare genetic diseases (UVGO) and those whose disease has not been described or named (the abbreviation SWAN - Syndromes Without A Name). Ultra-rare people are often the only ones with the disease in the Czech Republic.  Medicinal products for rare diseases = Orphan drugs Previously, a very neglected group of drugs. Medicinal products that are not developed by the pharmaceutical industry for economic reasons, but which respond to public health needs.  Medicinal products for rare diseases In order to be recognised as an orphan medicinal product, a medicine must go through a design process within the EU, which is assessed by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP). The final - European Commission. Valid - for all EU countries. The availability of orphan medicines in the Czech Republic is relatively limited. When entering the Czech market, these medicines can obtain the status of a so-called highly innovative medicinal product. This status allows temporary reimbursement of the medicine - but for a maximum of 3 years. After three years, the manufacturer of the medicine must provide all studies and documents required by the State Office for Drug Control (SÚKL) to approve reimbursement for all medicines. As the medicines can be used by a very small group of patients, it is almost impossible to document all the studies and documents in the form required by the CMA. These medicines then disappear from our market because they are not recognised for reimbursement. If the patient needs the orphan drug and cannot replace it with any other medicine available on the Czech market, the attending physician can provide this medicine on the basis of Section 16 of Act 48/1997 Coll. (on public health insurance)  National Strategy for Rare Diseases According to EU recommendations, each country should develop its own strategy for rare diseases. The main objectives of the National Strategies include: 1. Awareness and Education 2. Data Collection and Research 3. Patient and Family Support 4. Access to Diagnosis and Treatment 5. Orphan Drug Development 6. Coordination and Collaboration 7. Legal and Regulatory Frameworks  Orphan drugs in Europe The EMEA aims to make orphan medicines rapidly available (at European and national levels). In Europe, drug policy on this issue was established much later than in the US due to diffuse competences in the health sector. On 16 December 1999, the European Parliament and the Council adopted Regulation (EC) No 141/2000 on orphan medicinal products. Current objectives of the European authorities. To support the pharmaceutical and biotechnology industry in carrying out research and development of orphan medicinal products. To ensure that competent SMEs in specialised sectors participate in this development. At the same time increase knowledge of these diseases, their environment, improve communication and cross-border cooperation between different research centres, institutions, patients, etc. Medical devices  Medical devices are used in many diverse settings. Such health technologies are used to diagnose illness, to monitor treatments, to assist disabled people and to intervene and treat illnesses, both acute and chronic. Medical There are an estimated 2 million different kinds of medical devices on the world market, categorized into more than 7000 devices generic devices groups. A medical device can be any instrument, apparatus, implement, machine, appliance, implant, reagent for in vitro use, software, material or other similar or related article, intended by the manufacturer to be used, alone or in combination for a medical purpose.  Medical device (MD) means an instrument, device, equipment, software, material or other article, used alone or in combination, including software intended by its manufacturer for a specific diagnostic or therapeutic use and necessary for its proper use, intended by its manufacturer for use in humans for the purpose of diagnosis, prevention, monitoring, treatment or mitigation of disease, Medical the diagnosis, monitoring, treatment, mitigation or compensation of an injury or disability, devices the investigation, replacement or modification of an anatomical structure or physiological process, conception control, and which does not achieve its principal intended function in or on the human body by pharmacological, immunological or metabolic action, but whose function may be enhanced by such action. Medical devices Medical devices: Devices, including the In vitro medical devices necessary software, used to (IVDs): medical devices diagnose, prevent, monitor, such as reagents, treat or mitigate disease; to calibrators, control diagnose, monitor, treat, materials or tubes for mitigate or compensate for performing diagnostic tests injury or disability; to such as blood tests for investigate, replace or infection or urine tests for modify the human body or glucose using material from a physiological process; or the human body. to control conception. What is the Personal protective equipment protects the difference user x Medical devices are used in the interest of the patient. between personal A personal protective equipment is a device designed and manufactured to be worn or held by a person for protective protection against one or more risks to their health or safety (for full text see Article 3 of Regulation (EU) equipment and 2016/425 of the European Parliament and of the Council). medical devices? Classification of medical devices Duration of use: Transient - Usually intended for continuous use for less than 60 minutes. Short-term - Typically intended for continuous use for periods of 60 minutes to 30 days. Long-term - Typically intended for continuous use for periods exceeding 30 days Risk classes: Class I (low risk) - patches, corrective lenses Class IIa (medium-low risk) - tracheal tubes, dental filling material Class IIb (medium risk) - X-ray machines, intraosseous screws and plates Class III (high risk) - heart valves, total hip replacements, breast implants Medical devices legislation The Regulations on Medical Devices (Regulation (EU) 2017/745) and on In Vitro Diagnostic Devices (Regulation (EU) 2017/746) The Medical Devices Regulation applies since 26 May 2021. Manufacturers must comply with the Regulation when placing new medical devices on the market. It repeals Directive 93/42/EEC on medical devices and the Directive 90/385/EEC on active implantable medical devices. The In Vitro Diagnostic Devices Regulation applies since 26 May 2022. It repeals Directive 98/79/EC of the European Parliament and of the Council on in vitro diagnostic medical devices. Medical devices legislation - EU IVDs fall under the scope of Directive 98/79/EC on in vitro diagnostic medical devices. Unlike medicinal products, medical devices and IVDs are not subject to pre-market authorisation. Member States designate independent organisations which ensure that a conformity assessment is carried out for all devices (except low-risk devices for which manufacturers themselves can certify the conformity). Manufacturers must report any incident causing death to, or damaging the health of, a patient to the competent authorities. There are also substantial differences in the interpretation and application of the rules in the Member States, which undermines the main objectives of the Directive, i.e. the safety and performance of IVDs and their free movement in the internal market.  Pharmacy Medical device dispensary Opticians Contract dispenser Where to buy medical devices?  Who can dispense a medical device A medical device may only be dispensed by a pharmacy or medical device dispenser by a pharmacist with specialised competence, a pharmacist with specialist competence, a pharmaceutical assistant with specialised competence for a specialist dispensing unit, a pharmaceutical assistant with professional competence, or an orthotic-prosthetic practitioner qualified to practise without professional supervision if the dispensing of an orthotic-prosthetic medical device is involved. An optical medical device may only be dispensed in an optician's shop. Such a medical device may be dispensed only by an optometrist, a qualified optician or a qualified ophthalmic technician, or an optometrist or an ophthalmic technician.  Summary Names of medicines and their classification systems Food supplements Foods for special groups Orphan drugs