1.Why do we need research and development of the medicines?
Currently it is possible to treat only about a quarter of all diseases and the number of new diseases is constantly increasing. Pharmaceutical companies warn that the research and development of the new drugs takes very long time and it is costly and risky and because of all that and the high safety standards, it takes in average about 12 years. Also the price of the new drug development has increased several times and now the costs are about 1 billion euro, which equals to about one fifth of the annual costs of health care in Slovakia. To discover a new drug, we need to examine ten thousands of different chemical compounds and from the successful ones, only every third pays the costs of development. The importance of finding new original drugs is incontestable. Thanks to the genuine drugs, many diseases became curable or better tolerable (e.g. drugs can prolong the life of cancer patients, patients with mental disorders do not have to spend so much time in hospitals, HIV positive patients can thanks to drugs live without serious problems for decades; mortality from cardiac and vascular diseases has decreased). To secure our future and to be able to treat new diseases, we need to invest in research and development of new medicines.
2.Do we need all new drugs? How do the new drugs help?
Efficacy of the new original drugs can be easily assessed by comparing today's treatment options with the situation before 1990. Treatment level has increased in virtually all therapeutic areas:
3.How to verify the effectiveness and safety of medicines?
Drug manufacturers investigate and verify the safety of newly discovered drugs for many years in preclinical and clinical trials. Clinical trials take 3-10 years and thousands of healthy volunteers and patients participate. Research- oriented manufacturers conduct such trials also in Slovakia – they annually invest millions of euros in clinical research in Slovakia, which involves hundreds of doctors and thousands of patients. Clinical trials of new investigational medicinal products can be carried out just after being approved by the State Institute for Drug Control (SUKL) and the conditions under which it can be carried out, are specified in the law no. 140/1998 Coll, on medicinal products and medical devices.
However, the research continues also after getting the marketing authorisation and registration. Drug manufacturers assess and monitor the safety and efficacy of the drug in the so-called post-marketing trials. It may happen that a new indication of a particular drug can be discovered, which might mean a new direction of the research. E.g. aspirin, which was originally intended for the treatment of pain and as antipyretic, is now used as prophylaxis of heart diseases.
4 Why is the research and development of drugs risky, costly and time consuming business?
Only 250 compounds out of 10 000 newly discovered reach the pre-clinical phase. Only very few enter the clinical phase and only 1 of 5 tested in clinical trials is approved by the regulatory agency and registered.
Therefore it is not surprising that the number of newly discovered compounds is not particularly high. Also alarming is the fact that the number of newly discovered compounds is steadily decreasing. Despite of this fact, the costs of new drug development are constantly rising. This is primarily due to the ever increasing demands on the safety and efficacy of the new drugs. From the medical and ethical perspective, it is desirable that the new effective drug was available to the largest possible number of patients as soon as possible after the registration. Profit from the sale of the drug can the manufacturer then use for the further research.
After the end of patent and data protection is a genuine know-how made available to generic drugs manufacturers.
Good Clinical Practice (GCP) – is a set of internationally recognized quality requirements on ethical and scientific issues that must be followed when creating the design of the trial and also during the whole conduct of the clinical trial , in preparation of clinical trial documentation and clinical trial reports in trials that are carried out in humans. Compliance with the good clinical practice ensures and guarantees the protection of the rights , safety and health of the people participating in the trial, who are administered the investigational product or investigational drug or who are enrolled to the control group. The credibility of the clinical trial results is ensured.
Clinical trials on medicinal products (Clinical Trial) - all research done in humans, establishing or confirming clinical effects, pharmacological effects or other pharmacodynamic effects, which demonstrate any kind of side effects and which are used to find the absorption, distribution, metabolism and excretion of one or more investigational medicinal products for human use in order to determine their safety and efficacy; clinical trial also evaluates the bioavailability and bioequivalence of the study drug.
What is a clinical trial?
Clinical trials of products (future drugs that have not been registered yet) or medicinal products for human use is done in a healthy volunteers or patients. Clinical trials in humans are conducted in compliance with the Law on Medicinal Products (Act no. 140/1998 Coll medicines and medical devices) and also it needs to comply with the conditions of biomedical research determined by the special regulations.
Clinical trials are carried out in four phases:
What is the active ingredient, drug and medicine ?
The active substance, respectively drug is chemically uniform or non-uniform substance of human, plant, animal, chemical or natural origin, which is the carrier of the biological effect that can be used as a protection from the disease, for the diagnosis of the disease, treatment of diseases or for the modification of physical function. Medicinal product is a drug or combination of drugs and adjuvants adapted by a technological process to a dosage form, intended to protect from the disease, help to diagnose the disease, treatment of the disease or for modification of physical functions.
Human medicine is intended for use in humans.
Do you know what is meant by 'adjuvant'?
An adjuvant is a chemically uniform or non-uniform substance, that in an amount used for the manufacturing or preparation of the medicinal product itself has no therapeutic effect, but allows or facilitates the production or preparation of the product, helps its administration, improves its quality or stability and bioavailability.
What does the term drug registration mean?
Drug registration is the marketing authorization and record on the list of registered products.