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Advanced Therapies

Advanced Therapies, i.e., new therapies, include gene therapies, cell therapeutics and biotechnologically processed tissue products, so-called tissue-engineered products.

These are also called Advanced Therapy Medicinal Products (ATMP). They are to be understood as pharmaceuticals for highly innovative therapeutic approaches which are developed on the basis of genetic information and biological material (human tissue, living cells). Due to technological and scientific advances, they open up personalized and targeted treatment options for serious diseases where "conventional therapies" fail. In addition, these are new, particularly effective therapies that can offer a better prognosis to patients with rare diseases for which no treatment option is currently available.

ATMPs undergo complex compounding technologies and special control procedures. The Committee for Advanced Therapies (CAT) is responsible for the scientific assessment of the quality, efficacy and safety of this diverse group of pharmaceuticals. In Germany, responsibility lies with the Paul Ehrlich Institute (PEI).

Alternative terms, abbreviations
Advanced Therapy Medicinal Products, ATMP, new therapy, advanced therapy

Ambulantisierung

Outpatient care refers to the outsourcing of social and health care services from the inpatient to the outpatient sector. In Germany, the principle “outpatient before inpatient” applies.

The focus is on the goal of effective, needs-oriented patient management. The aim is to make treatment conditions more attractive for patients. Outpatient care in hospitals is an important step in the structural change in German health care and was anchored in the coalition agreement. By shifting inpatient services to the outpatient sector, the federal government hopes to reduce costs in the German health system]. 

Antibiotics

Antibiotics are used to treat infections caused by bacteria. They can work in different ways to fight harmful bacteria.  Absorbed through the digestive tract, antibiotics eventually enter the bloodstream. From there they spread throughout the body and reach the site of infection. 
There they attack the harmful bacteria and inhibit their growth and reproduction. They support the body’s immune system in fighting the infection. There are also antibiotics that kill the bacteria directly by destroying the bacterial cells.

However, they are not suitable or necessary for every infection. For example, antibiotics are not effective against viral diseases such as most colds, flu or measles.

Nowadays, outpatient antibiotic administration is a suitable option to avoid or shorten hospital stays in well-cared-for individual cases if the general condition of the affected person allows it. In the treatment of long-term or severe chronic diseases, such as cystic fibrosis, antibiotic infusions are often used. These are part of an individual treatment decision between the treating physician and the patient.

Autoimmune disease

In an autoimmune disease, the body's immune system classifies the body's own tissues and cells as foreign invaders and attacks them. Instead of fighting harmful viruses or bacteria, which normally trigger a natural defense (immune response), the immune system attacks healthy cells and tissues and triggers destructive inflammatory processes.

In the gastroenterological field, chronic intestinal inflammations such as Crohn's disease and ulcerative colitis are among the most common diseases of this type. Inflammatory rheumatic diseases such as rheumatoid arthritis (rheumatism) are also autoimmune and represent complex clinical pictures. 

Auxiliary tariff

The auxiliary tariff for pharmacies is based on a contractual agreement between the German Pharmacists Association and the statutory health insurance funds and is a set of rules for calculating the price of non-industrially compounded pharmaceuticals, in particular formulations.

Biosimilars

Biosimilars are copycat versions of biological pharmaceuticals (biologics) that come onto the market after the patent protection of an already approved pharmaceutical has expired. These are biotechnologically produced generics that are similar to the active ingredients of the original preparations, but are not identical to them. The approval of biosimilars is carried out centrally in the EU and is regulated by several guidelines of the European Medicines Agency (EMA). During the approval process, it must be demonstrated that there are no clinically relevant differences in structure and function between the biosimilar and the already approved reference biologic.

lternative terms, abbreviations
Biologika, Biogenerika, Follow-on Biologics

Blistering

Blistering is part of modern medication management and can be understood as the repackaging and sorting of medically prescribed pharmaceuticals. It is a procedure that requires special equipment and trained personnel. In addition, blister packaging is subject to legal regulations to ensure the safety of taking the pharmaceuticals. Pharmaceuticals are packaged in an easily understandable and clear format in so-called patient-specific blister packs (tube blisters). These contain the correct dosage for a certain period of time.

Blistering helps patients who need to take medication regularly. It makes it easier to take and reduces the risk of incorrect dosage or confusion. It also allows for detailed documentation of the pharmaceuticals the patient is taking.

Cell and gene therapy

Cell and gene therapy is a type of advanced therapy and is based on the use of cells and genetic material. It is tailored specifically to each individual patient in order to control, treat or even replace diseased cells in the human body.

This is a promising approach for the treatment of serious and chronic diseases. These include, for example, chronic infections such as hepatitis B, certain types of cancer and genetic or congenital diseases of the immune system.

Intensive medical research and long-term clinical studies are necessary to prove the effectiveness and safety of these treatment options and ultimately make them available to those affected.

Alternative terms, abbreviations
Cell and Gene Therapy, CGT

Cell therapy

Cell therapy belongs to the group of novel therapies. In order to treat serious diseases such as cancer, living cells, i.e., the body's own cells or cells from a donor, are used.

The cell therapy used consists of cells or tissues that have been cultured in such a way that biological or structural characteristics or even physiological functions have been changed before they are transplanted into the body of the patient. Cell therapy may also involve artificially grown cells or cell lines (e.g., stem cells). This means that, like gene therapy, it is specifically tailored to each individual patient in order to repair, support or replace diseased cells in the human body. An example of cell therapy is the transplantation of T-cells, i.e., white blood cells that are intended to fight cancer cells in the patient. The administration of blood stem cells in leukemia is also included.

Alternative terms, abbreviations
Cell Therapeutics

Cleanroom

Cleanrooms are required, among other things, for special processes for the compounding of patient-specific therapies. This is an enclosed area in which the concentration of the smallest particles (such as dust) is kept very low and which places high demands on the environment and the people working there. To avoid influences from outside air, the cleanroom is cut off from the outside world. In addition, access is strictly regulated and only permitted to qualified personnel. All surfaces in the cleanroom are designed in such a way that no dissolved particles are released into the air. Efficient ventilation systems ensure permanent air filtration. In addition, strict rules of conduct apply in the cleanroom workplace, such as only allowing trained specialists to enter or avoiding rapid movements. 

Alternative terms, abbreviations
Cleanroom laboratory

Clinical study

A clinical study is the most important tool of clinical research in medicine. It is a scientific procedure that investigates the safety and effectiveness of new medications, treatments or diagnostic approaches in humans. They follow specific protocols and are carried out by a qualified team of physicians and researchers.

Clinical studies can be divided into different phases and run for several years until a final decision is made whether or not to approve the medication. Their purpose is to test new therapy options for various diseases. Clinical studies are also very important for finding out which treatments are not effective or safe. Participating in a clinical study can be an additional way for patients to gain access to new treatments. However, it is crucial that potential risks and benefits of participating in a study are carefully weighed in advance. It must also be ensured that the study is conducted ethically and in accordance with applicable regulations.

This online search engine provides insight into ongoing clinical studies for a wide range of diseases.

Cytostatic agents

Cytostatic agents are used in the treatment of cancer. A cytostatic agent is a toxic chemical substance that is used as a medicinal substance primarily in chemotherapy. It disrupts, delays or prevents the cell cycle and thus prevents tumor cells from dividing and spreading. Chemotherapeutic agents are often administered in the form of cytostatic parenteral infusion solutions. These cytostatic preparations are specifically tailored to the individual patient. Variables such as height, weight, age and gender are taken into account, as well as the type of tumor and additional diseases. Due to their immunosuppressive effect, i.e. their ability to suppress the normal function of the immune system, cytostatics are used not only for cancer treatment but also for chronic, non-malignant diseases and to suppress rejection reactions after organ transplants.

Alternative terms, abbreviations
Chemotherapeutic agent

Diagnosis

A diagnosis is the naming of a disease. It is the result of a clarifying process in which symptoms are identified and assigned to a known clinical picture or disorder based on medical examination findings. In addition to the therapeutic measures or a treatment plan, the diagnosis also determines the expected outcome of the disease. 

E-prescription

In Germany, the electronic e-prescription replaced the pink paper prescription on January 1, 2024. However, it is not yet valid for all prescriptions for statutory health insurance policyholders. For example, medical aids such as bandages or orthoses must continue to be prescribed using the so-called form 16 (pink paper prescription). Additional types of prescriptions will follow by 2026.

Only by e-prescription can insured persons receive prescription pharmaceuticals, the costs of which are covered by the statutory health insurance (GKV), including formulations and blood products, which are only dispensed in pharmacies. The e-prescription can be redeemed using an electronic health card (eGK), an app or in the form of a paper printout at local pharmacies or online pharmacies.

However, for pharmacy-only pharmaceuticals that are paid for by patients themselves, physisicans have the option of prescribing them either via e-prescription or paper prescription. Prescriptions for pharmacy-only and prescription pharmaceuticals at the expense of the professional associations and accident insurance funds can be made by paper or e-prescription. This also applies to cytostatic preparations. Depending on the practice management system, it can be decided here whether the corresponding functionality is provided.

Formulation

A formulation is a pharmaceutical that is compounded in the pharmacy on an individual basis based on a physician's prescription. Unlike a ready-to-use medicinal product, it is not produced in advance. The preparation of a formulation is only possible by trained pharmaceutical personnel (e.g. pharmacists). No starting materials other than those specified in the prescription may be used. Any deviations are only permitted with the consent of the prescriber.

The pharmacy is also obliged to check a new formulation before each preparation for its plausibility with regard to dosage, form of application, physician and quantity of the medicinally active ingredients. Formulated pharmaceuticals must also be labeled with information such as the name and address of the providing pharmacy, method of use, instructions for use, active ingredients, date of manufacture and expiry date on the containers or outer packaging. 

Gene therapy

Gene therapies belong to the group of novel therapies. They are used in medicine to treat genetic defects or hereditary diseases in those affected.

The corresponding pharmaceutical is called a gene therapeutic. This is a biological pharmaceutical whose active ingredient contains or consists of genetic information (based on nucleic acid). How is a gene therapy applied? For example, by removing some cells from the body and inserting the corresponding genetic information (nucleic acids) in the laboratory (ex vivo). The cells are then multiplied and reintroduced into the body. Gene therapy can also be carried out directly in the body (in vivo). Depending on the type of gene therapy and the technology used, the genetic information (nucleic acids) can be integrated into the cell genome (the totality of all carriers of the genetic information of a cell) or remain in the cell only temporarily.

What therapeutic effects result from this? By inserting genetic material or genetic information into human cells or tissue, faulty genes can be repaired. Or genes that cause a disease are switched off. Newly developed genes are used to stimulate the human immune system to treat a disease. For example, mRNA-based pharmaceuticals can be used as gene therapy for cancer immunotherapy.
 

Alternative terms, abbreviations
Gene therapeutics, Gene therapeutic

Generics

Generics are ready-to-use medicinal products with active ingredients that are no longer protected by patent. Generics are often approved under the name of the active ingredient in connection with the compounder. These genericmedicianal products are intended to be therapeutically equally effective as the original medicinal product (bioequivalence). The preparation and form of pharmaceuticals as well as the form of metabolism and excretion of medicinal substances may differ from the original preparation.

There are simplified approval requirements for generics. They are usually offered at a lower price due to lower development costs.

Genetic variants

Every person has an individual genetic makeup (genes). If this changes, so-called genetic variabilities can arise. 
In medicine, genetic variants help to understand genetic characteristics and identify the risk for certain diseases. For example, there are variants that can increase the risk of breast cancer. These findings form, among other things, the basis for the development of therapeutic approaches.

Alternative terms, abbreviations
Genetic variability

Good Manufacturing Practice

In order to ensure that pharmaceuticals are compounded to the prescribed quality, certain requirements relating to production processes and environment must be met. These are published by the European Commission in the Principles and Guidelines of Good Manufacturing Practice (GMP) for Human Medicinal Products and are used to regularly review the processes. For investigational medicinal products intended for human use, the Principles and Guidelines for Good Manufacturing Practice for Investigational Medicinal Products established by the European Commission apply. Strict monitoring of all processes ensures that medical products always have the correct identity, purity and content.

The principles of the GMP guidelines include: 

• Proven use of ready-to-use medicinal products approved in Germany
• Ensuring complete traceability of all raw materials
• Highest requirements for technical equipment
• Self-inspections
• Microbiological environmental monitoring of all production areas
• Strict staff hygiene
• Specially trained, qualified staff and continuous training of employees
• Separation of responsibilities in the independent work areas of production, quality control and release
• Regular official inspections 

Alternative terms, abbreviations
GMP, Good Manufacturing Practice

Health care

In Germany, health care services include general and specialist medical care as well as outpatient and inpatient medical care in hospitals. Treatment usually begins in the general practitioner’s office. If further specialist treatment is required, the general practitioner will refer the patient to a medical specialist - primarily on an outpatient basis and therefore in a doctor's office. If outpatient medical care in the physician's office is no longer sufficient, the patient is referred to a hospital for outpatient or inpatient treatment. There, the treatment is continued clinically by other medical specialists. Medical specialists therefore either practice in their own medical practice or work in a hospital. Outpatient and inpatient treatment are coordinated with each other – with "outpatient before inpatient" as the rule. Inpatient treatment in the hospital includes all services that are necessary for medical care - these include, for example, operations.

As part of health care, all insured persons are entitled to receive care with prescription pharmaceuticals and dressings. If necessary, the treating physician will issue a prescription for this. The patient takes this prescription to a pharmacy. The pharmacy dispenses the prescribed pharmaceuticals/dressings directly to the patient or customer. The costs will be billed to the health insurance company (stated on the prescription). 

Alternative terms, abbreviations 
medical

Health system

Health systems vary from country to country. The German health system is used as an example here. This is very multi-layered and can be divided into three levels:
 

• Framework set by government regulations: Federal government, states and municipalities in accordance with the German federal structure
• Design of health care by self-administration with its institutions and associations
• Concrete care by health insurance companies, medical specialists, a wide range of health professions, hospitals, pharmacies and pharmaceutical companies, e.g. in the area of ​​specialty pharma, which ensure the development, compounding and procurement of pharmacotherapies.

Detailed information on the German health system is available here.

Healthcare supply chain

In order to enable rapid access to individual and innovative therapies, cooperation between all players in patient care is critical. These include physicians, therapists, health and nursing staff - as well as hospitals, outpatient care and rehabilitation facilities and pharmacies. Their care services are usually covered by health insurance companies. In addition, medical scientists and pharmaceutical companies, for example in the area of ​​specialty pharma, make an important contribution to developing treatment options and establishing efficient paths within the healthcare supply chain.

Alternative terms, abbreviations
Care players

Hemophilia

Hemophilia, commonly known as “bleeding disease,” is a rare, hereditary disorder of blood clotting. As a collective term, this includes various forms of coagulation disorders, each of which requires very complex disease management.  

HIV

A well-known virus that occurs worldwide is the HI virus (HIV: Human Immunodeficiency Virus). This is mainly transmitted through sexual contact and causes HIV disease, which in its final stage is known as AIDS. AIDS stands for “Acquired Immunodeficiency Syndrome.”

In countries like Germany, HIV infection is now easily treatable thanks to modern medications. HIV therapy is also called antiretroviral therapy (ART). Antiretroviral includes effectiveness against so-called retroviruses. These are viruses that are able to incorporate their genome (i.e. the entirety of the genetic material of a cell or organism) into that of the host cell. This HIV therapy combines several active ingredients that reduce the proliferation of viruses in the body and thus prevent them from developing into AIDS. If treatment is started early, people with HIV have a good chance of having a normal life expectancy and quality of life. 

Alternative terms, abbreviations
HIV infection

Individual medizine

Individual medicine refers to the comprehensive orientation of pharmaceuticals, therapies and care structures for the individual patient. It not only opens up new perspectives for understanding how disorders arise and progress, but above all, new approaches to providing patients with the most tailored healthcare possible as well - with the aim of replacing mass medical structures. The further development of individual medicine therefore includes the following fields of action: ​

1. ​ The compounding of pharmaceuticals tailored to serious complex diseases (Specialty Pharma) and which can be individualized.
2. The tailor-made administration of pharmaceuticals for individual use through more flexible dosages or more individual packaging (blistering), which can also make it easier for the patient to take the medication and also enables an efficient use of resources.
3. Innovative (genetic) diagnostics, therapeutic approaches and solutions that can be tailored to patient groups and individual patients together with various healthcare players.
4. More efficient care platforms that - depending on the health system - enable involved players, therapies and patient needs to provide more individualized care - where it is needed.

​

Specialty Pharma, individualized or personalized medicine are to be understood as sub-terms. Individual medicine has already fundamentally changed many specialist areas and will contribute even more to improving the therapeutic spectrum in the future. 
 

Alternative terms, abbreviations
Precision medicine, personalized medicine, individualized medicine

Infectious diseases

The study of infectious diseases, derived from “infection”, is also called infection theory or infectious medicine. It covers the entire area of ​​infections and infectious diseases. It is an interdisciplinary science in the fields of biology and medicine that deals, among other things, with the research, prevention and treatment of viral and bacterial infections in humans and the pathogens that cause them. In Germany, the Robert Koch Institute, part of the Federal Ministry of Health, is the leader in health monitoring at the state level.

Alternative terms, abbreviations
Study of infectious diseases, Infection theory, Infectious medicine

Infusion therapy

Infusion therapy is the controlled administration of fluids over a specific period of time, which is carried out on an inpatient or outpatient basis depending on the clinical picture. An active ingredient is administered directly into the body through the blood via an infusion.
A distinction is made between three forms of administration: 

1. using an injection needle, which is often placed in the crook of the elbow and opens into a blood vessel that carries blood circulating in the body back to the heart or
2. through a catheter, i.e. a plastic tube, into a blood vessel (e.g. for parenteral nutrition or
3. as permanent access to a blood vessel (especially for chemotherapy).

The costs for infusion therapies can vary - depending on the clinical picture, the costs are covered by the health insurance companies or must be financed by the patient themselves.

Monoclonal antibodies

Monoclonal antibodies are special proteins manufactured in the laboratory. They function like a targeting tool that recognizes and binds itself to a certain type of target in our body. The principle is comparable to a key that only fits into a certain lock.

Our immune system produces natural antibodies to fight foreign invaders such as bacteria or viruses. These antibodies are very diverse and can bind to different targets. Monoclonal antibodies, on the other hand, are designed to recognize only a specific target in the body, such as a cancer cell or a virus. They are therefore unique ("monoclonal") and come from a single, specially developed cell.
The compounding involves immune cells that produce the desired antibody and are then replicated in the laboratory. This creates a large number of identical antibodies that target exactly the problem in the body. These compounded antibodies can then be used in medicine to specifically treat disorders. They either support the immune system or act directly against harmful cells.

Neurology

The medical specialty of neurology includes all diseases of the nervous system - that is, diseases of the brain, sensory organs, spinal cord, peripheral nerves including nerve roots, and muscles. These include epilepsy, multiple sclerosis (MS), Parkinson’s disease or stroke. These illnesses are already among the most common diseases today.

The treatment always depends on the underlying disorder. Effective patient-specific therapies are now available for many neurological diseases. However, there are still diseases that cannot be cured. The main goals of therapy are to alleviate symptoms and improve the patient’s quality of life.

Oncology

Oncology is a branch of internal medicine that deals with benign and malignant diseases of the blood, malignant diseases of the lymph nodes and the lymphatic system, as well as malignant solid tumors such as breast cancer or lung cancer. Cytostatics are used in the treatment of cancer. In addition to cytostatics, there are targeted agents such as monoclonal antibodies, which also inhibit the growth of tumor cells.

Ophthalmic

Ophthalmics are pharmaceuticals and preparations for the local or systemic treatment of eye diseases and injuries. For example, they are used for diseases and injuries of the inner and outer eye as well as the eyelids. In addition, ophthalmic agents can also be used as an aid in diagnostics as well as in the use and storage of contact lenses.

Ophthalmis are administered in the form of eye drops, eye ointments, eyelid ointments or as eye inserts and eye sprays. The active ingredients include antibiotics.

Alternative terms, abbreviations
Eye medicines, Ophthalmologics

Ophthalmology

Ophthalmology or eye medicine is one of the oldest medical disciplines and includes the study of the detection, treatment and prevention of diseases of the eye and the surrounding structures.

In our increasingly aging society, degenerative eye diseases are becoming increasingly important. AMD - the age-related degeneration of the macula (the so-called "yellow spot" on the retina of the eye and responsible for visual acuity) - is the most common eye disease in the Western world. AMD causes significant visual impairment in people over 50, even leading to almost complete blindness in the late stages. Even though AMD cannot be cured, various therapies can slow the progression of the disease and preserve vision for longer. In addition to ready-to-use medicinal products, patient-specific therapies  in the form of IVOM formulations are also used for treatment. “IVOM” stands for “intravitreal operative medication.” These are syringes that introduce certain medicinal substances into the back part of the eye, the vitreous cavity, during eye surgery or injection. 

Pain therapy

Pain therapyis a collective term for therapeutic procedures that influence acute or chronic pain and aim to relieve or eliminate pain.

There are different approaches to this:

• Pharmacological pain therapy, like analgesics, for instance. These are medicinal substances that attack pain points in the body, for example by docking onto receptors in the nervous system and blocking pain signals. This means that they actively intervene in the development, transmission and processing of pain, thereby weakening it or even eliminating it.  Spasmolytics are also part of pharmacological pain therapy. These pharmaceuticals act as antispasmodics and relieve muscle tension in the body by interfering in various ways with the natural process of muscle contraction. This is done, for example, by blocking excessive muscle movements by reducing calcium production in the muscle cells. Therefore, spasmolytics are used, among other things, to treat muscle or menstrual cramps. But spasmolytics are also used in gastrointestinal diseases such as irritable bowel syndrome.
• Non-pharmacological pain therapy, which includes acupuncture, physiotherapy and rehabilitation.

Parenteral nutrition

Parenteral nutrition is the direct infusion of nutrient solutions into the bloodstream. It is necessary when sufficient nutrition cannot be supplied via the digestive system, for example during or after cancer therapy, in severe chronic intestinal diseases, after operations or in the palliative phase.

With parenteral nutrition, nutrients are administered directly into the bloodstream via infusions, i.e. the gastrointestinal tract is bypassed. Typical nutrient solutions are glucose or electrolyte solutions, vitamins and trace elements. 
In addition, there are patient-specific therapies in the form of infusions individually tailored to the patient's nutritional status. For example, cancer patients undergoing chemotherapy or dialysis patients have different nutritional therapy requirements than patients in the intensive care unit. 

Parenteral preparation

In clinical usage, the term parenteral refers to the administration of pharmaceuticals or nutrients by injection, infusion or inhalation into the bloodstream, bypassing enteral absorption. The latter is a process in which endogenous or foreign substances are absorbed by the body's own cells via the intestine. Medicinal substances for parenteral administration are called parenteralia.

This type of preparation includes, among others, cytostatic preparations, monoclonal antibodies, parenteral solutions with painkillers as well as infusion solutions containing antivirals such as antibiotics or blood products. They are used, for example, in the emergency treatment of serious acute diseases in order to achieve a rapid and targeted effect. 

Alternative terms, abbreviations
Parenteralium, parenteralia, parenteral application

Patient Care

The patient care process includes all measures taken by medical professionals such as physisicans and other medical and nursing professionals as part of the care of a patient. The basis for this is:

• collecting the individual disease history (anamnesis)
• physical examination and laboratory tests such as a complete blood count or imaging procedures, e.g. X-rays (diagnostics)
• medically prescribed measures to maintain, improve or restore physical functions or organ functions (rehabilitation)

We speak of "integrated patient care" when medical services from the various sectors of the health care system (e.g. outpatient sector, inpatient sector, rehabilitation) are coordinated with one another.

Patient-specific therapies

In modern medicine, the individual clinical picture and the current general condition/physical condition of each patient are increasingly becoming the focus. Because every disease progresses differently and those affected respond differently to the therapies used. A form of treatment selected for the individual patient with the best possible effect and the fewest possible side effects is called patient-specific therapy. Molecular factors such as genetic variants are usually used as a basis.

Examples of patient-specific therapies are specially tailored infusion solutions such as cytostatic preparations for cancer therapy in oncology. Antibody solutions are also counted. In addition, pain relievers, antibiotics, ophthalmics and parenterals / parenteral nutrient solutions are classified as patient-specific therapies.  

Alternative terms, abbreviations
Personalized therapy

Pharmaceutical compounding (AMG)

According to the definition set out in the Medicines Act (AMG), the pharmaceutical compounder is the holder of the authorization or registration of a pharmaceutical and brings it onto the market under its name. Anyone who compounds pharmaceuticals and active ingredients or other substances intended for the compounding of pharmacueticls on a commercial or professional basis requires a permit from the competent authority.

The complete text of the German law can be retrieved here.

Prevention 

This includes any measure that can prevent or delay an impairment of health (disorder, injury) or make it less likely. In addition to vaccinations, examples of preventive measures include early detection examinations (e.g. colon cancer screening, health check-ups).

Rare disease

A rare disease, also called an "orphan disease," is a disease that affects only a small percentage of the population. In the European Union, a disease is defined as "rare" if it affects a maximum of 1 in 2,000 people or 5 in 10,000 people. In total, over 6,000 rare diseases are listed. The total number of people affected is therefore correspondingly high, especially because around 250 diseases are reported each year.

More than 80% of rare diseases have genetic causes and usually lead to lifelong, sometimes significant, limitations in quality of life. Around half of the rare diseases cause their first symptoms in childhood. In addition, they are very complex, have multi-layered characteristics and are not adequately represented in existing care systems. In addition to the often late diagnosis, the frequent lack of therapeutic approaches contributes to severe disease progression.

One example is Marfan syndrome, a genetic disease that affects the body's connective tissue. People with Marfan syndrome often have long and slender body proportions, joint diseases and heart problems. It can also cause tears in the aorta, the large artery that pumps blood from the heart to the body, resulting in serious health problems. There is no cure for the disease, but it can be treated by carefully managing symptoms. 

Alternative terms, abbreviations
Rare disorders, orphan disease

ready-to-use medicinal product

A ready-to-use medicinal product is a pharmaceutical that is available in a specific, specified form and dosage and is dispensed by the compounder in the form in which it is intended for use in the patient. These pharmaceuticals are usually compounded industrially and do not need to be modified or prepared before they can be used by the patient. Finished pharmaceuticals are ready for use and include products such as tablets, capsules, injection solutions, ointments and other dosage forms. In Germany, finished pharmaceuticals must be approved by the Federal Institute for Drugs and Medical Devices (BfArM).

The opposite of a finished pharmaceutical is a so-called formulation. This is compounded individually for a specific patient in a pharmacy according to a physician's prescription. 

Security of Care

When treating the person with a disease, “continuity of care” should be guaranteed. Good care also depends on “timeliness and accessibility” as well as “availability” and “consistency”. This means that regardless of factors such as the patient's gender or professional status, the same treatment must be ensured if the severity of the disease is the same. Therapy planning must be evidenced-based in order to ensure the effectiveness and safety of pharmaceuticals.

The high demand for therapies that are individually tailored to patients poses major challenges to health care. Why? Treatment with individualized medicine is, in the majority of cases, lengthy and costly. The development from a broad-based approach to medicine and medical care towards more individualized medicine is subject to some political, regulatory and economic limits, depending on the health system. The past few years have brought progress in Germany and Europe – especially in the area of ​​serious, consultation-intensive diseases (Specialty Pharma). Tailored solutions that deal more efficiently with limited or not quickly available resources for pharmaceuticals or pharmacotherapies and enable more targeted provision are becoming increasingly relevant. Digitalization creates a basis for innovative processes and network-like structures, which, despite increased costs for specialty pharma applications, can also be accompanied by a lower cost explosion for healthcare systems.

“Appropriate care” for a patient means that a medical professional make a judgment about what is the right care. The treatment should be geared exclusively to the person affected and not to the economic viability of the institution such as a hospital or medical specialist. Nevertheless, the goal is to achieve the best possible ratio between the resources used and the result. It is essential to inform and educate the patient about all therapeutic options. 

Serious disease

People with a serious disease require continuous medical care. This means that their care not only requires intensive advice, but must also be closely supervised by medical and nursing staff. Serious diseases are often also chronic, such as life-threatening cancer or mental disorders. These often develop gradually. Chronically ill patients live with permanent health restrictions and must constantly deal with the management of their disease. Most people are diagnosed with one or more chronic diseases during their lifetime. Sex or social background are not critical here. The risk of developing chronic disease increases with age, although the majority of patients with chronic disease are not very old.

The consequences of chronic disorders are serious - both for those affected and for the health system  and society. These include a reduced quality of life and functionality in everyday life (e.g. incapacity to work, early retirement), a significant use of health services and premature mortality.

Alternative terms, abbreviations
Chronic disease, Complex disease, Consultation-intensive disease

Specialty Pharma 

Specialty Pharma focuses on the care of patients suffering from serious diseases whose therapy requires special knowledge. The area includes the treatment of complex and consultation-intensive disorders such as cancer, hemophilia or neurological diseases. Pharmacotherapies that are specifically tailored to the individual patient are used, and these are cost-intensive.  

So-called specialty pharmaceuticals  also include individualized therapies, the preparation of which is based on important variables such as blood values, body surface area and weight. Overall, specialty pharmaceuticals can help to ensure better care for seriously ill patients through targeted therapeutic use.

Alternative terms, abbreviations
Special care

Specialized pharmacies

Specialized pharmacies focus on one or more specific subject areas including appropriate services. In addition to providing advice and dispensing medicinal products, these pharmacies produce tailored formulation pharmaceuticals for the individual patient (patient-specific therapies). The required raw materials are checked in the pharmacy. However, specialized pharmacies also produce pharmaceuticals for injection in the form of infusions (such as solutions containing active ingredients for cancer treatment).

In 2022, over 12 million formulations were compounded in Germany for those insured under the statutory health insurance scheme (GKV) alone.

Specialty pharmaceuticals

Specialty pharmaceuticals - also called specialized medicinal products - include individualized therapies whose preparation requires special knowledge. This is based on important variables such as blood values, body surface area and weight. In addition, compounding follows strict guidelines (Good Manufacturing Practice).

Early specialized medicinal products treated complex diseases affecting smaller populations, such as multiple sclerosis, cancer, pulmonary hypertension, hemophilia, and hepatitis C. Newer specialized medicinal products target widespread, more common chronic disorders that require taking a pharmaceutical for several years or for life. These include, for example, rheumatoid arthritis and asthma.

Health insurance companies and other cost carriers may classify the medicinal products or pharmacotherapies that they classify as specialty pharmaceuticals differently. However, what they all have in common is that they require more attention and management due to their high costs, complex administration procedures and intensive monitoring processes.  

Overall, specialty pharmaceuticals can help ensure better care for patients with serious disease through targeted therapeutic use and at the same time reduce healthcare costs.

Alternative terms, abbreviations
Specialized medications, Specialty pharmacotherapies, Special pharmaceuticals

Supply of medications

In Germany, the supply of medications is a crucial component of the health system and is ensured by over 500 compounders, around 20 supplier and approximately 17,000 pharmacies. It ensures that people receive the pharmaceuticals they need to treat diseases, relieve symptoms or prevent them. The pharmaceutical offering includes more than 100,000 registered medicinal products. These can be divided into over-the-counter, pharmacy-only, prescription pharmaceuticals and narcotics.

According to the provisions of the Medicines Act (AMG), ready-to-use medicinal products  may only be placed on the market in Germany if they are approved or registered or are exempt from approval or registration (standard approvals). On the one hand, the approvals can be granted nationally by the responsible federal authorities, the Federal Institute for Drugs and Medical Devices (BfArM) and the Paul Ehrlich Institute (PEI). On the other hand, this can be done centrally by the EU Commission after evaluation by the European Medicines Agency (EMA).

For an overview of relevant pharmaceuticals in Germany, click here.

Supply of medicinal products (AMG)

The supply of pharmaceuticals requires a permit, which in Germany is regulated by the following law: “Medicines Act (AMG) § 52a Supply of medicinal products”

The full text of the law can be found here.

Therapy

In medicine, the term "therapy" refers to the treatment of disorders, injury or other health disorders - with the goal of relieving symptoms, curing the disease, or stopping or slowing its progression. Therapy can take different forms depending on the type and severity of the disease and the individual needs of the patient.

Tissue engineering

Tissue engineering, also known as tissue culture, is the umbrella term for the artificial production of biological tissues through special cell or tissue cultivation. This aims to regenerate or replace diseased cells or damaged tissue in sick people.

How is tissue culture done? Artificial, special material compositions are used as a scaffold on which the cells (stem cells) are arranged. Through cell cultivation on this scaffold, the organisms are supplied with nutrients and thus grow into functional tissues. In addition, tissue engineering technology makes it possible to study cells and tissues in the laboratory to better understand the mechanisms of disorders. It also contributes to the development of new therapies and is used in active substance testing.

The resulting tissue engineered product (TEP) is classified as a biological pharmaceutical in the group of novel therapies. These include, for example, skin replacements, bone or joint implants and artificial blood vessels.

Alternative terms, abbreviations
Tissue engineering, tissue cultivation, tissue culture, biotechnologically processed tissue preparation, tissue engineered product, TEP