Healthcare biotechnology has a tremendous impact on meeting the needs of patients and their families. Biotechnology in healthcare not only encompasses medicines and diagnostics which are manufactured using a biotechnological process, but also cell and tissue engineered products, and includes the use and the application of key biotechnology tools in the research and development of all innovative medicines. The majority of innovative medicines, whether manufactured using biotechnology or through a traditional process for small molecules, are made available by applying healthcare biotechnology.

From diagnosis to treatment and in terms of medicines manufactured, healthcare biotechnology is already delivering on its promises, read more about the difference that biotech medicines are making at our facts and figures page.

Innovative treatments

Healthcare Biotech is providing patients with innovative treatments to fight disease as well as innovative diagnostics.

Biotechnology is providing doctors with more tools which are helping to move from the treatment of diseases to prevention and cure. A whole new range of tools are being developed to support the human body to utilize its own capacities to fight infectious or cancerous diseases as well as injuries.

Thanks to the unravelling of the human genome, biotechnology is also increasing the number of disease targets for conventional drug therapy. Today conventional drugs target fewer than 500 disease targets, but in the future this is likely to rise to between 5-10 000 targets. The pharmaceutical industry will be powered more and more by biotechnology. The increased understanding of complex biological processes is opening up vast new areas of possibilities to fight and cure disease. Below are some more examples of how breakthrough biotechnologies are providing new solutions to disease, injuries and the wear and tear of life.

Unmet medical needs

"Healthcare biotech responds to patients' unmet medical needs"

Today there are many more diseases than treatments. Just 10 000 of the 30 000 known diseases have treatments available. Greater understanding of disease and the causes of disease is helping to produce better therapies that can more effectively address medical needs. New insights into the biology of disease and more precise understanding of why some people react differently lie at the heart of biotechnology. The promise of more targeted treatments to individual groups of patients as well as providing treatments for diseases that so far have eluded treatment are providing us with new opportunities to meet challenging but common diseases like heart disease, cancer and Alzheimers as well rare diseases.

Tailor-made medicines

"Healthcare biotech can tailor medical treatment to patients"

For patients, finding the right medication with less trial and error is critical. Healthcare biotechnology is helping to bring made to measure treatments to patients.

Improvements in diagnosis

"Healthcare biotech can help prevent and better diagnose disease"

Patients often have difficulty in getting a correct diagnosis. Biotechnology is offering new tools to doctors and patients to provide better diagnosis and more effective but less intrusive and uncomfortable testing for patients.

To find out more about innovative biotech treatments or how biotech is helping to meet unmet medical needs, improve diagnostics and tailor medicines to groups of patients, click on any of the following:

Advanced Therapies EuropaBio's Advanced Therapies homepage

“Advanced therapies” is an umbrella term that covers the three new techniques that are revolutionising modern medicine: cell therapy, gene therapy and tissue engineering. These techniques are already producing new treatments, and in the future are likely to produce many more.

In cell therapy, new cells are introduced into tissue to treat a disease – either from the patient’s own body (autologous), from another person (allogenic) or from an animal (xenogenic). One current example is the use of human adult stem cells in treating leukaemia. Around the world, researchers have been making progress towards using stem cells to treat conditions ranging from heart disease, spinal cord injury and brain damage to muscle damage, low blood supply and even deafness and baldness.

In gene therapy, genes are introduced to replace, repair or switch off defective ones. It has been used, so far with varying degrees of success, in severe combined immunodeficiency (SCID), cystic fibrosis, muscular dystrophy and sickle-cell anaemia, among others.

Tissue engineering is aimed at replacing or repairing defective tissue, and is generally seen as relating to bone, cartilage and blood vessels such as veins and arteries. This expanding field covers the techniques of bioengineering and cell biology (and often cell therapy itself). It is already being used to repair or replace burnt skin, for example.

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Rare diseases and orphan drugs EuropaBio's Orphan Medicines homepage

A disease is defined as rare in Europe when it affects less than 1 in 2,000 citizens. Rare diseases are life-threatening or chronically debilitating diseases with a low prevalence and a high level of complexity. 6000 to 8000 rare diseases have been identified, affecting about 30 million European citizens at some point in their life. 

The European Union has defined an orphan medicine as one that is intended for a life-threatening or seriously debilitating condition that has no satisfactory method of treatment and also:

  • Affects 5 people or less for every 10,000 citizens (known as the “prevalence criterion”);

    or 
     

  • Where the treatment – without incentives – would not justify the investment in its development (known as the “insufficient profit criterion”).

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Proteomics

Proteomics is the science which studies the physiological function of proteins and their effects on diseases. Some diseases are caused if genes do not produce the proteins (or enough proteins) the body requires or if the body produces wrongly folded proteins. Biotechnology is using recombinant (artificially created) DNA and cell cultures to produce missing or defective proteins. Replacement protein therapies include Factor VIII-a protein which is essential for the blood-clotting process and which some haemophiliacs lack, or insulin - a protein hormone that regulates blood glucose levels.

A great deal of research is ongoing to determine the role that proteins play both in the cause and cure of disease.

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Pharmacogenetics

Pharmacogenetics studies the effect genes may have on an individual’s response to a drug. Pharmacogenetics uses biotechnology-based technologies to not only better diagnose disease but also to provide new ways to match medicine doses and medical treatments to individual groups of patients. The evolving area of pharmacogenetics will increase both the safety and efficacy of treatments by diminishing the trial and error for patients trying to find the optimal dose and treatment. Pharmacogenetics holds great promise to offer more select drugs to treat elusive variations of common as well as rare diseases and widen the numbers of diseases that can be treated effectively as well as limiting the occurrences of adverse drug reactions on patients.

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Diagnostics

We can now detect many diseases and medical conditions more quickly and with greater accuracy due to the sensitivity of new, biotechnology-based diagnostic tools. A familiar example of biotechnology's benefits is the new generation of home pregnancy tests that provide more accurate results much earlier than previous test-generations.

Another good example is PCR technology: The Polymerase Chain Reaction (PCR) is a technology that imitates a cell’s ability to replicate DNA by generating multiple copies of specific sequences of DNA through amplification. In clinical diagnostics, a specimen of genetic material weighing only one-trillionth of a gram can be repeatedly copied by PCR to provide sufficient material to detect the presence or absence of a virus as well as to quantify its levels in the blood. PCR tests were the first that could accurately measure the amount of HIV in a patient’s blood. This provides reliable information on the disease course and shows when changes are needed in a patient’s medication.

A new blood test has been developed through biotechnology to measure the amount of low-density lipoprotein (LDL), or "bad" cholesterol, in blood. The new biotech test measures LDL in one test, and fasting is not necessary. We now use biotechnology-based tests to diagnose certain cancers, such as prostate and ovarian cancer, by taking a blood sample, eliminating the need for invasive and costly surgery.

The human health benefits of biotechnology detection methodologies go beyond disease diagnosis. For example, biotechnology detection tests screen donated blood and organs for the pathogens that cause AIDS, hepatitis and a variety of other infectious diseases. Doctors will someday be able to immediately profile the infection being treated and, based on the results, choose the most effective antibiotics.

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Genetic testing

The wealth of genomics information made available by the Human Genome Project is greatly assisting doctors in diagnosing hereditary diseases. There are currently over a thousand human hereditary diseases that can be identified using genetic tests. The majority of these tests detect the presence of a mutation or mutations in a single gene which lead to monogenic (single gene) disorders, most of which are relatively rare diseases.

Unlike monogenic diseases, there are many diseases that are caused by a combination of environmental factors and one or more hereditary factors. Many common diseases that affect many millions of people arise through complex interactions between the environment and a number of alternative genes called susceptibility genes. Soon doctors will have access to tests for detecting susceptibility before the onset of clinical signs if patients so wish. The presence of disease susceptibility does not always cause the disease but is a risk factor for that disease, just as smoking is a risk factor for lung or heart disease. These tests will identify patients with a propensity to diseases caused primarily by environmental factors, such as diet, giving patients an opportunity to prevent the disease by avoiding the environmental triggers. Genetic testing is also critical to the development of pharmacogenetics which uses biotechnology-based diagnostics to better diagnose disease and provide new ways to match medicine doses and treatments to the individual.

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