Revolutionary drugs that could mean the difference between life and death

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Series Details Vol.7, No.39, 25.10.01, p28
Publication Date 25/10/2001
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Date: 25/10/01

By Brian Ager

OVER the last century, European breakthroughs in pharmaceutical research and development have produced numerous treatments and cures for many life-threatening diseases.

Thanks to advances in vaccines, antibiotics and early-detection techniques, major achievements have been made in the fight against infectious diseases and childhood illnesses, some forms of cancer, nervous disorders, stomach ulcers, asthma, hypertension and AIDS.

While surviving childhood was a challenge in 1900 and the average life expectancy in Europe was only 55, a child born today can expect to live until almost 80. Research-based pharmaceutical firms are playing a leading role in the global fight against disease which ensures people not only live longer but have better quality of life. They have developed more than 90 per cent of all new medicines worldwide over the past 20 years.

A snapshot survey indicates that pharmaceutical and biotechnology companies are working on the research and development of more than 700 medicines and vaccines in all categories.

More than 130 medicines are currently in development to prevent and treat a range of infectious diseases, in addition to 100 new products aimed at fighting HIV and AIDS.

Also in the pipeline are 300 treatments to fight cancer; 120 for cardiovascular diseases (heart disease and stroke); 30 for arthritis, 25 for osteoporosis, 20 to treat diabetes, depression and asthma, 15 or so for Alzheimer's and schizophrenia and almost as many again for Parkinson's, epilepsy, and multiple sclerosis.

Here are just a few examples of major projects in late stages of research and development:Coronary artery disease: Probably the most common cause of death in the world. When cholesterol-lowering medicines were introduced, the risk of death from heart disease was substantially lowered. Current research is targeted at inhibiting an enzyme known as endopeptidase, which might open new horizons in cardiovascular treatment strategies.

AIDS: Although there is no vaccine yet, industry's commitment to address the global crisis has been rapid and massive. The disease was first recognised in 1981, and HIV as its causative agent by 1983. An antibody test to detect infection became available in 1985 and while there was only one treatment for HIV/AIDS in 1987, there are now more than 60.

Total anti-infective and other viral research continues to expand, with many compounds either in clinical trials or awaiting marketing authorisation. However, recent data suggest that while the number of anti-retroviral drug compounds in the pipeline peaked at around 250 three years ago, the number being researched has since declined to 150.

It may be more than coincidence that this has occurred since the advent of hostile attacks on patents and trade-related intellectual property rights (TRIPs) focused particularly on the HIV/

AIDS sector. Alzheimer's: As palliative treatments for Alzheimer's disease proliferate, the focus of future innovative therapeutics will turn to drugs with the potential to alter its course, which will exploit mechanisms in the brain other than nerve signals between interconnecting cells.

Cancer: Researchers are developing an arsenal of precision weapons that attack cancer in individual cells and genes. A new generation of molecules, able to target malignancy in cells more effectively, will be a powerful instrument in cancer treatment. Scientists are learning how to turn the body's own defences against malignant tumours instead of using chemotherapy, radiation or surgery. They have about 400 potential treatments in development, up from 20 a decade ago.

But there are still many challenges, as human beings are prone to more than 100 different cancers, any of which may take different forms and respond to different treatments.

Septicaemia: Currently in the final stages of human testing, a new generation of antibiotics targeting septicaemia is expected to be given a marketing authorisation soon and is seen as a breakthrough for doctors fighting a losing battle against drug-resistant bacteria.

The breathtaking pace of gene discovery in the last two decades, coupled with the birth of recombinant DNA technology, in which the components of human DNA are rearranged to address deficiencies on a genetic and cellular level, is providing new hope and has given rise to the concept of gene therapy.

With the unravelling of DNA, the primary building block of life, came an accelerated understanding of the structure of diseases. Each incremental innovation has built on previous discoveries and paved the way for the next, culminating in the current effort to sequence and understand the functions of all living organisms.

The application of the human genomics knowledge to clinical practice and drug development will allow us to predict a patient's individual response to treatment and create new "personalised" medicines according to genetic variations.

The application of these medicines will not only affect the global burden of disease but also impact on the typical pattern of health care, with an operational shift from acute treatment to prevention and cure. Early detection will lead to a better management of inherited susceptible diseases, such as arthritis, osteoporosis, cancer, heart disease, Alzheimer's, diabetes, asthma and depression. New pathways are also open for the treatment of rare disorders. A revolution is under way in both life sciences and technology, which will transform health care.

Our industry is playing a leading role in taking up this unprecedented challenge, which will be of benefit to all humanity. Research-based pharmaceutical companies are the engines of innovation.

In 2000 alone, the pharmaceutical industry in Europe has invested €17 billion - almost a fifth of all industrial research and development in Europe. However, it should be noted that this huge investment is almost entirely funded by private money and that it now takes on average 12 to 13 years for a promising new compound to be extensively tested and eventually approved as a new marketable medicinal product, which puts the average research cost for a single new medicine at €600 million.

As the development of pharmaceutical products is a high-risk endeavour, with very uncertain returns, it is imperative that intellectual property rights are protected so the revenues from these products can be used for supporting research in new and better products for diseases, including those which particularly affect the developing world.

Without the benefit of intellectual property protection, the resources for such research will be greatly diminished, and would ruin the hopes of millions of patients who look to the industry to find cures, as well as vaccines and treatments for conditions which affect them.

Evidence from around the world shows that strong intellectual property rights are the engine that has driven the discovery of the current arsenal of effective medicines (including anti-retrovirals), which have made the world healthier and are so desperately needed.

Weakening intellectual property rights for pharmaceuticals (as it is being debated in the WTO TRIPs Council) will not be a stimulus for greater access to essential medicines. On the contrary, it will only slow the development of the drugs which could eventually cure AIDS and other deadly diseases.

  • Brian Ager is director-general of the European Federation of Pharmaceutical Industries and Associations (EFPIA).

A review of European breakthroughs in pharmaceutical research and development over the last century. Article forms part of a special report on pharmaceuticals.

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