• What are the types of advanced therapies?

    • Cell therapy

    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).

    A person’s own cell and tissue can offer a patient a wide range of health care solutions, from prosthetic and restorative to therapeutic or even cosmetic in nature.

    Under normal conditions damaged joint cartilage does not – or only poorly - regenerates in the body. For several years now, cell therapy for restoring defects to knee cartilage has been available by growing a patient's own cartilage cells to repair cartilage defects. Active research, involving human cell-and tissue-based products, is being conducted in the regeneration and repair of bones, tendons, nerves and ligaments.

    • Stem cells

    Research into stem cells result in important cell-based therapies to treat serious diseases and conditions like Parkinson’s disease, Alzheimer’s disease, spinal cord injuries, as well as diabetes, stroke, burns, skin disorders and heart disease. Researchers work on three types of human stem cells – adult, foetal or embryonic.

    • Gene therapy

    Despite the high standard of today's medical treatments, and the number of already available drugs, many of the most debilitating human diseases do not have a cure yet. The molecular basis of many inborn disorders, such as haemophilia, cystic fibrosis or muscular dystrophy, has been exposed by the discovery of affected genes. In gene therapy, genes are introduced to replace, repair or switch off defective ones.

    In many forms of cancer, genetic predisposition plays as important a role as environmental factors in tumour growth, and malignancy. Identifying the gene for such diseases and redirecting its course is one of the most promising ways to cure certain diseases.

    Gene therapy has entered a phase of active clinical investigation in many areas of medicine. Human clinical trials have been started for the treatment of severe immunodeficiencies, cystic fibrosis, hypercholesterolemia, haemophilia, muscular dystrophy, many types of tumours (e.g. melanoma, prostate, ovarian and lung cancer), AIDS, and cardiovascular disorders.

    • Tissue engineering

    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.

  • What are "advanced therapies"?

    “Advanced therapies” is an umbrella term that covers the three new techniques that are revolutionizing 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.

  • Why are biotech drugs relevant to orphan diseases?

    Biotechnology provides powerful tools to develop diagnostics and treatments for orphan diseases. As most of the rare diseases are genetic, at least 80% of rare diseases have identified genetic origins. That is why medicines manufactured through biotechnology and gene technology can provide a solution to treat rare diseases. 

    In gene therapy, genes are introduced to replace, repair or switch off defective ones. One example of a genetic disease is the cystic fibrosis which is already treated by gene technology.

  • Why does there need to be a European Regulation about orphan drugs?

    Some 20-30 million Europeans are affected by rare diseases. Before the creation of the EU Regulation, there were almost no treatments for these diseases. Since the Regulation, more than 650 applications1 for orphan designations were submitted relating to over 200 different rare conditions. There are now 58 medicinal products (as of July 2009) that have received a positive opinion by the European Medicines Agency (EMEA).

    But challenges still remain. Patients still face delays in getting access to these approved treatments and there are still many diseases with no treatments.

    1 Summaries of opinion on orphan designation. EMEA website: http://www.emea.europa.eu/htms/human/orphans/opinion.htm (as of 15 September 2009)

  • Is there a European Regulation to encourage the development of orphan drugs?

    Yes, the Orphan Drug Regulation 141/2000 came into force in 2000, the Regulation states that patients suffering from rare conditions should be entitled to the same quality of treatment as other patients. 

    The aim of the regulation is written in article 1:
    .to lay down a Community procedure for the designation of medicinal products as orphan medicinal products and to provide incentives for the research, development and placing on the market of designated orphan medicinal products".

    The regulation was, therefore, created to stimulate research and development and to bring to the market the appropriate medications for orphan disease patients.

    The most important incentives are the market exclusivity for an orphan drug for a period of 10 years after the grant of marketing authorization, reduced fees for all steps of the market authorization centralized procedure at EMEA (European Medicines Agency). 

    The drugs need to meet certain criteria to be called as "orphan", the Committee for Orphan Medicinal Products (COMP) has the role to decide whether or not a drug is eligible to be called "orphan".

  • What is an “orphan drug"?

    It is estimated that over 6,000 rare diseases exist, although some say the actual number is closer to 8,000. Since the prevalence of such diseases is low and the development cost of medicines is high, industry is reluctant to invest in the absence of a foreseeable return.

    The medicines for these diseases or conditions are therefore indicated as ‘orphan medicines’, having no ‘sponsor’ or ‘parent’ to develop them.

    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”);
    • Where the treatment – without incentives – would not justify the investment in itsdevelopment (known as the “insufficient profit criterion”).
  • What is an orphan disease?

    In the EU, rare diseases that are also life-threatening or chronically debilitating with a low prevalence and a high level of complexity are defined as orphan diseases.

  • What is a rare disease?

    A disease is defined as rare in Europe when it affects less than 1 in 2,000 citizens (Orphan Drug Regulation 141/2000).

    Nowadays 6000 to 8000 rare diseases have been identified, affecting about 30 million European citizens at some point in their life.

  • Are there support instruments to provide assistance in IP matters to SMEs?

    IPR-Helpdesk and InnovAccess have joined efforts to provide assistance in IP matters to SMEs and other stakeholders.

    IPR-Helpdesk focuses on the specific IP issues involved in R&D and innovation in EU funded projects. The website is aimed at contractors taking part or wishing to take part in EU-funded research and technological development projects. Extensive free resources and documentation section on general IPR issues, how and why to protect ideas, a glossary and an IPR bulletin as well as specific information on managing IPR in an EU research project.
    InnovAccess gives you direct access to national resources and general IP issues. The website provides extensive general information on why and how to protect your ideas, on the costs of the protection and also lists specific rules for biotechnologies among other areas. Country specific information provided by the National Patent Offices is also available.
    Catalogue of IPR tools for small businesses

    This is a catalogue of online tools for businesses about intellectual property rights, developed as part of ongoing cooperation with the USA on this topic.