Medical biotechnology helps people with rare diseases

“Medical biotechnology is one of Germany’s high-performing sectors: in 2021 there were 26 approvals for new biopharmaceuticals; and other biotechnological therapeutics and vaccines are currently being tested in clinical trials in 669 projects. It is particularly successful because there is a high medical need for the drugs it produces. This is particularly evident in the treatment of people with rare diseases, for whom there were no treatment options at all for a long time.” That explained Dr. Frank Mathias, Chairman of vfa bio and CEO of Rentschler Biopharma SE, on the occasion of the publication of the industry report "Medical Biotechnology in Germany 2022", which the Boston Consulting Group (BCG) strategy consultants developed for vfa bio, the biotechnology interest group in the vfa.

In addition to an economic industry analysis, the focus of this year's report is on orphan drugs, i.e. drugs for the treatment of people with rare diseases. Of the 200 drugs that have been approved with orphan drug status since 2000, around 30% are biopharmaceuticals, i.e. genetically engineered drugs. They are used in different areas, but particularly often in rare forms of cancer or metabolic disorders. However, the fact that only around 8.000 of around 150 known rare diseases can be treated so far makes it clear that development work still needs to be expanded.

“It's not easy, and further support is needed. But some of the known difficulties in the field of orphan drug development can be overcome through collaborations between healthcare stakeholders," says study author Dr. Jürgen Lücke, Senior Partner at BCG. “Digital solutions developed by treatment centers and companies could make diagnosis easier and help those affected to receive effective therapy more quickly – or to participate in a suitable clinical study. Case registries set up cooperatively by users and companies could provide data for optimizing therapy with orphan drugs for more diseases than before.”

The report provides exemplary insights into what medical biotechnology can probably achieve for people with rare diseases in the coming years: T cells genetically modified in the laboratory could soon not only fight certain lymphomas and leukemias, but also solid tumors such as synovial sarcoma. Antibodies that specifically shut down components of the immune system should make it possible to treat autoimmune diseases such as cold agglutinin disease or myastenia gravis (a muscle weakness). Gene therapies are expected to be able to alleviate some serious visual disorders such as retinitis pigmentosa.

The Biotech Report 2022

The 60-page report analyzes the activities of all companies in medical biotechnology in Germany and illuminates the overall market (pharmacies and clinics) for biopharmaceuticals with data collected by IQVIA. It records original preparations as well as biosimilars. He also goes into the industry as an employer and orphan drugs.

He can under www.vfa-bio.de/publikationen can be downloaded as a pdf or ordered free of charge.

vfa bio represents biotechnology in the vfa, which is committed to the interests of its 48 leading global manufacturers in health, research and economic policy. vfa bio is committed to using the medical and economic potential of biotechnology and making Germany the leading biotechnology location in Europe. 30 companies currently belong to vfa bio.

Source:  Press release vfa bio from 07.06.2022