Biotechnology-derived medicinal products are fully part of EU healthcare but there are still many unknowns which call for adequate controls, the European Commission told EURACTIV.

Biotechnology is the use of living systems or organisms in the creation of industrial products. A biologic medical product is manufactured in a living system such as a microorganism, plant or animal cells.

According to a report published (September 2016) by the European Biopharmaceutical Enterprises (EBE), biologic medicines have truly transformative potential, offering cures to patients when previously at best clinical signs could be treated.

Bio-drugs’ potential

The share of biodrugs in the global pharmaceutical market is expected to reach about 20% in 2017, with the most traditional pharmaceutical companies now having biologic medicines in their pipeline, said Barbara Freischem, the EBE’s Executive Director.

The life-changing value of biologic medicines is illustrated with diseases like hepatitis C and rare diseases. “The modern hepatitis C drugs actually cure patients from the infection, and thus additionally prevent the infection of others,” she said.

Combined with biomarkers and diagnostics, it becomes increasingly possible to identify patients that are likely to respond exceptionally well or not at all to a given treatment option, she explained.

“This ‘personalised medicine’ or ‘precision medicine’ approach can spare patients treatments they are not likely to respond to and identify the option that is best for them,” Freischem said.

Getting rid of ‘silo mentalities’

But supporters of biotechnology admit there is still a long way to go to translate scientific discoveries into therapies.

Denis Horgan, the executive director of the European Alliance for Personalised Medicine (EAPM), told EURACTIV that patients are urgently awaiting new therapies – and too often not getting them.

“Researchers and companies also face significant development obstacles as only one-in-ten biotechnology entering clinical trials ever make it to the finish line,” Horgan said, adding that cutting-edge science has allowed the breakthrough of personalised medicine but it needs to be integrated into daily healthcare.

According to Horgan, the development of biotechnology will necessitate the removal of “silo mentalities” in different disciplines and with individual member state health systems.

“An illustration of this is when the European Medicines Agency (EMA) has approved 44 drugs since 1995 which address indications in one or more of these cancer diseases. However, only 15 of these therapies are correlated with companion diagnostics (CDx), based on 5 biomarkers,” he said.

Big data and clinical trials

Biotechnology therapies will also require complex international clinical trials involving highly selected patient populations, the collection of human biological material and the use of large databases for bioinformatics.

“There are, of course, huge collection, storage, sharing and – not least – privacy and ethical issues surrounding all these data,” Horgan said.

Another challenge is that many of the diseases treatable with modern biologic medicines include very rare conditions. Some are so rare that you may need all or most sufferers to take part in a trial.

“That means that there are none or very few to treat afterward when you are trying to obtain a marketing authorization,” said Freischem.

The cost

Cost is a major issue too. Biotechnology proponents say better diagnostics will ease the burden on healthcare systems in two ways.

First, by allowing a more preventive approach: gene technology will flag up the likelihood of an individual developing a particular disease and provide a good idea of how it will develop, thereby encouraging early intervention.

Secondly, effective treatment means that patients are much less likely to require expensive hospital beds and will be able continue working, thereby contributing to the economy.

EU state-of-play

At EU level, biotechnology-derived medicinal products are made available via a centralised process, in which the Commission grants a marketing authorisation valid in all member states after a scientific assessment performed by the European Medicines Agency (EMA).

European Commission spokesman Enrico Brivio said the EU pioneered the introduction of the concept of “biosimilar”, which are the “generics” of biological medicines, thereby supporting wider patient access to biological medicinal products.

For the EBE, the authorisation of biosimilars and of biologics of other categories aiming to treat the same condition can stimulate competition in the biologics market.

“Provided that the physician, who can best judge the specifics of biologic treatments, has a central role in making the treatment decision, such competition can contribute towards sustainability of health care systems,” Freischem said.

Referring to the advanced therapy medicinal products (ATMPs), which are made of cells or tissues as well as gene therapy, Brivio stressed that new scientific developments are creating the expectation that this new class of medicinal products can address some of today’s medical needs.

However, he warned there were still many unknowns and that adequate controls need to be pu tin place to prevent detrimental consequences for public health.

“It is also important to avoid that patients are exposed to treatments that do not work,” the Commission spokesperson said.

At the legislative level, the EU has adopted the ATMP Regulation, which according to Brivio, has protected patients from unsound treatments.

“Moreover, the Regulation has provided a clear legal framework, which has been instrumental in promoting innovation and securing a leading position of the EU in this field as demonstrated by the large number of clinical trial on ATMPs that are being conducted in the EU,” the EU official noted, adding that the executive is also working on the development of Guidelines on Good Manufacturing Practice specific for ATMPs.