The business model of biotech firms often relies heavily on intellectual property rights, in particular patents, as they are often the most crucial asset they own in a sector that is extremely research-intensive, high risk, and with relatively low imitation costs.
This is particularly the case for SMEs, many of which are relatively recently established, and an important number of which have yet to take a product to market. Often, biotechnology SMEs are established on the basis of one or more patents developed within, or in partnership with, public research organisations or universities, and thus, these intellectual property rights are essentially their products for a long time – for some, even the final product. Investors in biotech companies are generally well aware of the centrality of patents and the survival of such companies may very well depend on their ability to convince investors that they have a solid IP strategy and that risks are kept to a minimum. Therefore, a support network to ensure SMEs are protecting their IP as solidly as possible, is vital.
Biologicals have the inherent potential to provoke (unwanted) immune reactions. Therefore, immunogenicity assessment through clinical studies plays a major role in the development of biological medicines.
Interchangeability of medicinal products refers to the situation where one product is “switched” for another equivalent product in a clinical setting, without a risk of an adverse health outcome. Regulatory agencies such as the European Medicines Agency (EMEA) do not assess the interchangeability or substitutability of a biosimilar.
Currently, no clinical studies have been designed or undertaken to assess the clinical outcome of repeated switches (changes) of a biological medicine, whether using two original biological medicines or an original and a biosimilar.
The EMEA has specifically stated that “since biosimilars and biological reference products are not identical, the decision to treat a patient with a reference product or biosimilar medicine should be taken following the opinion of a qualified health professional”*. In addition, there is currently no convincing scientific data to prove that repeated product switching of biological medicines (whether biosimilar versions or not) does not lead to negative clinical consequences.
For further information please see our publication about Biosimilar Medicines
Biosimilar medicines are follow-on versions of original biological medicines. Biosimilars can be developed during the period in which the originator product is protected by patent exclusivity, but they can only be marketed after the patent protecting the originator product has expired.
Biosimilar medicines are independently developed to have the same mechanism of action as the original biological medicines, and are designed to treat the same diseases as the innovator’s product.
For further information about biosimilars please see the European Commission Consensus Paper “What you need to know about Biosimilar Medicinal Products”, which is available in English – German – French – Spanish – Italian – Portuguese.
Innovative medicines generally benefit from a certain period of intellectual property protection via patents and other exclusive rights such as data protection and market exclusivity.
Patent rights give the patent holder (often, but not always, the manufacturer), the right to prevent others from manufacturing, selling, using and importing a product, or using a process or selling a product made by that process during a limited period of time, e.g. 20 years from the date of application.
Data and market exclusivity mean that there is a period of time after approval before a competitor can enter the market with a follow-on product that relies wholly or partly on the originator’s data on safety and efficacy for its regulatory approval. The follow-on product can often use an abbreviated regulatory approval procedure.
Follow-on versions of chemical medicines that enter the market after expiry of IP protection are called “generics”. Follow-on versions of biological medicines are called “similar biological medicinal products” or “biosimilars”. In both cases, the originator product is called the “reference product”.
For further information please see our publication about Biosimilar Medicines
Manufacturing biological medicines is generally more complex than the production of traditional (chemical) pharmaceuticals. There are a number of reasons for this, including the nature of the starting material and the very high level of precision required in the manufacturing process.
The starting material for most biological medicines is a genetically modified cell line. Each biotech company uses unique cell lines and develops its own proprietary (unique) manufacturing processes to produce biological medicines.
The unique starting material and the complex manufacturing processes mean that it is not possible to exactly reproduce a biological in the same way a pharmaceutical (chemical) generic can be produced.
Biological medicines are far more complex and usually much bigger than chemical medicines, which are produced by chemical synthesis. Size is one of the most obvious distinctions: the molecules of a biological medicine are much larger, more complex, mimicking substances produced by the human body (such as enzymes, insulin, and antibodies) than the small molecules which make up classical drugs (see the picture - molecular weight as an indicator of structure complexity).
Biological medicines are produced using a living system or organism. This means that their manufacturing and precise characterization tends to be more difficult than for chemical medicines, which are more easily identifiable and can be exactly reproduce
EuropaBio is a founding member of the EPAA – the European Platform for Alternative Approaches to Animal Testing, and fully supports the principle of the “3 Rs” (refine, reduce, replace animals in testing). We fully support to minimize animal suffering in testing and promote biotechnology as being at the cutting edge in delivering alternatives to animal testing.
EuropaBio has successfully promoted biotech companies researching and providing alternatives to animal testing. A study has reported that four new biotech testing methods can reduce the need for animals and cut validation times in half. These newly discovered assays for bacterial contamination detection (known as pyrogens) have the potential to reduce animal tests in Europe by 200,000 rabbits each year. Most interestingly, they can also be used for new cell therapies where no appropriate test had previously been available.
For more in-depth information on EuropaBio’s position on animal testing, please see our Position Paper, which you can find here.
Personalized medicines are an exciting new area that is becoming possible because we can now identify more accurately the right treatment for patient through analysis of their genetic data.
New technologies such as pharmacogenetics and proteomics use biotechnology-based technologies to give a better diagnosis of a disease by using patients’ genetic information, and also to match the right drug, at the right dose, at the right time to the patient. The evolution 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. These new technologies are contributing to the development of personalized medicines.
Personalized medicines are about tailoring medical treatments to patients. For patients this means finding the right medication with less trial and error and helping to deliver made-to-measure treatments.
Many years can be spent in identifying the therapeutic molecule, determining its genetic sequence and working out a process to make the treatment stable, biologically active and reproducible. As a result, the biotech industry spends more on research and development (about 20- 25% of revenue) compared to the mainstream pharmaceutical industry (about 15% of revenue).
A biotech treatment requires specialized and complex manufacturing techniques and distribution processes, making the mass handling of therapies very difficult. Since biotech drugs are derived from natural sources, they are often less stable than synthetic molecules and necessitate special handling and distribution.
Most biotech treatments can not be administered orally, but need to be injected or infused. This requires a high purity and sterility for biologic medicines, adding to their cost.
One of the main benefits of biotech treatments is that they often target patients with relatively uncommon diseases or those who constitute a small subset of patients with a highly prevalent condition such as asthma. However, the development and approvals process costs the same as a more widely applicable treatment, but with a smaller patient basis that can take advantage of the treatment.