Advanced biotherapeutics

Biotherapeutics by definition require mastering complex innovative technologies, whether in their design, formulation or related analytical techniques. In addition, in many cases, their operating mechanism acts on the patient’s immune system, and certain molecules have demonstrated great therapeutic abilities but also significant side effects. As a result, several approaches are being pursued, such as targeted therapies, the use of vectors (a true extension of biotherapeutics), or personalized medicine (a fundamentally different approach).

What are the technologies of the future? How do we speed up R&D, in particular through the use of data and prediction models? How do we work on side effects? How do we improve patient targeting without switching to personalized medicine?

The production of innovative biotherapeutics has completely revolutionized the pharmaceutical industry. Macromolecules such as Anti-TNF-alpha, monoclonal antibodies, or recombinant proteins require specialized equipment and know-how. Bioproduction generally involves long processes, with many production stages including purification and quality control. The choice of the production line is key to obtain the right industrial performance. The production line sizing is delicate, and it is almost impossible to adjust it to cope with fluctuations in demand. Finally, production on several sites is a complex activity from a regulatory point of view. As the products are fragile and often injectable, they need to be sterile. These challenges are amplified for the new CAR-T cell therapies, which also require special logistical control.

How do we switch from batch production to continuous production? What are the best organisms to produce biotherapeutics? How do we reduce the length of the processes and the complexity of the controls while maintaining the same level of security? How do we improve the logistics of biologics? What industrial strategy should we adopt?

Innovative biotherapeutics are injectable products; they are therefore sterile products, and their administration may require the intervention of a healthcare professional. In addition, their circulation in the body depends on the injection conditions, and crossing the blood-brain barrier remains an unresolved challenge to this day. Finally, targeting an organ or a specific area of the body is a key issue to increase therapeutic activity while containing side effects.

What are the best technologies for developing targeted biotherapeutics? How do we go beyond the current limits of science? What are the solutions to deliver or activate the biologic to the right place? How can biotherapeutics be combined with other technologies to optimize them?

Biologics are expensive due to the complexity of their development, production, and supply chain. The increasingly significant therapeutic role they play therefore constitutes a challenge for healthcare systems. The marketing of innovative biotherapeutics is subject to extensive scientific and clinical demonstration phases, as well as economic phase, in order to fuel discussions with decision-makers in charge of the regulation and financing of these therapies. In addition, to improve their administration, some biotherapeutics are coupled with the use of a medical device, and these arrangements are struggling to find an administrative way for their marketing.

How to accelerate market access for innovative biotherapeutics? How do we demonstrate the value of biologics? How do we ensure the correct use of innovative biotherapeutics by prescribers?

The first biosimilars appeared in the first decade of the 21st century. Due to the complex nature of producing biological drugs, manufacturing a biosimilar is not straightforward, and the manufacturer must demonstrate the similarity of the active substances according to very strict specifications which involve holding clinical trials. The objective is to prove that the biosimilar will provide the same efficacy and safety as the reference biologic, after which its marketing authorization is issued. Despite these procedures, pharmaceutical manufacturers are interested in this path because of the size of the market, and they are restructuring a market that is still young.

What strategy should be adopted in the face of the arrival of biosimilars? Which priority market segments will biosimilars reach? What are the regulations in the different regions of the world?

Innovative biotherapeutics benefit from a generally positive image. However, their degree of innovation, their production process, and their use in severe pathologies can worry patients. In recent decades we have seen an increase in vaccine hesitation for example. Significant side effects or administration by injection hamper good adherence and compliance to treatment.

How do we measure and promote the acceptability of innovative biological therapies? How do we improve patient compliance? How do we place the patient at the center of the innovation process? How can we improve care pathways?