Conquering Technical Barriers for Plasma Therapies

By Admin
Posted 11 / 29 / 2016

The recent Economist Intelligence Unit survey revealed that plasma and other blood-derived therapies are still part of biopharma’s growth plans despite the excitement around novel therapies and continued momentum behind the development of recombinants. We spoke with Brand Name colleagues Pedro Almeida, Head of Advanced Drug Delivery, Global Marketing and Raphael-Johannes Guebeli, Technical Product Manager for Liquid Formulation, to get their take on the evolution of the plasma market, regulatory implications of plasma therapy development, and the challenges biopharma faces as they develop these therapies in different regions of the world.

Although the majority of survey respondents are focused on developing novel therapies and mAbs, 32% of survey respondents are already developing or plan to develop plasma or other blood-derived therapies. Can you tell me more about the plasma market and how you see it evolving?

Raphael: In the market for blood/plasma-derived therapies, I can see two different areas evolving. On the one hand, there is a niche market encompassing therapies being developed based on blood cells. Blood stem cells, for example, are already used in the clinic during the treatment of diseases such as leukemia or anemia. There is also a trend to develop more advanced blood cell-based therapies, e.g., by further differentiating blood-derived stem cells. This market is in early development without any products having noticeably reached market approval to date.

On the other hand, there is the more traditional, big market for plasma-derived protein therapies, most notably the IgG, human serum albumin and blood factor products.

The market for plasma-derived protein therapies is one of the first areas that evolved for the supply of therapeutic proteins, apart from insulin. The purification of therapeutic plasma-derived therapies began over 60 years ago and it’s a very established industry with only a few big players. While this market is mature, there are new developments to help make the processes safer, for example, reducing the risk of virus contamination, which is an ongoing challenge.

Pedro: There is definitely an impact on new approaches to blood-derived therapies following this trend. On the other hand, the more traditional plasma industry continues to show constant growth as there are therapeutic approaches where there are still no existing alternatives. In terms of growth and trends, some of the innovative solutions have to do with increased concentration of components, such as IgG. Guaranteeing the stability of these highly concentrated formulations, as well as patient compliance, continues to be the two needed continuous developments in an industry that tends to be more conservative.

The regulatory landscape remains a concern for biopharma as they look to expand into new markets and therapies. What kinds of regulatory implications arise with plasma-derived protein therapies?

Pedro: The plasma industry has a longer history within modern pharmaceutical manufacturing, where regulatory concerns have a larger impact and evolve with new notions of risk. The traditional plasma industry’s dependence on human-derived plasma (with multiple contamination risks for the patients) led to the need for a strong evolution and global harmonization of rules to guarantee maximum patient safety. Strong regulatory control around contamination and impurities, for example, continues to have a strong presence in this industry, and are also extended to new approaches, like recombinant plasma proteins, as a risk-minimization approach. This demands a constant evolution and care, not only with purification operations, but also with raw materials introduced in the process.

Raphael: I agree, there is strong regulatory control of the market for plasma-derived protein therapies. Despite the fact that the regulatory guidance is strong and straight-forward, there are still opportunities to improve and stream-line the regulatory processes. More companies tend to outsource the supply of raw materials, formulation and chemicals. We need to contribute to addressing these regulatory and safety concerns – a lot of companies rely on us to offer assistance and tools to solve regulatory challenges.

Are you noticing different challenges depending on the region of the world?

Raphael: Plasma-derived therapies are still expensive to produce and are costly as medications when compared to small-molecule medicines. How can healthcare systems in emerging countries provide these therapies to their patients? If you look at Asia, there is a noticeable movement to manufacture plasma-derived therapies locally. Will these emerging countries have enough financial resources in their health systems to afford these therapies? Will they become cheaper due to local production, improved processes and economies of scale?

Pedro: I agree. We have noticed certain trends in emerging countries to advocate for local production, as an attempt to expand access to products and control costs. For example, in Brazil there is a trend toward bringing processing facilities into the country itself. Proximity of manufacturing makes local access to therapeutics easier and in several cases is even incentivized by local governments, as part of the public health interest.