Pharmaceuticals of the Future

The impact of innovative medicines on human health has been one of the great achievements of humankind.

FREMONT, CA: One of the greatest accomplishments of humanity has been the effect of novel medications on human health. Smallpox, diphtheria, typhoid, polio, rubella, measles, and mumps were once widespread and frequently fatal diseases, but they are now successfully controlled and frequently eradicated. With the FDA's clearance of the first biotech drug in the US, a genetically modified form of human insulin, a new wave of avant-garde medications was introduced in 1982.

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The biotech sector has since experienced fast expansion, and in 2021, the FDA approved the 100th monoclonal antibody therapy. With a compound annual growth rate of 12 per cent and more than USD 300 billion in global sales, biopharmaceuticals account for around 60 per cent of therapies under research.

Naturally, the public's awareness of biotech innovation was greatly influenced by the world's nervous anticipation for creating vaccinations and antiviral medications during the Covid pandemic. This culminated in the unprecedented accomplishment of developing the first batch of a candidate mRNA vaccine within a remarkably short time following the initial genomic sequencing of the SARs-CoV-2 virus.

A 50,000-step method for developing the mRNA vaccine was developed using numerous supply chain and manufacturing advances. Surprisingly, more than 7 billion doses of various vaccines have been distributed internationally in just 18 months.

Despite these successes, there is still much work to be done. For instance, just five per cent of the more than 7,000 rare diseases that impact more than 300 million people globally have viable treatments.

The Pace of Change

Monoclonal antibodies and recombinant therapeutic proteins have largely been responsible for the influence of biotech medications over the past ten years. The pipeline for new, highly inventive medications is filling up quickly, resulting in an unprecedented range of therapeutic approaches.

In December 2021, there were more than 12,600 projects in clinical development, with roughly 50 per cent of them being for small molecule chemistry products and 50 per cent for biopharma products.

These studies were spread over a variety of therapeutic areas, with oncology accounting for 50 per cent of them, followed by autoimmune diseases (11 per cent), central nervous system (10 per cent), and infectious diseases (7 per cent).

Another 20 per cent of these clinical development programmes are centred on monoclonal antibodies. Included in this are newer, more sophisticated multi-specific antibodies and antibody-drug conjugates (ADCs), which use highly selective monoclonal antibodies combined with other substances like chemotherapy medicines to target particular cells like cancers while preserving neighbouring healthy cells.

In another 4.3 per cent of these efforts, healthy, functional cells are used as the therapy to cure a condition or disease in which the patient's cells are ill or damaged.

Gene-modified cell therapy (348 projects, 2.7 per cent), in which a functional gene is inserted into a cell-based therapy, is particularly interesting. For instance, chimeric antigen receptor T-cell (CAR-T) therapies have shown to be incredibly effective at treating and successfully curing several blood malignancies that were once fatal.

In this personalised medicine, the T-cells are genetically modified and then reinjected back into the donor patient after being purified from the patient's blood cells. The modified T-cells activate the patient's immune system to selectively attack the particular malignancy.

Gene therapies, in which a patient's genes are manipulated to treat or prevent a disease, are one of the most intriguing kinds of treatments now under investigation (281 projects, 2.2pc). RNA treatments, such as antisense medications, obstruct messenger RNA translation and consequently stop the synthesis of specific disease-related proteins.

Oncolytic viruses, in which tumour-seeking viruses infect tumour cells and multiply inside them until the cells burst, release signals that enable the immune system to identify cancer and start an immunological attack on it. The first in-vivo phase 1 study for gene editing (CRISPR-CAS9) was also implemented in 2021.

A new drug must travel a long and arduous path from discovery to clinical testing to patient care. A new treatment takes an average of USD 2.6 billion and more than ten years to create, and just 12 per cent of products that enter clinical trials end up being approved for sale.

To beat these chances, a large amount of risk and money must be invested. For instance, Eli Lilly(NYSE: LLY) spends 25 per cent of its net sales on R&D and has invested enormous sums of money over the past 30 years in the search for Alzheimer's disease treatments.

An emphasis on flexible, agile facilities with the capacity to create a variety of products is pushing fundamental changes in manufacturing facility design, operations, and supply chain due to the diversity in the biotech pipeline.

Although there are many obstacles to overcome, including access to end-to-end high-quality data management from research through manufacturing operations, digitisation can be a fundamental enabler of a competitive manufacturing advantage.

New production process efficiencies, higher quality, and lower costs are still needed to be competitive. Key trends include the following, many of which depend on vendor innovation:

Multi-product, More Adaptable Facilities

Continued usage of single-use systems in clinical and commercial settings. Purification yields and expression titres have steadily increased as host cell lines, culture mediums, expression methods, vectors, promoters, and other factors have improved. Continuous processing, including downstream chromatography and upstream perfusion. Digitalisation and Industry 4.0 implementation, with an emphasis on automation and data analytics.

Biopharma manufacturing uses a lot of energy and water, so it's important to strike a balance between that and a constant focus on environmentally friendly practices.