A Decade of Transformative Treatments: Looking Forward and back in the Pharma Industry

What we asked: “Looking ahead to the next 5–10 years, what will be the key disruptors and/or what can be improved upon in the pharma industry?”

Response from: Anya Harry, Vice President and Chief Medical Officer, West Pharmaceutical Services

“In the last decade, transformative treatments have shown tremendous potential in increasing durability of effect and the hope for a cure in diseases as varied as sickle cell, blindness (an inherited form called Leber’s), spinal muscular atrophy, acute lymphoblastic leukemia, large B cell lymphoma, and hepatitis C. Advancements in gene therapy have been realized through the elucidation of the human genome and with the development of technologies, such as viral and nonviral vectors, to deliver these precious payloads.

“Progress in genomic sequencing technology led to much needed real-time and portable genomic surveillance during outbreaks in regions with limited public health infrastructure, as observed with the Ebola and Zika outbreaks of 2014. Such technology later enabled the industry’s swift response to the COVID-19 pandemic through rapid identification of the SARS CoV-2 pathogen, the development of therapeutics, and mRNA vaccines and public health measures – all in an unprecedented timescale. There are now several mRNA vaccines and RNA-based therapies on the market – as well as in clinical development.

“Whether sequencing organisms, or disease-causing mutations, the availability of genomic information, combined with newer capabilities for nucleic acid synthesis, gene editing, and novel delivery systems have played a key role in the growth of cell and gene therapy. Genetically modified cells for personalized medicine appear more attainable than ever before. As the cell and gene therapy markets grow rapidly, especially for rare diseases with limited treatment options, new companies are emerging, with currently more than 1,000 companies working on advanced therapies globally. These trends are contributing to growing pipelines due to improved processes, resulting in shortened development timelines. On average, drugs designated as Breakthrough Therapies have shorter premarket development and faster approval.

“The cell and gene therapy field is still in its infancy, and the targets thus far have been limited to liquid cancers and orphan diseases. The research has demonstrated the feasibility of these technologies in this area, but the real potential is in solid tumors, chronic diseases and regenerative medicine. To continue uninterrupted, it will be essential to address expectations from regulators in improving manufacturing, containment, and storage standards for all players. In addition to regulatory challenges, access to these potentially transformative treatments is limited due to costs. Solutions such as allogeneic cell therapies versus autologous may mimic a more traditional pharma model, thereby improving scalability – and potentially improving access.

“Additionally, the well established field of biologics – proteins, monoclonal antibodies and vaccines – is advancing rapidly, bringing new treatments and therapies to the forefront of medicine. There are several key emerging trends worth highlighting based on the therapeutic modalities that have become evident over the last decade. With monoclonal antibodies, for example, we see the expansion of product subclasses such as the biologic fusion of antibodies with peptides, proteins, or small molecules (ADCs); radionucleotide ligands, nanobodies; and Fab fragments, all with the hope of improving targeting and increasing efficacy.

“In the protein and peptide therapeutic modalities, we see the use of rational design helping to accelerate the synthesis of novel glucagon-like peptide-1 (GLP-1) agonists, alongside other approaches such as in combination with other molecules for dual and triple combinations for the treatment of diabetes and obesity.

“While it is valuable to look back and celebrate the achievements in our industry’s journey, it is imperative to highlight the anticipated areas where challenges require a call to action, including digital enhancements at home and potential interconnections with data platforms that may improve the patient experience. Such offerings (including large language models and generative AI) come with the promise of opportunities to improve decision making and streamlining care – although they may also be associated with cybersecurity risks and bias. Furthermore, the burden of healthcare is likely to continue to grow as vulnerable populations struggle with increasing chronic disease and the unknown impacts of a digital world. As we move towards solutions that may offer more personalized treatments and therapies, prevention and access should move to the forefront of all our efforts.”

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