As we step into 2024, the pharma and biotech industry experiences ongoing evolution. In this blog, I’ll aim to shed light on some of the significant developments, technologies, and insights poised to influence the sector throughout the upcoming year.
1. The rise and risk of cell therapies
Cell therapies, including revolutionary treatments like Chimeric Antigen Receptor (CAR) T-cell therapy had a massive surge in investment and use in 2023. The FDA has approved 6 CAR-T cell therapies to date, which have been used to treat terminal haematological cancers. CAR-T holds great promise but has been limited in broader application in solid tumours due to a lack of tumour infiltration. Current research aims to not only improve this and expand CAR-T’s beyond blood-based cancers, but also to apply CAR-T to non-oncological indications including severe autoimmune conditions.
In late 2023, the FDA reported that they had received up to 20 reports of T-cell malignancies (secondary cancers) in patients receiving CAR-T cell therapies, stating that they are now “investigating the identified risk of T-cell malignancy with serious outcomes, including hospitalization and death and are evaluating the need for regulatory action”. Developers stand behind the safety of their drugs and no causal relationship has been identified. For severely terminal patients, any risk of T-cell malignancy is far outweighed by the potential benefit of CAR-T cell therapy. However, as the therapy begins to be applied more broadly and to less terminally ill patients, the potential risks become much more pertinent. In 2024, whilst the CAR-T train is likely not to be derailed, developers will need to keep safety firmly in mind, particularly in autologous therapies, where the patient’s own cells are genetically modified with viral vectors, and reintroduced. It is this aspect that is suspected to increase the risk of secondary cancers. With the FDA spotlight more prominent in 2024, both preclinical and early phase data with CAR-T’s must ensure that safety is investigated thoroughly.
2. The importance of diversity
In 2022, the FDA announced a draft guidance aimed at addressing the lack of diversity in clinical trials. Clinical trial diversity is the inclusion of diverse participants who differ based on race, ethnicity, age, sexual orientation, geography and socioeconomic status. A lack of diversity in clinical trials has significant repercussions for patients and developers with clear disparities in efficacy depending on the recipient population. The goals for increasing diversity are to build trust, promote fairness and generate good biomedical knowledge. The directive from the FDA, investigator and public awareness will make clinical trials in more diverse patient groups a standard rather than outliers. This will not only benefit patients but allow drug developers and investigators new opportunities to explore varied responses and disease targets. For the scientific community, diverse trials will also lead to an improvement in our understanding of human physiology and response to disease and treatment. Investigators may seek to expand trial horizons, including populations previously underrepresented and largely disproportionally burdened. For 2024 and beyond, ethics in clinical trials, especially in relation to equitable inclusion will remain crucial.
3. Riding the waves: vaccine development continues to gain momentum
As many try and forget the “lost years” of the pandemic, the lessons learnt have been and continue to be relevant as we try and emerge from the COVID-19 shadow. The official end of the pandemic should be seen as a remarkable scientific triumph. A triumph not possible without an unprecedented public and private investment. Pre-COVID the investment in vaccine development was just under $1 billion. This increased to over $ 1 billion during the pandemic and continues to grow in the wake. Preparedness for the next pandemic is now of firm focus. It is still predicted, however, that investment in vaccine development still needs to increase 10-fold to properly prepare for another COVID-like pandemic. In 2024, and beyond, development both in infrastructure for early pathogen detection, manufacturing and importantly, equitable access to developed vaccines are of paramount importance. The latter was a major failing in the pandemic response and requires significant investment to ensure low- and middle-income countries are not left behind again when vaccines become available.
The COVID pandemic helped usher in a new age of vaccinology. On the back of the success on the mRNA COVID vaccine, the possibilities for vaccine developments are significantly wider and at the time of writing, there are over 3000 vaccine trials currently active cementing this is a major focus point for institutions and industry for the year.
2024 holds a great deal of promise in the biotech and biopharma industries. At Synexa, we are excited to play our role in improving human health, applying our wide range of capabilities and services to support all our clients on their respective journeys. The only constant in this industry is change and we are proud to continue innovating and working with our clients to best support research that is not only thought-provoking, dynamic and interesting, but also impactful.
Blog written by Dr. Nicholas Woudberg, Head of Scientific Strategies at Synexa
About Synexa and Scientific Strategies
Synexa is a specialized biomarker and bioanalytical services firm, established over 20 years ago. We have been supporting our clients at all stages of clinical development, from discovery to phase 3 studies. Our services cover analysis across the biological continuum, from nucleic acid, soluble biomarker, cell and tissue. Our team of dedicated scientists, working across labs in South Africa, Europe, the UK and US, relish solving intricate and challenging biological problems.
A key extension of this is Synexa’s Scientific Strategies Team. There are many challenges and risks progressing a compound into clinical development. Synexa’s Scientific Strategies team can help de-risk your development by providing support around key biomarker and bioanalytical considerations. The dedicated team can offer standalone consulting support, providing objective, actionable solutions to your biomarker and bioanalytical challenges.