Tim Farries
Principle Consultant, Biopharma Excellence by PharmaLex
Kate Coleman
Senior Director and Principal Consultant, PharmaLex
The development of COVID-19 vaccines has alerted the public to the importance of biotechnological advances in medicine and their promise for the treatment of many illnesses.
Similar to new vaccines, medicines referred to as gene therapies use modified viruses or other genetic material (DNA or RNA) to carry genes for the synthesis of therapeutic proteins in the body where they are needed. Several of these advanced medicines have been approved already, but more are in development for the treatment of genetic diseases, cancers and for tissue repair. Cures for once incurable diseases can now be foreseen.
Managing access to treatment
The COVID-19 pandemic has not only highlighted opportunities in these advances, but also the challenges in getting these therapies to patients. This translation of innovative science into commercially viable medicines requires skilled planning to anticipate and mitigate risks throughout their development.
Biological processes used to manufacture these novel therapies commonly suffer inherent variability that necessitates expertise to ensure they are produced with consistent quality and in sufficient volume to meet patient demand. Luckily, Ireland has long been an important hub for biological pharmaceutical manufacturing and the reputation of our workforce in this area is world renowned.
Supply chain issues
However, the pandemic has also brought significant challenges to this industry. Companies have experienced issues with the quality and availability of critical raw materials due to the impact of pandemic control measures on resources and supply chains.
Ireland has long been an important hub for biological pharmaceutical manufacturing and the reputation of our workforce in this area is world renowned.
Furthermore, the specialised manufacturing facilities have been appropriated for the COVID-19 vaccine production necessary to bring the world back to normality.
COVID-19 has provided an unprecedented public insight into the pharmaceutical industry and its ability to provide novel medicinal products that are safe and efficacious. This greater awareness demands full transparency regarding clinical data, regulatory approval and interpretation of the risks associated with novel medicinal products.
Seeking regulatory approval
The process for obtaining regulatory approval for gene therapies is immensely complicated, and this lengthy journey differs from the hyper-accelerated route made available for pandemic vaccines.
However, the successful delivery of COVID-19 vaccines to large patient populations worldwide demonstrates the feasibility for widespread delivery of gene therapies. Pharmaceutical innovators are now aware of more efficient regulatory routes of approval and of the wealth of knowledge and experience in the industry available to them, particularly in Ireland. These resources will be a key driver for future success, leading to increased availability of novel therapies to the patients that need them.