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Clinical Trials Q1 2024

Tackling challenges and taking clinical trial design into the future

Science technology concept. Scientific examination. Scientist.
Science technology concept. Scientific examination. Scientist.
iStock / Getty Images Plus / metamorworks

Dr Robert O’Connor

Director, HRB-National Clinical Trials Office

Decades of research are yielding promising avenues to manage and potentially cure numerous severe diseases. However, significant challenges lie ahead.

Rapid advancements in medical science are leading to exciting possibilities in clinical trials. New treatments like immune system enhancers for previously untreatable cancers offer hope for long-term remission.

While complete eradication of diseases might not always be the outcome, gradual progress is already clearly evident in the increasing number of individuals surviving serious illnesses like cardiovascular disease.

Evolutions in trial design

Trial methodologies are also evolving. Traditionally, trials focused on one medicine/technology addressing a single disease. Now, complex approaches like platform, umbrella and basket trials allow for simultaneous testing of various approaches against multiple aspects of a disease, leading to faster and more efficient results.

Additionally, with a deeper understanding of diseases, we’re witnessing a shift towards individualised medicine and once common illnesses are being classified into unique subtypes, paving the way for personalised treatment plans.

Truly personalised medicine

Gene editing, cellular therapies and novel vaccines are at the forefront of revolutionary advancements. Trials are exploring the potential of correcting faulty genes within the body or engineering immune cells or the wider immune system to combat diseases. Such trials are already underway in specialist centres in our nation.

Trial regulation grows ever more complex and costly.

It is also likely that we will increasingly see technology and medical approaches merged to give the best performance for a particular individual, and there is no doubt that machine learning (often misnamed AI) will increasingly figure, too.

Patients leading the way

Patient-centric trials are another crucial development. Previously often overlooked, patient needs and perspectives are now being increasingly prioritised. Patients actively participate in shaping trial design, ensuring clear communication and helping identify outcomes that directly impact their human wellbeing rather than the disease itself.

Advances come with challenges

Trial regulation grows ever more complex and costly. Regulations are vital. However, increasingly, lack of investment in the extra cost of regulation is impeding progress, and regulation without resourcing can wreck purpose.

Lifesaving technology comes at an increasing cost and, indeed, the first multimillion-euro treatment has recently been approved for a rare condition in Ireland. The economics of a ‘cure’ will increasingly tax us intellectually and practically, as will the growing tide of patients who can have great extensions to their lives but require ongoing and costly treatment.

Hence, arguably, we are reaching a new frontier where the challenges around trials and health technology are increasingly societal rather than scientific.

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