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Emerging Therapies in Medicine: Are They Ready for Clinical Practice?
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Innovative therapies are swiftly progressing from the realm of research into the realities of everyday clinical care. In our most recent Pulse survey, we engaged over 2,000 physicians from across the globe to assess the preparedness of next-generation treatments for integration into clinical practice, as well as the barriers impeding their adoption. Delve into the survey findings to uncover physicians’ perspectives on the practical impact of these medical advancements, and how close they are to becoming a standard part of patient care.
- What Are Emerging Therapies in Medicine?
- Challenges in Bringing Emerging Therapies to Clinical Practice
- Global Survey Results: How Ready Are Emerging Therapies in Clinical Practice According to Physicians
- Future Perspective: Real-World Readiness in Emerging Therapies
Emerging therapies, such as genetic medicines, cell therapies, and multispecific antibodies, are transforming the landscape of a wide range of medical specialities. These approaches offer precision and personalisation, with the potential to transform the treatment of many diseases.
Oligonucleotide therapies, for example, are now licensed for conditions such as spinal muscular atrophy and Duchenne muscular dystrophy. Tumour-infiltrating lymphocyte therapy has been approved for advanced melanoma. Next-generation CAR-T therapies, including off-the-shelf and tandem-CAR designs, are under investigation for solid tumours.* Meanwhile, multispecific antibodies that activate T cells, block checkpoints, and target tumour antigens are progressing through early clinical development.
Despite their promise, these therapies face considerable challenges. Complex manufacturing processes, high costs, and evolving regulatory requirements can delay adoption. In addition, the need for specialised infrastructure and training may restrict their accessibility, particularly in healthcare systems with limited resources.
As these innovations move closer to mainstream practice, it is essential to consider their real-world potential from the perspective of healthcare professionals.
What Are Emerging Therapies in Medicine?
Emerging therapies refer to a new class of advanced medical treatments that go beyond traditional small-molecule drugs and standard biologic agents. Rather than focusing solely on symptom management, these next-generation approaches aim to address the root causes of disease, often through genetic, cellular, or immunological methods designed to repair, correct, or modulate biological dysfunction at its source.
Oligonucleotide therapies are a leading example. These short strands of genetic material can modify how genes are read or how proteins are produced. In spinal muscular atrophy (SMA), specific medications can increase production of the survival motor neuron (SMN) protein, improving motor function and survival in infants and children.*
On the other hand, the latest FDA-approved Tumour-infiltrating lymphocyte (TIL) therapy is used in cases of advanced or unresectable melanoma that has progressed after prior immunotherapy or targeted therapies.* The treatment consists of using a patient’s own T cells that have naturally entered the tumour. These cells are expanded in the lab and reinfused to enhance the immune response. This approval marks a significant milestone as it is the first time the FDA has approved an immune cell therapy for a solid tumour.*
On the list of advanced therapies in medicine under investigation are next-generation CAR-T therapies and multispecific antibodies. The first includes “off-the-shelf” allogeneic CAR-T products and tandem-CARs designs that recognise more than one tumour antigen, improving targeting and persistence in treatment.
Similarly, multispecific antibodies can enhance therapeutic specificity and efficacy by orchestrating complex biological interactions.* Still in early clinical development, they are promising in connecting different cell types, activating multiple signalling pathways, and offering more potent therapeutic effects, particularly in cancer and other diseases.
Challenges in Bringing Emerging Therapies to Clinical Practice
Next-generation treatments offer remarkable promise for the future of healthcare, yet they continue to encounter significant challenges related to manufacturing, delivery, safety, and cost before they can be fully integrated into routine clinical practice. Their development, production, and administration demand specialized expertise, infrastructure, and resources that differ considerably from those required for conventional pharmaceutical therapies.
Manufacturing complexity is one of the biggest hurdles. Genetic and cell therapies often rely on personalised production, each dose tailored to the patient’s own cells or genetic material. CAR-T therapies, for instance, require extraction, modification, and reinfusion of a patient’s T cells, a process that can take several weeks and must be performed in highly controlled facilities. Efforts to create “off-the-shelf” CAR-T or allogeneic cell products aim to simplify logistics, but raise new concerns about immune rejection and durability.*
Cost and scalability also remain significant barriers. Emerging therapies for spinal muscular atrophy can cost hundreds of thousands of dollars per year, posing challenges for payers and health systems. Manufacturing multispecific antibodies or oligonucleotide drugs at scale also demands specialised facilities and materials that drive up prices.
Infrastructure and training are equally critical. Administering advanced cell or gene therapies often requires specialised teams and centres capable of handling complex preparation, infusion, and monitoring protocols. These challenges can limit access, especially in regions without tertiary research hospitals or advanced laboratory support.
Ultimately, regulatory and reimbursement pathways continue to evolve. Traditional frameworks were designed for small molecules and biologics, not for living or genetic medicines. Balancing patient safety with timely access remains a key challenge for agencies worldwide.
Global Survey Results: How Ready Are Emerging Therapies in Clinical Practice According to Physicians
In our most recent Pulse survey, 2,071 physicians provided their insights on the preparedness of advanced therapies within modern medicine. Participants were asked to consider the following question: “In your view, how ready are emerging therapies to make a meaningful impact on everyday clinical practice, given the challenges they face?”
Globally, 37% of respondents believe that emerging therapies are promising in clinical care, although they are still in their early stages of development. The second majority, at 22%, agrees that they are nearly ready to be implemented, but a few barriers remain.
Considering the challenges, 20% of respondents acknowledge that high costs, inadequate infrastructure, and training needs limit the implementation of next-generation treatments. Only 5% of participants think it’s too early for advanced therapies to have a meaningful impact in everyday clinical practice.
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Future Perspective: Real-World Readiness in Emerging Therapies
As research advances and new approvals accelerate, the central question for many clinicians is not whether emerging therapies work, but how ready they are for daily medical practice. From a physician’s perspective, readiness involves more than clinical efficacy. It depends on access, practicality, and the ability to integrate these therapies safely into existing workflows.
Many doctors view these treatments with cautious optimism. The results seen with CAR-T cells in hematologic cancers and tumour-infiltrating lymphocyte (TIL) therapy in advanced melanoma demonstrate that engineered immune approaches can achieve lasting responses in otherwise untreatable diseases. However, logistical barriers — such as inadequate financial support, lack of regulatory knowledge, risks in using live tissues, and the complex path to market approval — make implementation challenging outside specialised centres.*
Similarly, oligonucleotide therapies and multispecific antibodies offer exciting new mechanisms of action but often require careful monitoring, high-cost delivery systems, and collaboration across multiple disciplines.
Accordingly, there’s a need for real-world evidence to complement trial data. While early studies show promise, long-term outcomes, quality-of-life data, and post-marketing safety monitoring are still limited for many of these therapies.*
The medical community recognises the potential of next-generation treatments to redefine standards of care. Yet, readiness for broad clinical use will depend on continued collaboration among researchers, regulators, and frontline clinicians to ensure that medical innovation translates into equitable and sustainable patient care.
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