Emerging therapies in medicine are rapidly advancing from research stages to real-world care. In our latest M3 Pulse survey, we asked over 2,000 physicians worldwide about the readiness of next-generation treatments for clinical practice and the challenges they face. Explore the survey results to discover what physicians think about the real-world clinical impact of these innovations and how close they are to daily medical practice.
How are advanced therapies in medicine reshaping your area of practice? Share your thoughts in the comments section.
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 new or next-generation medical treatments that go beyond traditional small-molecule drugs or classic biologics. Instead of simply managing symptoms, many of these therapies aim to correct or modify the underlying causes of disease, often using genetic, cellular, or immune-based approaches.
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 hold enormous potential in healthcare, but still face challenges in manufacturing, delivery, safety, and cost before becoming part of everyday clinical care. Their development, production, and delivery require expertise, infrastructure, and resources that differ significantly from conventional pharmaceuticals.
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 latest M3 Pulse survey of 2,071 physicians, respondents analysed the readiness of advanced therapies in medicine. The survey question was: “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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M3 Pulse is a one-question online survey we conduct every month with our M3 panel members. It´s a fun and easy way to share your opinions about trending healthcare topics, like the shortage of physicians, with healthcare professionals worldwide. If you want to participate in this month´s M3 Pulse, register and join the M3 panel today.
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.
What’s your experience with advanced therapies in real-world care? Join the conversation in the comments section below and tell us where you see the most significant challenges ahead.
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