What happens when a patient does not have access to sufficiently effective or appropriate treatment? Under precisely defined conditions, a hospital exemption may allow access to an unapproved advanced therapy medicinal product. This can offer patients with rare or serious diseases a treatment option that would otherwise be unavailable to them.
The first hospital exemption in the Czech Republic was granted to the CREATIC Center of Excellence at the Faculty of Medicine for dendritic cell-based anticancer immunotherapy.
For patients with rare or serious diseases, there is often no effective standard treatment. In some cases, available treatment options have been exhausted; in others, they are not sufficiently effective, safe, or suitable for a particular patient. The development of drugs for rare diseases is complicated by the small number of patients and the considerable diversity of individual diagnoses.
Clinical trials are difficult to organise, drug development is financially and technologically demanding, and its economic return is often uncertain. As a result, many of these conditions are not a priority for large pharmaceutical companies. It is precisely in such situations that personalised medicine and the associated hospital exemption can play an important role.
what does a hospital exemption mean?
A hospital exemption is a specific regulatory tool designed for unregistered medicinal products for advanced therapy. Under precisely defined conditions, it allows for their non-routine manufacture and use for specific patients based on an individual medical recommendation. In the Czech Republic, the product must be manufactured in accordance with the principles of Good Manufacturing Practice (GMP), meet strict requirements for quality, safety, and control of the entire process, and must be administered in a healthcare facility.
Hospital exemptions are approved by the State Institute for Drug Control (SÚKL), which also oversees their implementation. Thanks to a clearly defined regulatory framework, it is possible to safely integrate research, manufacturing, and clinical care and shorten the path of innovative treatments to patients for whom time may be of the essence.
The CREATIC Center of Excellence at the Faculty of Medicine, Masaryk University, received the first hospital exemption for an advanced therapy medicinal product in the Czech Republic. It concerns an anti-tumor immunotherapy based on dendritic cells.
Dendritic cells are a natural component of the human immune system. They can be thought of as cellular “teachers” that capture tumor cells or parts of them and present them to T-lymphocytes, thereby triggering a targeted immune response against the tumor. Dendritic cell immunotherapy is based on this principle. First, monocytes—a type of white blood cell—are extracted from the patient’s blood, and dendritic cells are prepared from them under laboratory conditions. These cells are then exposed to the tumor’s specific characteristics so that they can effectively stimulate the patient’s immune system to fight the tumor cells. Interleukin 12 (IL-12), a signalling molecule that promotes the activation of T lymphocytes and other components of the immune response, plays an important role in this process.
After production is complete and a thorough quality control check has been performed, the medicinal product is administered to the patient—usually by injection—in doses according to the prescribed treatment plan. The goal is to help the immune system better recognise and respond to cancer cells. Because the medication is derived from the patient’s own cells, the risk that the immune system will recognise it as foreign is reduced.
Therapy for a specific patient
The production of modern therapy medicinal products is fundamentally changing the concept of what it means to “manufacture a drug.” This is not a standard product manufactured in large batches. Each dose is created individually from a specific patient’s cells and is intended solely for that patient. Living cells are an extremely sensitive material. Therefore, these medicinal products are produced in special cleanrooms, and every step must comply with the principles of Good Manufacturing Practice (GMP). This includes precisely defined work procedures, strict hygiene rules, controlled airflow, and continuous quality verification. These measures help maintain the required quality of the medicinal product and protect patient safety. In cell therapies, therefore, manufacturing is not merely a technical phase but an integral part of the entire treatment pathway. Research, manufacturing, quality control, and subsequent administration to the patient form a single, closely interconnected process.
At the CREATIC Center of Excellence at the Faculty of Medicine, Masaryk University, an infrastructure has been established that enables this journey to be realised—from scientific discovery through production in accordance with Good Manufacturing Practice (GMP) to the administration of the medicinal product to the patient in collaboration with a clinical facility.
Personalised therapy
The concept of a “hospital exception” is not based solely on legislation, production procedures, and regulatory processes. Behind it are specific patients and situations in which the options for standard treatment are gradually being exhausted.
One such story began with the diagnosis of a severe brain tumor in a boy who was only eighteen months old. Although he underwent chemotherapy and radiation therapy, the disease kept recurring. At the age of three, he therefore underwent radical surgery. Part of the subsequent treatment, tailored specifically to his condition, included the administration of a personalised medicinal product prepared from his own cells. In 2017, the boy was treated at the Department of Pediatric Oncology at the Faculty of Medicine, Masaryk University, and Brno University Hospital. The dendritic cell-based medicinal product was developed for him at our faculty—that is, in an environment whose scientific findings and accumulated experience now form the foundation of the CREATIC Center of Excellence at the Faculty of Medicine, Masaryk University. In this case, the medicinal product was administered in accordance with Section 8 of the Act on Medicinal Products under the responsibility of the attending physician. Therefore, this was not yet a treatment carried out under a hospital exemption.
The boy is now 12 years old and has been in remission—that is, showing no signs of active disease—for nine years. Dendritic cell immunotherapy was one component of his comprehensive treatment. His story illustrates why it is important to seek new treatment options even when standard procedures are insufficient. For this type of cellular immunotherapy, a hospital exemption now provides a clearly defined and strictly controlled framework. It allows for its use in clinical practice while maintaining requirements for safety, quality, and good manufacturing practices.
Personalised immunotherapy represents more than just technological progress. It also brings about a shift in the approach to treatment—from a one-size-fits-all solution toward a focus on the specific patient, the nature of their disease, and their individual needs. This approach is also linked to the Fair Medicine principle, which the CREATIC Center of Excellence at the Faculty of Medicine, Masaryk University, upholds. It emphasises the need to seek sustainable pathways to modern therapies even for small patient groups for whom the development of a conventionally registered drug may not be economically feasible due to the low number of patients. The hospital exemption helps bridge the gap between research, production, and the availability of treatment in clinical practice. Thanks to the professional and regulatory framework established by the State Institute for Drug Control (SÚKL), modern therapies can safely reach the patients who need them.
It is precisely for these patients that the CREATIC Center of Excellence at the Faculty of Medicine, Masaryk University, is developing a comprehensive pathway for modern therapeutic drugs—from scientific discovery through controlled production to safe administration. Because where one-size-fits-all solutions fall short, a personalised approach can offer a new treatment option.
