When Standard Treatment Options Are Not Enough: Personalised Therapy in Practice
Advanced and individualised therapies are particularly important where patient numbers are small, diseases are highly diverse and standard treatment options may be limited or exhausted. For the dendritic cell medicinal product for which CREATIC obtained a hospital exemption, this principle is closely linked to a specific clinical need. The experience behind today's programme began to develop many years before the hospital exemption was authorised.
Why Are Some Treatment Needs Difficult to Address Through the Standard Pathway?
For many patients with rare diseases, an effective standard treatment may not be available, or all available treatment options may already have been exhausted.
There are several interconnected reasons. Rare diseases affect small numbers of patients and can be highly complex and diverse. This makes treatment development substantially more expensive, makes clinical trials more difficult to conduct and reduces the economic return on investment. As a result, therapies for these diagnoses are often not a major focus for large pharmaceutical companies, which contributes directly to the limited number of authorised medicines.
This is where CREATIC’s personalised medicine approach comes in.
For many patients with rare diseases, an effective standard treatment may not be available, or all available treatment options may already have been exhausted.
There are several interconnected reasons. Rare diseases affect small numbers of patients and can be highly complex and diverse. This makes treatment development substantially more expensive, makes clinical trials more difficult to conduct and reduces the economic return on investment. As a result, therapies for these diagnoses are often not a major focus for large pharmaceutical companies, which contributes directly to the limited number of authorised medicines.
This is where CREATIC’s personalised medicine approach comes in.
From a Universal Medicine to Therapy Prepared from the Patient's Own Cells
Dendritic cell immunotherapy is not a universal medicine for all patients. It is a therapy prepared from the individual patient's own cells. Outside the body, these cells are specifically modified and activated so that, once returned to the body, they can help stimulate the immune system to recognise tumour cells. The autologous nature of the therapy also reduces the risk of an unwanted immune reaction against the administered cells.
The hospital exemption plays a key role here, allowing physicians to use this personalised therapy without standard marketing authorisation. This makes it possible to respond to an individual patient's clinical need and shorten the time between medicinal product development and treatment - an important factor in the context of rare diseases.
Personalised immunotherapy is not only a technological advance. It represents a shift from a universal treatment approach towards the individual patient and their needs. This is why the approach is aligned with the principles of Fair Medicine, focusing on patients who might otherwise not have the opportunity to receive a promising new treatment option.
Dendritic cell immunotherapy is not a universal medicine for all patients. It is a therapy prepared from the individual patient's own cells. Outside the body, these cells are specifically modified and activated so that, once returned to the body, they can help stimulate the immune system to recognise tumour cells. The autologous nature of the therapy also reduces the risk of an unwanted immune reaction against the administered cells.
The hospital exemption plays a key role here, allowing physicians to use this personalised therapy without standard marketing authorisation. This makes it possible to respond to an individual patient's clinical need and shorten the time between medicinal product development and treatment - an important factor in the context of rare diseases.
Personalised immunotherapy is not only a technological advance. It represents a shift from a universal treatment approach towards the individual patient and their needs. This is why the approach is aligned with the principles of Fair Medicine, focusing on patients who might otherwise not have the opportunity to receive a promising new treatment option.
The Story That Preceded Today's Hospital Exemption
The term 'hospital exemption' encompasses not only laws and regulatory processes, but also individual patients and situations in which standard treatment options are gradually exhausted.
One such story began when a serious brain tumour was diagnosed in a boy at just 18 months of age. Although he underwent chemotherapy and radiotherapy, the disease repeatedly returned.
At the age of three, he therefore underwent radical surgery. His subsequent individually designed treatment also included administration of a personalised medicinal product prepared from his own cells.
In 2017, the boy was treated at the Department of Paediatric Oncology, University Hospital Brno. The dendritic cell medicinal product was prepared for him at the Faculty of Medicine, Masaryk University - the scientific environment and experience on which the Centre of Excellence CREATIC builds today.
In this particular case, the medicinal product was administered under Section 8 of the Czech Act on Pharmaceuticals, under the responsibility of the treating physician. It was therefore not yet treatment under the hospital exemption framework.
Today, the boy is 12 years old and has remained in remission for nine years, meaning there are no signs of active disease. The dendritic cell immunotherapy for which CREATIC has now obtained a hospital exemption was one part of his complex treatment. His story illustrates the importance of looking for new options when standard approaches are no longer sufficient.
For this cell-based immunotherapy, the hospital exemption now provides a clearly defined and strictly controlled framework. It allows the therapy to be used in clinical practice while meeting requirements for quality, safety and manufacturing. In the Czech Republic, hospital exemptions are authorised by the State Institute for Drug Control (SÚKL), which also supervises their use.
Stories like this remind us why CREATIC is building the entire pathway for advanced therapy medicinal products - from scientific knowledge and GMP manufacturing to safe administration to patients.
An individual patient's story shows why searching for additional treatment options can be so important. At the same time, it reminds us that personalised therapy requires clearly defined rules, quality control, traceability and regulatory oversight. The next article focuses on these conditions and on the role of the Czech State Institute for Drug Control (SÚKL).
The term 'hospital exemption' encompasses not only laws and regulatory processes, but also individual patients and situations in which standard treatment options are gradually exhausted.
One such story began when a serious brain tumour was diagnosed in a boy at just 18 months of age. Although he underwent chemotherapy and radiotherapy, the disease repeatedly returned.
At the age of three, he therefore underwent radical surgery. His subsequent individually designed treatment also included administration of a personalised medicinal product prepared from his own cells.
In 2017, the boy was treated at the Department of Paediatric Oncology, University Hospital Brno. The dendritic cell medicinal product was prepared for him at the Faculty of Medicine, Masaryk University - the scientific environment and experience on which the Centre of Excellence CREATIC builds today.
In this particular case, the medicinal product was administered under Section 8 of the Czech Act on Pharmaceuticals, under the responsibility of the treating physician. It was therefore not yet treatment under the hospital exemption framework.
Today, the boy is 12 years old and has remained in remission for nine years, meaning there are no signs of active disease. The dendritic cell immunotherapy for which CREATIC has now obtained a hospital exemption was one part of his complex treatment. His story illustrates the importance of looking for new options when standard approaches are no longer sufficient.
For this cell-based immunotherapy, the hospital exemption now provides a clearly defined and strictly controlled framework. It allows the therapy to be used in clinical practice while meeting requirements for quality, safety and manufacturing. In the Czech Republic, hospital exemptions are authorised by the State Institute for Drug Control (SÚKL), which also supervises their use.
Stories like this remind us why CREATIC is building the entire pathway for advanced therapy medicinal products - from scientific knowledge and GMP manufacturing to safe administration to patients.
An individual patient's story shows why searching for additional treatment options can be so important. At the same time, it reminds us that personalised therapy requires clearly defined rules, quality control, traceability and regulatory oversight. The next article focuses on these conditions and on the role of the Czech State Institute for Drug Control (SÚKL).
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