Rare diseases, cell and gene therapy
China has a rapidly expanding pipeline of cell and gene therapies. For some rare conditions, a patient may be able to access a regulated product or clinical trial that is not yet available in Europe or is difficult to access in practice.
Evidence status: Access to regulated or trial-based innovationWhat is the medical problem?
Rare genetic, haematological and immune diseases often have few treatment options. Availability of new therapies differs greatly between China, Europe, the United States and Japan.
What could a treatment pathway look like?
Diagnosis, genetic confirmation, previous treatment and organ function are assessed in advance. The search is then not for ‘a treatment in China’, but for one exact product or trial whose inclusion criteria match the patient. Access, costs, follow-up and risks must be confirmed in writing before travel is planned.
What do we know about effectiveness?
This category ranges from fully regulated medicines to early clinical trials. Chinese approval is relevant information, but does not automatically mean that the product is regarded as standard care in Europe. Evidence must be assessed product by product.
Important selection considerations
Cell and gene therapies can cause serious immune, liver, haematological and other complications. Only regulated centres with full complication management and long-term follow-up should be considered.
Medical selection before travel
The options described are not a guarantee of treatment. Availability and suitability depend on diagnosis, indication, evidence level, individual risks and assessment by the treating medical team. Experimental care is identified as such.
Would you like to know whether a pathway could be medically appropriate?
A treatment journey always begins with an assessment of the diagnosis, medical history, previous treatments, medication, risks and the specific treatment objective.