If you are making decisions around healthcare funding or policy, you will influence other individual’s access to treatment.
We have collated resources for professionals who do not work directly in the fields of gene and cell therapy development or delivery, but who need a grounded understanding of the subjects for their work.
Fundamental biological concepts in gene and cell therapy
As more conversations about gene and cell therapy are taking place in the public sphere, it is important to be clear in the terms used. The use of a term in a medical or legal context can differ from its colloquial understanding. This ambiguity can lead to miscommunications, resulting in a distrust of new technologies or an over-optimism about their potential.
Our 'Cell and Gene Fundamentals' section contains information about the biological concepts underpinning gene and cell therapy.
Methods and techniques in gene and cell therapy
Our 'Methods and Tools' sections contains information about the technologies and methods used in researching and developing gene and cell therapies.
The route to the clinic
The European Medicines Agency (EMA) have created a guide showing the timeline required to develop a new therapy and bring it to the clinic.
These resources provide further information about the medicines development process:
Discover and Development of Medicines - EUPATI Toolbox
Clinical trials
All new therapies must go through the clinical trials process to confirm their safety and efficacy. You can read about the different phases of the clinical trials process below.
Ongoing clinical trials
If you are interested in learning about ongoing trials for a particular condition, you may be able to find a national registry of clinical trials on either your national health provider’s website, or a government website.
National support organisations for specific conditions may curate a list of ongoing clinical trials in your region.
Be aware that being listed on a clinical trials registry does not mean that a trial is safe, ethical, or scientifically sound.
You can read about the regulation of clinical trials at a European level below.
Regulation of gene and cell therapies
After a therapy has been proven to be both clinically effective and safe, it must receive regulatory approval before it becomes clinically available. This is also referred to as market approval, market authorisation, or simply authorisation or approval.
In the EU, the regulatory body responsible for granting regulatory approval is the European Medicines Agency (EMA).
Regional approval and availability of therapies
When a therapy has been approved, it may be some time before it is available through regional health services. This is determined at a regional and national level, rather than at an EU level.
Several factors influence this timeline. Some of the most prominent are:
- Arranging a reimbursement scheme.
- Availability of suitable facilities, equipment, and trained staff. Facilities and equipment may need to be modified or upgraded in order to correctly deliver a new therapy. As well as this, staff will need to be trained in delivering the new therapy. A healthcare service may not be able to accommodate the facilities or provision of a new therapy in a particular region.
You can read an overview of the factors affecting availability of approved therapies below.
Reimbursement of medicines manufacturers
In order to make a therapy available through a national healthcare service, the government must come to an agreement with the manufacturer around reimbursement of their costs. You can find information about reimbursement of medicines below.
Accessing unauthorised or unproven therapies
There are several channels through which an individual might receive a therapy which is not yet authorised for widespread clinical use in their region.
Media coverage about new therapies may use the terms ‘unproven’, ‘experimental’, ‘investigational’, and others to mean the same thing. This confusion can help unscrupulous clinics offer therapies which are not grounded in scientific evidence.
Travelling for healthcare: European Reference Networks
If a therapy has been approved by the EMA, but is not available in all locations, patients in EU countries may be entitled to travel for care. This is facilitated through a European Reference Network (ERN). These are international networks which allow for the sharing of specialist healthcare knowledge. The decision to travel for care can be advised by the ERN, but ultimately must be made by the patient in consultation with their trusted team.
Travelling for healthcare: other travel
If an individual is unable to access a therapy within their own region, they may choose to travel for healthcare.
As listed above, this can mean:
- travelling to a region where the therapy is already approved by regulatory bodies
- travelling for an unregulated therapy, or one offered through a regulatory loophole
If clinics are receiving patients who are travelling out of their home country for care, it raises several issues in terms of duty of care to the patient, including:
- follow-up care
- additional care in the event of complications
- coordination with the patient’s healthcare team in their country of residence
- effect on future treatment options
These issues should be considered when developing policy around private clinics accepting non-resident patients. It is also important for policy-makers to consider how laws might be circumvented to offer unregulated therapies, and to address the safety issues this raises.
Patient involvement
Clinical research relies on patients’ willingness to contribute their time and data, at a minimum. Patient involvement relies on the idea that this research should be conducted with patients, recognising them as essential partners and experts in their own experience, rather than as passive research subjects.
If you are seeking patient expertise on a specific condition, we recommend that you contact a national patient support/advocacy group in your region for guidance.
Ethical and social issues around gene and cell therapies
The subject of gene and cell therapy can be a contentious one. Some people object on religious, ideological or personal grounds to the techniques used in research, to gene editing at an embryonic level, or to the idea of the therapies themselves.
You can find resources discussing some of the ethical questions raised by gene and cell therapiesin our 'Ethics and Society' section.
Even when a therapy has received regulatory approval, and been made available at a regional level, may still prove difficult for some patients to access. Many societies already experience inequality of healthcare, due to financial or social circumstances. A therapy provided free-of-charge through a national health service can still incur heavy costs related to travel, care, and time taken off work, to name a few.
Healthcare campaigners have expressed concern that, while advanced therapies can dramatically improve the lives of those receiving them, they carry the risk of all new technologies: that they will amplify existing systemic inequalities. As these therapies are relatively new, very little research has been completed on issues of healthcare equity in gene and cell therapy specifically. Local and national patient advocate groups may be best-positioned to comment on these issues as they related to specific conditions.
Specific information and expertise
If you are interested in finding about the use or investigation of gene and cell therapies to treat a particular condition, we recommend that you start with our condition-specific factsheets. We are constantly adding new topics to our factsheets.
You can find information about ongoing clinical trials in Europe on the EU Clinical Trials Register.
You may be able to find a national registry of clinical trials on either your national health provider’s website, or a government website.
Some national support organisations also curate lists of ongoing clinical trials of interest to the communities they serve.
We do not have the capacity to put you in direct contact with experts in the field for comment or interview. Our factsheets and Spotlight on Research pages may be a useful starting point for identifying scientific experts, and national support agencies may be able to recommend individuals with lived expertise.
We advise patients to consider the following questions when speaking to their healthcare provider, to ensure that they make a decision which aligns with their own healthcare priorities. They may offer a useful perspective on the individual decision-making process.