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Gene and cell therapies: an introduction for policy-makers

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.

A note on language use

The language used in regulatory and clinicians may differ from those used in the context of patient support or advocacy. For example, you may see the term ‘orphan diseases’ used to refer to certain rare diseases in a regulatory context. (This relates to diseases potentially treatable by ‘orphan medicines’, which you can read about here). Many patients and patient advocates consider this term unhelpful in patient-facing material and other non-regulatory contexts. If you are in doubt about terminology, it may be valuable to consult with patient advocates.

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.

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.

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 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.

Be aware that being listed on a clinical trials registry does not mean that a trial is safe, ethical, or scientifically sound.

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.

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