Access to medicines: What you need to know

Access to medicines

Medicines can mean a lot of things to people living with inherited metabolic disorders (IMDs), this may range from foods used in a specialised diet, to medication used to manage a condition, all the way to gene therapies that may alleviate the symptoms of IMDs.

In this section of our Research Ready Hub, we’ll introduce you to clinical trials, give you a step-by-step to how to take part and will give you the tools to know more about access to medicines as part of our education programme.

Clinical trials: In a nutshell

What is clinical research?
The term clinical research relates to projects which aim to find out more about an illness, condition, treatment, therapy, or care by directly involving people.

What are the benefits of taking part in research?
Clinical trials help doctors understand how to treat a particular illness. It may benefit you, or others like you, in the future. If you take part in a clinical trial, you may be one of the first people to benefit from a new treatment.

Does it cost me anything? Will I be paid? 
Some clinical trials offer payment, which can vary depending on what’s involved and expected from you. Some trials do not offer payment and just cover your travel expenses.

Wondering how to get involved? Here's the step-by-step process to taking part in clinical trials

Step One: Discussion
You can suggest taking part in a clinical research study or trial to your GP or consultant or they may suggest it to you.

Step Two: Find a study
Your clinician will look for studies and trials you might be suitable for. You can also search yourself on the Be Part of Research Website.

Step Three: Referral
If your clinician identifies a suitable study or trial they will refer you to the organisation running it and send your medical records. You cannot take part without being referred and you also cannot contact the study delivery team directly yourself at this stage.

Step Four: Screening
The study delivery team will look at your medical notes to see if you are suitable. They may ask your clinician to run some more tests with you.

Step Five: Enrolment
The study delivery team will get in touch with you. They’ll explain the aims, treatment being trialled and what to expect in terms of side effects and hospital visits. You and your clinician will be asked to sign a consent form giving permission to share your medical information for the study or trial.

Step Six: Taking part
Clinical research studies and trials can run for anything from a few months to several years. During that time you may need to go into hospital for a few hours every few weeks to receive the treatment, or you may be able to take it at home. You may be monitored for any effects and asked to fill in questionnaires or answer questions. This may also take place in hospital.

Step Seven: Findings
After the study or trial is finished its findings will be written up as a paper and published in an academic journal.

Hear from the experts

We’ve spoken to Professor Paul Gissen and Naomi Litchfield to give you a better insight into what to expect from clinical trials.

Click the videos below to find out more.

Want to learn more? Take part in our Access to Medicines Education Programme

We realise that the terminology used in studies and trials can sometimes be overwhelming.

Other times, you might just want to know how something works. To empower you with the relevant information, we have developed an education programme. 

Watch this space! More information will be released shortly. 

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