We appreciate the significant toll a neuromuscular condition can have on you and some people have a strong desire to participate in any research that can give people access to new treatments as soon as possible. However, it can be dangerous to roll out new treatments without regulators, pharmaceutical companies and the NHS to do their due diligence to ensure you benefit from the right, new treatment for you. Participating in a clinical trial is the key to helping others access such treatments safely.
It is essential that you are informed of the strict criteria that has been put in place to protect you should you choose to participate. It is important that you know exactly what is involved in any trial and any potential risks associated with participating, and that you only participate in trials that are officially registered to ensure that your health and safety are protected. Not every trial may be right for you and participating in trials that are not legitimate may put your health and safety at risk, as they may not have the same safety guidelines outlined below.
Guidelines for Official Clinical Studies
Informed Consent: To perform an official clinical study, medical researchers must outline a ‘protocol’ that clearly states their research questions and intentions. After reading and comprehending this protocol, patients will sign an ‘Informed Consent’ document to show that they have been provided sufficient information of exactly what will be involved, and the risks and benefits of participating. It is common to include other activities to measure a patient’s understanding before allowing them to consent to participate so that they are completely informed. It is important to note that signing an informed consent document is required to participate in the study to protect your health and safety, but it is NOT a contract and participants are allowed to drop out at any time.
Monitoring and Review: Official clinical studies must be reviewed, approved, and monitored by an Institutional Review Board of doctors, medical researchers, and other community members. The board will ensure that you are protected, the study is ethical, and risks are minimised. Some clinical studies may also be monitored by data monitoring committees, which monitor the safety and scientific integrity of the trial, and have the power to stop the trial if it is harming participants or is not effective. Should you choose to participate in a clinical trial, you should understand that the trial is constantly being monitored, and your health and safety is a priority.
Involvement of Healthcare Providers: Participating in a clinical study will not impede your usual care. Typically, patients continue to see their usual medical appointments. Your specialist neuromuscular consultant will make sure that participating in the study will not conflict with your current treatments and medications, which is why it is important to discuss your participation with them before, during, and after the trial.
Identifying Potential Participants: Official clinical trials will provide strict criteria on who can participate in the trial. The research team is often looking for a specific group of people and will ensure that only people who fit these characteristics participate. This is called the eligibility criteria and will be outlined in the protocol, along with the reasons for targeting this group of people.
Funding: A red flag that a study may be unofficial is if the team asks you to pay any amount to participate. Official clinical studies will not ask for any payment, and will often reimburse you for your participation and any extra costs that you endured.
We understand the eagerness to participate in clinical trials, but it is important that you are fully aware of these guidelines and why they are put in place. Official clinical studies must fulfil numerous requirements to ensure that participants are protected and that the trial can achieve its scientific purpose.
All official clinical trials have to be registered, and you can check what trials are currently going on here
If there is a trial you are interested in, speak to your neuromuscular consultant, who can advise you on whether you are suitable for the trial and help ensure that your health and safety will be protected. Please see below for a list of questions you may want to ask your consultant or the trial research team to see if a trial is right for you.
Questions to Ask When Considering Participating in a Clinical Trial
- Is the trial officially registered at https://clinicaltrials.gov/?
- What are the possible interventions that I might receive? How will they be determined (i.e. by chance)? Will I know which I am receiving?
- What exactly will I have to do? Are tests and procedures involved? How often will I have to visit the hospital/clinic?
- How do the risks, side effects, and benefits compare with those of my current treatment?
- How is the trial being monitored?
- Who will oversee my medical care while I am participating?
- After the trial: Will I receive the results? Will I be reimbursed? Is there any long-term follow-up care involved?