In this video, I teach you more about Multiple Sclerosis Clinical Research. Specifically, and the CAST of CHARACTERS who conduct an MS clinical trial! Want to up your game about Multiple Sclerosis Clinical Research? Then start watching right now!
Dr. Jay Avasalara, a neurologist at the University of Kentucky, has penned an editorial proposing changes to improve minority representation in clinical trials. CREDIT Mark Mahan for the University of Kentucky
In an editorial published in CNS Spectrums, Jay Avasarala, MD, PhD, takes the research community to task for its lack of minority representation in Phase III clinical trials for drugs to treat Multiple Sclerosis (MS).
Noting that the disease course of MS in African American (AA) patients is more aggressive, he urged researchers to make more effort to stave off the persistent slide in minority representation, which he believes skews efficacy and disability data and prevents physicians’ ability to extrapolate whether drugs are effective in these populations.
“The MS phenotype in the African American patient is an ideal model to study drug efficacy since the disease follows a rapidly disabling course,” he wrote. “AA MS patients admitted to US nursing homes are six years younger but more disabled compared to Caucasian American (CA) patients with MS. Since phenotypes between CA and AA can be clinically distinct, it is remarkable that not a single study has compared how drugs perform in such diverse groups.”
According to Avasarala, a neurologist specializing in Multiple Sclerosis and neuroimmunology at UK HealthCare’s Kentucky Neuroscience Institute, minority recruitment for clinical trials for MS drugs has declined from 7.7% in 2002 to about 2% in 2013.
In 2014, the FDA’s Center for Drug Evaluation and Research (CDER) launched the Drug Trials Snapshots initiative that endeavored to increase minority representation in drug trials and improve public transparency by providing drug trial data online. While Avasarala acknowledges that the initiative was a good first step, he emphasizes that it does not require drug package inserts to include efficacy data from minority populations, making it difficult for prescribing physicians to determine whether the drug will help patients from minority ethnicities.
In the editorial, Avasarala proposed several changes to help spur minority recruitment, which will in turn improve the quality of data for minority populations and make it easier for physicians to treat their AA patients.
“First, I believe we should require pharmaceutical companies to collect post-marketing data in all minority groups who receive FDA-approved drugs for management of MS and classify responsiveness based on ethnicity,” he said, noting that the FDA has required manufacturers to provide post-marketing data on drug safety for many years.
Avasarala also advocates for a requirement that package labelling include efficacy data from minority populations and that no publication should accept study data without a clarifying statement that acknowledges the lack of sufficient data to make reasonable conclusions in non-Caucasian minorities.
Since the 1970’s, science has documented that MS among African Americans is clinically distinct in its progression and presentation, according to Avasarala, but research into treatments that address their particular phenotype has not kept pace.
“The scientific community has published reams of data , but all that matters to a patient is, ‘OK, doc, how can you treat me?’ “What drugs would you recommend?” And we fall short for African Americans, because we simply don’t have the data.”
“I feel powerless to help them. There needs to be a change. And change ought to begin in the form of a policy shift.”
Avasarala states that his research at UK HealthCare will focus on the translational aspects of MS, datamining, the application of retinal imaging techniques to advance disease diagnosis, and studying in vitro blood-brain barrier models to facilitate drug transportation.
What they are and how they work
Clinical trials are carefully controlled and federally monitored research studies on drugs, procedures or other treatments that must pass rigorous testing before they are granted government approval.
Clinical trials and medical research
A clinical trial compares the effects of one treatment with another. It may involve patients, healthy people, or both.
[Original article on NHS Choices website]
You can ask your doctor or a patient organisation if they know of any clinical trials that you may be eligible to join.
You can also search for information on a number of websites and register your interest in taking part in research. The main ones are described below.
The UK Clinical Trials Gateway (UKCTG) website pulls through information about clinical trials and other research from several different UK registers. If you sign up to it, researchers can contact you about research that might be suitable for you.
You can also search the UKCTG site in various ways to find trials relevant to you, and you can contact researchers yourself.
The World Health Organization’s Clinical Trials Search Portal provides access to clinical trials in countries all around the world.
For some conditions, you can find out about clinical trials from the websites of charities.
Examples are:
Arthritis Research UK: current clinical trials and studies
Cancer Research UK: find a clinical trial
Multiple Sclerosis Society: MS clinical trials
Target Ovarian Cancer: clinical trials information centre
Clinical trials help doctors understand how to treat a particular disease or condition. 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.
However, there is also a chance that the new treatment turns out to be no better, or worse, than the standard treatment.
To hear other people’s experiences of taking part in a clinical trial, visit healthtalk.org: clinical trials.
Some clinical trials offer payment, which can vary from hundreds to thousands of pounds depending on what is involved and expected from you. Some trials don’t offer payment and just cover your travel expenses.
It’s important to find out about the inconvenience and risks involved before you sign up, and to carefully weigh up whether it is worth it.
Bear in mind:
it can be time consuming – you may be expected to attend a number of screening and follow-up sessions, and some trials require you to stay overnight
there may be restrictions on what you can and cannot do – for example, you may be asked to not eat or drink alcohol for a period of time
you may experience unknown side effects from the treatment
All clinical trials of new medicines go through a series of phases to test whether they are safe and whether they work.
The medicines will usually be tested against another treatment called a control. This will either be a dummy treatment (a placebo) or a standard treatment already in use. Learn more about the placebo effect.
Phase one trials:
A small number of people, who may be healthy volunteers, are given the medicine.
The drug is being trialled in human volunteers for the first time.
Researchers test for side effects and calculate what the right dose might be to use in treatment.
Researchers start with small doses and only increase the dose if the volunteers don’t experience any side effects, or if they only experience minor side effects.
Phase two trials:
The new medicine is tested on a larger group of people who are ill. This is to get a better idea of its effects in the short term.
Phase three trials:
Carried out on medicines that have passed phases one and two.
The medicine is tested in larger groups of people who are ill, and compared against an existing treatment or a placebo to see if it’s better in practice and if it has important side effects.
Trials often last a year or more and involve several thousand patients.
Phase four trials:
The safety, side effects and effectiveness of the medicine continue to be studied while it is being used in practice.
Not required for every medicine.
Only carried out on medicines that have passed all the previous stages and have been given marketing licences – a licence means the medicine can be made available on prescription.
If you take part in a clinical trial, you will usually be randomly assigned to either the:
treatment group – where you’ll be given the treatment being assessed, or
control group – where you’ll be given an existing standard treatment, or a placebo if no proven standard treatment exists
While the treatments are different in the two groups, researchers try to keep as many of the other conditions the same as possible.
For example, both groups should have people of a similar age, with a similar proportion of men and women, who are in similar overall health.
In most trials, a computer will be used to randomly decide which group each patient will be allocated to.
Many trials are set up so nobody knows who’s been allocated to receive which treatment. This is known as blinding, and it helps reduce the effects of bias when comparing the outcomes of the treatments.
When you express interest in a trial, a doctor or nurse is likely to tell you something about it in person. You’ll also be given some printed information to take away. You may come back with some questions you feel haven’t been answered.
What is the aim of the trial and how will it help people?
Who is funding the trial?
What treatment will I get if I do not take part in the trial?
How long is the trial expected to last, and how long will I have to take part?
How long will it be before the results of the trial are known?
What will happen if I stop the trial treatment or leave the trial before it ends?
What would happen if something went wrong? It’s rare for patients to be harmed by trial treatments, but you may want to ask about compensation if this were to happen.
How much of my time will be needed?
Will I need to take time off work?
Will I be paid?
Will the costs of my travel to take part in the trial be covered?
If the trial is testing a new drug, will I have to collect it from the hospital, will it be sent to me by post, or will I get it through my doctor?
Will I have to complete questionnaires or keep a diary?
What are the possible side effects of my treatment?
How could the treatments affect me physically and emotionally?
Who can I contact if I have a problem?
Will someone be available 24 hours a day?
How do I find out the results of the trial?
As with any treatment, you can’t be sure of the outcome. You may be given a new treatment that turns out not to be as effective as the standard treatment. Also, it’s possible you’ll experience unexpected side effects.
And bear in mind that you may have to visit your place of treatment more often, or have more tests, treatments or monitoring, than you would if you were receiving the standard treatment in usual care.
You may decide to stop taking part in a trial if your condition is getting worse or if you feel the treatment isn’t helping you. You can also choose to leave at any point without giving a reason and without it affecting the care you receive.
At the end of the trial, the researchers should publish the results and make them available to anyone who took part and wanted to know the results. If the researchers don’t offer you the results and you want to know, ask for them.
Some research funders, such as the National Institute for Health Research (NIHR), have websites where they publish the results of the research they have supported.
Before a clinical trial of a new medicine can begin, a government agency called the Medicines and Healthcare products Regulatory Agency (MHRA) needs to review and authorise it.
The MHRA inspects sites where trials take place to make sure they’re conducted in line with good clinical practice.
The Health Research Authority (HRA) works to protect and promote the interests of patients and the public in health research. It is responsible for research ethics committees up and down the country.
All medical research involving people in the UK, whether in the NHS or the private sector, first has to be approved by an independent research ethics committee. The committee protects the rights and interests of the people who will be in the trial.
Clinical trials can help:
prevent illnesses by testing a vaccine
detect or diagnose illnesses by testing a scan or blood test
treat illnesses by testing new or existing medicines
find out how best to provide psychological support
find out how people can control their symptoms or improve their quality of life – for example, by testing how a particular diet affects a condition
Many clinical trials are designed to show whether new medicines work as expected. These results are sent to the MHRA, which decides whether to allow the company making the medicine to market it for a particular use.
If research has identified a new medicine, the MHRA must license it before it can be marketed. Licensing shows a treatment has met certain standards of safety and effectiveness.
Safety must be monitored carefully over the first few years of a newly licensed treatment. This is because rare side effects that weren’t obvious in clinical trials may show up for the first time.
In England and Wales, the National Institute for Health and Care Excellence (NICE) decides whether the NHS should provide treatments.
The results of clinical trials are usually published in specialist medical journals and online libraries of evidence.
Some of the most well-known examples are:
You can use a search engine such as Google to look for articles and read summaries (abstracts). But you can’t usually see the full articles without a subscription to the journal.
Also, research papers are not written in plain English and often use many medical, scientific and statistical terms. They can be very difficult to understand.
You’ll often see stories about research findings in mainstream media. But while news stories are easier to read than original research papers, sometimes the findings are exaggerated or sensationalised.
The NHS website aims to make this clearer for you. Behind the Headlines is an independent service that analyses health stories that make the news.
It aims to explain the facts behind the headlines and give a better understanding of the research that was carried out.
