Often we hear of new treatments becoming available, but have you ever wondered what each new treatment had to go through to get approved by a regulatory body like the FDA? Before a new treatment is approved for commercialization, it needs to go through a meticulous trial process to prove a number of things: Is the drug safe? What are the potential side effects? Does the drug do what it’s supposed to do? All of these questions and more need to be answered before a drug can be considered for approval by the FDA, so that’s where clinical trials come in. Here’s a breakdown of what’s involved in the drug development process, from preclinical through to commercialization and post-approval monitoring.
(Click to enlarge)
How can I participate in a clinical trial or find out more?
- You can learn more about research and clinical trials by joining or logging into PatientsLikeMe and clicking on the Research tab
- Use the PatientsLikeMe Clinical Trial Finder to search for trials that could be a good fit for you
- Check in with local associations and hospitals to see if they are recruiting for any trials
- Talk to your healthcare provider/clinician to see if there are opportunities they are aware of and how you can participate
- If you’re a member of PatientsLikeMe, make sure you consistently update your profile so we can let you know about research survey opportunities that are right for you
Interested in finding out more about how PatientsLikeMe members are impacting change in the clinical trial sphere? Check out these stories:
It’s Clinical Trials Day, and patients are driving change
“My expertise is as a person with Parkinson’s”: Member Lisa brings the patient voice to drug development
Bringing the patient voice to clinical trials
Share this post on Twitter and help spread the word.
- Filed Under: Conditions, Openness, Research
- Tags: clinical trials, clinical trials 101, drug development, multiple myeloma, patient interview, patient voice, phase I, phase II, phase III, phase IV, preclinical
With the recent FDA approval of Radicava (edaravone), we wanted to know more about how it works and what it means for patients living with ALS. We asked Maria Lowe, Pharm.D., BCPS, and our Health Data Integrity team, to give us a snapshot of the drug, how it’s used and what you should know. Maria’s rundown is meant to give you more context so you can have better conversations with your care team – as always, talk with your physician before starting any type of new treatment.
Radicava: The quick hits
- Radicava works as a free radical scavenger.
- When free radical (toxic by-products of cells that are highly unstable and reactive) levels are too high, cells are damaged resulting in oxidative stress (which may damage motor neurons). The way in which Radicava works to help patients with ALS is not fully understood yet; however, researchers believe that by getting rid of these free radicals it can help prevent some cell damage.
- In clinical trials, treatment with Radicava was found to slow the decline in functional disability as measured by ALSFRS-r scores for some patients. However, it is important to note that Radicava does not stop the death of motor neurons (it’s not a cure).
- Radicava may be taken in conjunction with riluzole (consult healthcare provider).
- Radicava is administered intravenously (60 mg IV over 60 min) daily for 14 days, followed by a 14-day drug-free period. Subsequent cycles are administered daily for 10 days over a 14-day period followed by 14-day drug-free periods. Because this drug is meant for long term use, patients will need to have a peripherally inserted central catheter (PICC line) or some other kind of catheter installed.
Clinical trials and FDA approval
- FDA approval for Radicava was based on a Phase III clinical trial conducted in Japan only in Japanese patients. Patients enrolled had to have ALS for less than two years, have normal respiratory function as well as the ability to complete most activities of daily living.
- Radicava may be processed and function differently in different patient populations. Post marketing data will be important for determining the effects and impact of the drug in patients other than those who participated in the clinical trials.
Side effects and reactions:
- The most common side effects reported in clinical trials include bruising, problems walking (gait disturbance), and headache.
- Hypersensitivity reactions: Radicava may cause allergic reactions that could happen after the infusion has finished.
- Symptoms to watch for: Hives, swelling, dizziness, wheezing, shortness of breath, itching, and fainting.
- If you’ve experience allergies to other medications before, inform your healthcare provider.
- Sulfite allergic reactions: Since Radicava includes sodium bisulfite as an ingredient, those with sulfite allergies should be cautious when taking this drug.
- Those with asthma have a higher risk of developing sulfite sensitivity. Let your healthcare provider know if you have asthma.
- Symptoms to watch for: Hives, trouble breathing/ swallowing, itching, swelling, dizziness, asthma attacks (in people with asthma), wheezing and fainting.
- If you have a sulfa allergy, you may or may not have a reaction to this drug. Sulfa allergies are a result of hypersensitivity to a chemical structure called sulfonamides, which are common in many antibiotics. Sulfites are chemically different from sulfonamides and any reaction to sulfites is not related to sensitivity to sulfa drugs. Both can cause reactions, but they are not related.
- If you are pregnant or plan on becoming pregnant and/or are breastfeeding, let your healthcare provider know. It is not known if Radicava will harm the baby or if it passes into breast milk.
Availability and access
- Radicava is expected to be available in August of this year as a brand name drug and it’s estimated it will cost around $145,000 per year in out of pocket costs (does not account for insurance coverage).
- Because the drug isn’t available yet, many insurance companies may not have developed policies regarding coverage. Different insurance programs will develop such policies of coverage of new drugs at different times.
- MT Pharma America (MTPA), the manufacturing company for Radicava, has developed a program called Searchlight Support to help people with ALS learn more about this treatment and to help them find and secure coverage through their insurance.
- Searchlight Support offers out of pocket support options only to certain eligible patients with commercial insurance. Patients with Medicaid, Medicare and other government funded insurance may not qualify.
Interested in learning more about whether Radicava is right for you?
Check out the medication guide and contact the Searchlight Support program to learn more about the potential financial support programs and to find an infusion center near you.
Share this post on Twitter and help spread the word.
Disclaimer: At PatientsLikeMe, we’re all about transparency, so please keep in mind that this piece is solely to provide a snapshot of Radicava so you can stay informed and make better decisions with your care team. This content isn’t sponsored and we don’t have a financial relationship with the drug manufacturer.