23 posts tagged “FDA”

Want to know more about Radicava (edaravone)? Here’s a snapshot:

Posted August 17th, 2017 by

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.

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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.


“I really felt that we were heard” — PatientsLikeMe staff member Dan shares his experience at the FDA psoriasis conference

Posted August 2nd, 2016 by

August is Psoriasis Awareness Month, and we’re kicking things off with a recap from the FDA’s public meeting on psoriasis back in March. The meeting was part of their Patient-Focused Drug Development Series that aims to bring the patient voice to research.

Sally Okun, Vice President for Advocacy, Policy and Patient Safety at PatientsLikeMe notes that, “The most effective part of the meeting was the patient stories. Even though a lot of quantitative data has been gathered by the FDA, they’re learning firsthand how difficult it is for these patients. Having an event that’s so patient-centric – where people are telling their stories and those attending can submit questions through the webinar – really lets patients make their voices be heard.”

The PatientsLikeMe psoriasis community also gave feedback through a survey in the weeks before the meeting, and this data was shared with the FDA. Check out the full report of what members had to say.

And Dan, one of our community moderators here at PatientsLikeMe, attended as both a member of our staff and a patient. We caught up with him to chat about his experience. Here’s what he had to say:

What was it like to attend this event as someone living with psoriasis?

I have had psoriasis for 23 years. I wasn’t alone, as my sister and brother had very mild symptoms, and my father and his brother had more moderate symptoms. So for me, having the disease itself wasn’t scary from a medical point of view. But I certainly suffered from social stigma as plaques cover all of my legs, arms and much of my torso.

As I learned from meeting my peers at the PFDD psoriasis conference, many psoriasis patients tend to hide their symptoms as best as they can by wearing clothes that conceal it, and by not participating in activities that would expose it to the public. I, too, was very challenged by the fact that my condition was so visible.

I also have ADD and an acquired learning disability (auditory delay due to an early childhood fever), on top of which I experienced a fair amount of trauma, which resulted in PTS.

I think this is probably why my symptoms were so bad since psoriasis is closely linked to stress and mental health. As a teen, I was grasping for coping mechanisms and esteem builders and I found solace and comfort in climbing, swimming, and hiking outdoors. When I started to experience my psoriatic symptoms, I couldn’t imagine giving up these activities. And so, I simply had to endure and explain my disease to everyone. I rarely, if ever, met anyone else who allowed their psoriasis to be as visible, and I felt extremely isolated. Not to mention my peers also had little or no exposure to people living with this condition aside from myself, thus increasing my issues with stigma.

Going to this meeting was amazing; I was surrounded by patients just like me for the very first time.

It felt incredibly rewarding! However, because I’d spent so much effort over the years suppressing my issues with stigma and the discomfort of psoriasis, it was challenging to process the emotions that came flooding back as I listened to the other patients’ experiences. I was almost brought to tears more than once. Not just for my peers and their suffering, but in recognizing a new awareness about my experience.

My peers were all gathered here to advocate and to express their hardships regarding psoriasis, and in the process they demonstrated incredible personal strength in dealing with this condition and speaking up about it. I was really impressed and inspired.

I also realized how strong I had become by managing the symptoms and stigma with little to no peer support for so many years. This in itself was also a very powerful experience; incredibly validating.

What were the most talked about issues at this meeting?

Much of the meeting revolved around two main issues: the pain and discomfort of psoriasis, and the stigma of having a visible and disfiguring illness. But also many interesting aspects of the patient experience were brought up. For example, nearly every African American present as a patient reported disparities in diagnosis. They all reported that their physicians were not aware that African Americans could even have psoriasis, leading to years of medications prescribed for infections they never had, side effects, and feeling that the medical system had completely failed them.

The prominent topic was around the levels of pain and itching that people experience with this disease, but then it migrated to include discussion of the cognitive and emotional toll of the illness. The level of patient suffering was palpable and very powerful, and sent a strong message to the FDA and the community attending.

Some people experienced constant burning feelings, like their skin was on fire. There were descriptions of feeling like their skin was encased in a cast of plaques, stiff, and uncomfortable, as well as sharp pangs of the open cracks and sores, which any movement or itching created. For many the pain was crippling.

There was also a lot of mention of the stigma experiences from patients recalling their experience as young children and adolescents, managing the emotional challenges of growing up combined with having a misunderstood and stigmatizing skin condition.

Psoriasis manifests in flakey white scales on top of fiercely inflamed red skin, which often cracks open and bleeds. Most people that we meet in our daily lives have no idea what psoriasis is, and are often afraid to even ask. This can lead to us being treated like we are infectious. In many ways it’s hard to blame them, the affected areas often look like Hollywood made-up zombies…

We also we leave bits of ourselves everywhere in the form trails or even piles of silvery flakes on chairs, under our desks, all over the house, and the cracked inflamed skin often leaves blood spots on any light colored clothing, bedding, and furniture.

I have always stepped past my shame and used the opportunity to apologize for the gross mess that I leave because it gave me an opportunity to educate people about what I have so hopefully they wouldn’t be scared of me, but many others in the group reported having a really difficult time talking about their illness with others.

Many coping mechanisms were described like avoiding dark clothes where the flakes would be very visible, or avoiding light clothes as they could highlight the blood stains. Many people also talked about always wearing long sleeves and pants, and going so far as closing the cuff’s of their shirts and pants with adhesive tape to prevent the flakes from spilling out everywhere in public.

Everyone also described this constant emotionally taxing vigilance of trying not to itch or scratch our affected skin as it makes more flakes, which is hard because of the overwhelming itchiness and burning pain. To compound this, many patients in the room had psoriatic lesions on their buttocks and genitals. Can you imagine being in work, or class, out shopping and being consumed with trying to manage the urge to stick your hands in your pants and just itch away? A tragic reality that results in shame, distraction, and, in the end, exhaustion.

Then there is the ever-present social stigma risk mitigation; always shaking out your collar, and sweeping flakes off one’s shoulders away so you don’t look like your are a walking trail of dandruffy, diseased skin.

Even when we are surrounded by educated and empathetic people we know or at least project that they are all affected by our appearance and the messes of “bio waste” we leave in our wake, there is this constant knowledge that we are less desirable as friends, lovers, and even just associates and co-workers; as one panelist reported that they felt as though people treated them as if the were a leper. It’s nearly impossible to disassociate our identity from our disease, because everyone sees our disease right in front of them.

There were a lot of reports of absenteeism from things like school, work, and even fun social events like going to the beach; anywhere public where the emotional effort of managing stigmatization 24/7 was simply too much to handle on a day-to-day basis. There were even questions brought up about whether we were more susceptible to STDs. The meeting facilitator at the FDA explained that they had never heard so many patients attribute such levels of emotional and cognitive fatigue, and brain fog/mental exhaustion to psoriasis. She seemed to be recognizing this as a new and disabling symptom. The FDA was listening!

In the end, we heard each other’s voices amplifying the realities to the FDA and the public that psoriasis clearly affects people on very deep levels; the obvious physical pain and itching, as well as the social stigma fears; the internal shaming and awkward external conversations we have with everyone we meet about our bodies and how our bodies corrupt everyone else’s living and work spaces.

I think the prevailing message was this isn’t a painful and uncomfortable skin issue, this illness touches every aspect our lives especially our mental health.

The irony is, like all autoimmune disease, psoriasis is exacerbated by stress, and having psoriasis causes a lot of stress. I don’t think the medical community has ever really quite understood the interplay between these symptoms and conditions.

Did you feel like the meeting was patient-centric and that your voice was being heard?

I really felt that we were heard. Everything in the program was designed to capture the patient’s voice.

They had this amazing system set up: the facilitator or the FDA staff would ask a panelist a question, and, as soon as they received their answer, they would turn to the audience and ask us the same questions in a multiple choice format. The community listening online would respond from their devices, and the patients in the audience all had received small wireless handsets allowing us to provide our experience. They would then project the results on large screens positioned around the room allowing us to analyze the answers in real time, often prompting the FDA staff to ask follow up questions so they could really gain an in depth understanding of the issue at hand.

You attended as both a PatientsLikeMe employee and a psoriasis patient. What was it like to engage in something like this with an organization like the FDA with that kind of dual perspective?

I think this was the most difficult part for me for two reasons. I wanted to make sure that I was an active participant as a psoriatic patient, and I also was trying to network with other patients and providers to let them know about PatientsLikeMe, and capture the experience so that I could relate it to other patients like me who may attend future events. It was certainly challenging to do all three at the same time.

Going to this event and participating as a patient meant so much to me, knowing that I’d be communicating my experience to these senior staff members of the FDA who will be guiding the future of psoriasis therapies. And hearing other people’s stories about their experience really helped me understand more about my own experience. I realized that I had never really fully processed the challenge of having a stigmatizing visible condition. I also realized that I had a strength that I had not recognized — although my psoriasis has been very challenging, I have managed to live a very full life and have been able to manage the emotional toll that psoriasis exerts. I also felt a little absolved: Has my depression and fatigue been partially fueled by my psoriasis? Have I been shaming myself for years for not overcoming these two issues, when they were not moral failings but part of a larger systemic health condition associated with my autoimmune disease?

On the whole, I’m very glad I went to the PFDD conference on psoriasis; it affected me profoundly and I think that the FDA really was listening and absorbing the experience of the psoriasis patient community. I was proud to be there and I was inspired by the strength of my peers. I really thank PatientsLikeMe for providing me with this rare once-in-a-lifetime opportunity.

 

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