White Paper
The FDA instituted the risk evaluation and mitigation (REMS) protocol in 2007 for certain medications with known or potential serious health risks. It exists to ensure that the benefits of certain groundbreaking medications exceed the risks. Today, healthcare providers and patients adhere to REMS protocols for drugs used to treat a variety of conditions, including many of the newest and most promising biologics.
For example, cell therapy and immunotherapy are at the forefront of treatment for many cancers. Treatments like CAR-T cell therapy are quickly becoming the standard of care for blood cancers like leukemia—and with incredible results. According to the American Society of Hematology, CAR-T therapy, which takes cells from a patient and genetically modifies them with an artificial cancer-targeting cell, has demonstrated remission rates of 76%.1
Yet these game-changing therapies aren’t without their complications, so many require REMS protocol. Cytokine release syndrome (CRS) is the most common acute toxicity following CAR-T therapy, occurring in between 37% and 93% of patients across different studies.2 The majority of patients only experience minor CRS side effects when detected early and fever is almost always the first sign. Flu-like symptoms can develop within the first week of treatment and resolve within eight days.
Thirty-three percent of patients experienced immune effector cell-associated neurotoxicity syndrome (ICANS)3 which can present more serious side effects, like confusion, language disturbance, motor weakness, seizures and headache. Worse yet, respiratory distress and multi-organ failure are possible.
What makes adverse events like CRS and ICANS particularly hard to treat is that care providers simply can’t predict who will be affected by these potentially dangerous conditions or how likely it is to be one of the severe cases—which can take a tremendous emotional toll on patients who are already fighting cancer.
This presents a serious challenge for biopharma companies: cell therapies represent an incredible advancement in the treatment of blood cancers, yet the potential for serious side effects can’t be overlooked. How can pharma companies leverage patient apps, connected devices and remote patient monitoring to decrease the risk of complications for patients on life-saving cell therapies and other treatments that require REMS protocol?
And above and beyond monitoring for serious adverse events, how can biopharma companies use these digital products to ease the burden of REMS protocol adherence for providers and patients?
60
Currently, the FDA requires REMS protocols on 60 different medications.
37% - 93%
Occurring in between 37% and 93% of patients, cytokine release syndrome (CRS) is the most common acute toxicity following CAR-T therapy.
1 https://www.hematology.org/newsroom/press-releases/2023/a-promising-outlook-car-t-cells-improve-patient-quality-of-life#:~:text=Overall%2C%2076%25%20of%20patients%20achieved,follow%20up%20for%20the%20study
2 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8671280/#:~:text=The%20CRS%20is%20the%20most,and%2093%25%20across%20different%20studies
3 https://www.hematology.org/newsroom/press-releases/2023/a-promising-outlook-car-t-cells-improve-patient-quality-of-life#:~:text=Overall%2C%2076%25%20of%20patients%20achieved,follow%20up%20for%20the%20study
For patients with a wide range of medical conditions, new and novel drugs that require REMS protocol are a lifeline. Yet the hurdles for both providers and patients can be significant–in two specific areas.
REMS protocols are designed to ensure the benefits of a “riskier” drug outweigh the health risks to the patient. Currently, the FDA requires REMS protocols on 60 different medications and what’s required depends on the individual drug. Common requirements include medication guides and patient package inserts, patient educational materials, special certifications for providers and pharmacies, detailed patient monitoring and patient enrollment into registries.
This puts a burden on both providers and patients. Patients may have to go through extended inpatient monitoring, periodic lab testing, regular doctor appointments and more, while also having to meticulously detail dosing and side effects. The monitoring and documentation burden on providers and care teams is also significant, with requirements varying drug by drug.
Side effects in patients taking REMS protocol drugs aren’t unusual. In fact, as treatments like cell therapy become ubiquitous, healthcare providers and hospitals are rarely taken by surprise when a patient develops CRS, ICANS or another complication. These side effects are expected in all patients. What’s not predictable is how severe it will be and for which patients. That means that every event has to be treated with the utmost urgency—an unsustainable and non-scalable dilemma that ultimately limits the quality of care patients receive and limits the quantity of therapy made accessible to patients.
Unfortunately, current monitoring processes, both inpatient and outpatient, are highly manual, inconsistent and largely dependent on caregivers and patients themselves. Within a clinic or hospital setting, monitoring relies on analog tools (e.g., blood pressure and temperature readings), which puts a heavy burden on nursing staff.
Once patients are discharged, the burden on the patient and their caregiver is immense—it requires consistent monitoring for early signs of adverse effects. This can create significant stress and emotional burdens for patients and caregivers. Additional appointments and lab work represent a financial and time cost as well.
Patients and caregivers on the challenges of monitoring for adverse events
There is a significant opportunity for biopharma companies to develop and launch digital companion solutions that simplify REMS adherence while also detecting adverse events earlier—before they become serious. Utilizing BrightInsight’s Disease Management Solution configured for REMS is the best way to meet protocol requirements, prevent adverse events and create value and differentiation in an increasingly crowded sector.
Both in a hospital or clinic setting and at home, remote monitoring capabilities are an invaluable resource for tracking early indications of adverse events.
If and when adverse events arise, companion apps can guide patients and caregivers through next steps, depending on severity of symptoms and possible complications.
Taking a drug that requires REMS monitoring can be complicated for patients who aren’t familiar with clinical information. Digital solutions can walk them through the process, boosting confidence and adherence.
A comprehensive patient app connects patients and caregivers to the support they need to find success in both onboarding to a REMS protocol and staying adherent during the life of the treatment.
The burden of REMS protocol drugs doesn’t solely fall on the patient. There’s a huge administrative and clinical burden for providers as well. An integrated HCP portal can reduce this burden by putting the most important patient data front and center.
The BrightInsight Disease Management Solution collects a wealth of real-world data, giving biopharma companies the insights they need to improve and enhance the drug lifecycle with the goal of removing REMS protocol requirements over time.
Learn how our Disease Management Solution can help you improve the patient and provider experience when dealing with REMS protocol drugs.
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