ACR Reacts to List of Initial Drugs Impacted by Medicare Drug Price Negotiation Program

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The following is a press release issued by the ACR on Tuesday, August 29, 2023.

The American College of Rheumatology (ACR) issued its initial reaction to the recent announcement from the Centers for Medicare & Medicaid Services (CMS) regarding which ten drugs will be subject to pricing negotiations, including Enbrel and Stelara, medications frequently used to treat rheumatic diseases.

As part of the implementation of the Inflation Reduction Act, Medicare can now negotiate the price of select medicines directly with pharmaceutical companies.

“It’s clear that lower costs are needed to improve rheumatology patients’ access to necessary drug therapies and treatments. High drug costs create an enormous financial burden for too many Americans living with rheumatic disease,” said Douglas White, MD, PhD, president of the ACR. “However, even with anticipated lower costs from negotiations, there is concern that patient access will remain limited if Congress fails to exempt Medicare Part B reimbursements from the sequestration reductions included in the Budget Control Act of 2011. On behalf of rheumatology providers, we are optimistic that these negotiations will yield savings for our patients. However, we ask that policymakers address the root causes of drug pricing increases throughout the drug supply chain to truly help patients afford their medications.”

ACR Introduces New Guidelines to Screen, Monitor and Treat Interstitial Lung Disease in Patients with Rheumatic Conditions

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The following is a press release issued by the ACR on August 22, 2023:

The American College of Rheumatology (ACR) released summaries of two new guidelines for the Screening and Monitoring of Interstitial Lung Disease in People with Systemic Autoimmune Rheumatic Disease and for the Treatment of Interstitial Lung Disease in People with Systemic Autoimmune Rheumatic Diseases (SARDs). The guideline summaries provide recommendations for screening, monitoring, and treating patients with rheumatic conditions like rheumatoid arthritis, systemic sclerosis (SSc), and idiopathic inflammatory myopathies (IIM) who may be at risk of interstitial lung disease (ILD) development or progression.

“Interstitial lung disease is a major cause of morbidity and mortality across several systemic autoimmune rheumatic diseases,” said Sindhu R. Johnson, MD, Ph.D., lead author on the guidelines and director of the University of Toronto’s clinical epidemiology & healthcare research program. “Guidance was needed for which tests to use for screening and monitoring this particular disease.”

A few recommendations from the guidelines include:

  • A conditional recommendation to screen patients at high risk of ILD with HRCT chest, PFTs, or both
  • A conditional recommendation to monitor ILD progression with PFTs and/or HRCT chest, albeit with different frequency.
  • A conditional recommendation to monitor with ambulatory desaturation testing.
  • A conditional recommendation against screening and monitoring with a 6-minute walk test distance (6MWD), chest radiography, and bronchoscopy.
  • For people with SARD-ILD other than SSc-ILD, we conditionally recommend glucocorticoids as a short-term, first-line ILD treatment.
  • A strong recommendation against glucocorticoids as a first-line ILD treatment for people with systemic sclerosis-associated ILD.
  • A conditional recommendation for use of mycophenolate, rituximab, cyclophosphamide, and azathioprine as first-line ILD treatment options.
  • A conditional recommendation against using leflunomide, methotrexate, TNF inhibitors, and abatacept as first-line ILD treatment options.
  • For people with SARD-ILD progression despite first ILD treatment, it is conditionally recommended to use mycophenolate, rituximab, cyclophosphamide, and nintedanib as treatment options.
  • For people with Sjogren’s-ILD and IIM-ILD progression despite first ILD treatment, a conditional recommendation against using tocilizumab as a treatment option.

“In those with systemic sclerosis-ILD, we strongly recommend against using glucocorticoids as a first-line ILD therapy or after ILD progression because glucocorticoids confer an increased risk of scleroderma renal crisis,” said Johnson. “Given the moderate certainty of the evidence for harm and low certainty of the evidence for benefit, we voted strongly against using this treatment for patients with systemic sclerosis-ILD.”

These new recommendations will provide guidance for clinicians on the screening, monitoring, and use of a wide range of treatments for patients with rheumatic diseases. The ACR developed these with the best available evidence and consensus across a range of expert opinions and incorporated patient values and preferences. These guidelines may reduce regional variation in patient care and may be used for patient advocacy.

“We know that early detection and hastened referral to care, in collaboration with pulmonology, is critical for the best patient outcomes,” said Sonye K. Danoff, MD, Ph.D., guideline author, pulmonologist and director of the Interstitial Lung Disease/ Pulmonary Fibrosis program at Johns Hopkins University School of Medicine. “Because symptoms of ILD (cough, shortness of breath, fatigue) can be subtle or result from other common diseases, the diagnosis of ILD can be delayed. Increasing awareness of the groups at highest risk for developing ILD and implementing appropriate screening and treatment practices should have long-term benefits.”

Full manuscripts have been submitted for journal peer review. Both are anticipated to be published in rheumatology journals by early 2024. View the complete summaries of the guideline recommendations at https://rheumatology.org/interstitial-lung-disease-guideline.

How is the immune system actually supposed to work?

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Our immune systems are amazing. Even when they go wrong, they’re still fascinating — at least to me.

For those of us who aren’t in healthcare or medicine, it’s not always easy to understand and separate out all the cells, the roles they play, etc. Even people who didn’t focus their studies on immunology can be stymied by it, too!

So, how does the immune system work? For the answer, take a look at this video from Kurzgesagt.

Kurzgesagt is one of my favorite YouTube channels, working to make it easier to understand big ideas from health and immunity to our climate crisis and beyond. It’s about making learning fun, regardless of the topic — and that’s absolutely my jam!!

If you want to learn more, check out their recent book — Immune: A Journey Into the Mysterious System That Keeps You Alive. It’s a gorgeous book, full of the same type of art you see in the video.

Note: the Bookshop link in this post is an affiliate link. If you purchase the book from Bookshop, I receive a small kickback at no additional cost to you. If you like that idea, you can check out more of my favorite books here.

Fibromyalgia Patients may benefit from Tocilizumab (Actemra)

Fibromyalgia Patients may benefit from Tocilizumab (Actemra)

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That what an article published August 10, 2023, on HCP Live says.

Now, it’s important to note that fibromyalgia isn’t well understood. That can make it hard to treat because we don’t know what treatments get to the heart of the disease. Actemra is an interleukin-6 inhibitor, often used for Still’s Disease and some other rheumatologic conditions. But, as the article says:

Investigators reported a case series of patients with fibromyalgia receiving tocilizumab treatment. Symptoms were assessed using the revised Fibromyalgia Impact Questionnaire (FIQ), which evaluated pain levels, and the 2016 criteria of the American College of Rheumatology (ACR). Symptoms were compared using the Wilcoxon signed-rank test and neutrophiles from patients with fibromyalgia and matched controls were isolated for transcriptome analysis. Each patient underwent a 40-joint sonography at baseline, including the elbows, wrists, knees, ankles, metacarpophalangeal joints, and thumb interphalangeal joints.

The FIQ and general fibromyalgia symptoms improved at both 4 and 12 weeks with 4 (67%) patients achieving a pain reduction of ≥30% at the 4-week mark and 3 (50%) achieving the same pain reduction at the 12-week mark. No significant changes in joint inflammation, as assessed by the 40-joint sonography, was observed.

There were possible differentially expressed genes identified in primary patients when compared with controls after treatment with tocilizumab. Additionally, 2 genes upregulated in patients with fibromyalgia (C3ARI and PI3) when compared with matched health controls.

The full journal article is available open access, too, in case you’re interested. In it, the authors point out:

We presented a total of two primary and four secondary FM patients who had received subcutaneous tocilizumab for a minimum of 12 weeks. All patients had severe symptoms despite standard treatments. Patients’ FIQR and fibromyalgianess both dropped at 4 and 12 weeks. Four (67%) of them reached a pain reduction of ≥30% at 4 weeks, and three (50%) reached a pain reduction of ≥30% at 12 weeks. Possible differentially expressed genes were identified in primary FM patients when compared with controls and after tocilizumab treatment. Conclusions: FM patients likely benefited from subcutaneous tocilizumab therapy. A randomized controlled trial is needed to verify its efficacy.

Obviously, 6 participants is a super low number. This is something that will require more research, for both those with primary or secondary fibromyalgia. It’s also going to be important to study younger people than 36, the youngest person in this study. Aside from that individual, the next youngest person was 43.

One incredibly interesting thing they noted:

Tocilizumab has been approved to treat cytokine storms in COVID-19 patients []. It could be interesting to investigate how FM symptoms change in patients treated with tocilizumab due to COVID-19.

I agree that this would be an interesting idea — especially because we know from another recent post that MAS is the king of cytokine storms.

Do you have fibromyalgia? If so, would you be willing to try a biologic to see if it helped your symptoms?

ACR Launches New Toolkit Aimed at Measuring RA Outcomes

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The following is a press release issued by the ACR today August 9, 2023:

The American College of Rheumatology (ACR) is excited to announce the launch of its brand-new Rheumatoid Arthritis (RA) Measures Toolkit. In recent years, the landscape of RA care has witnessed remarkable progress, greatly improving outcomes for patients living with this chronic condition. The key to this success: vigilant monitoring of disease activity and functional status, which has enabled rheumatologists to tailor therapies and optimize treatment outcomes. The new RA Measures Toolkit will help rheumatologists and their practices harness the power of standardized RA outcome measures.

“There are currently no national resources available for rheumatologists that guide the effective implementation, collection, and use of disease activity and functional status outcomes for people with RA,” said Jinoos Yazdany, MD, MPH, Professor of Medicine and Chief of Rheumatology at San Francisco General Hospital, University of California, San Francisco and one of the lead authors of the RA Measures Toolkit. “We wanted to develop a tool that allows our community to share best practices and innovations in collecting RA outcomes.”

The toolkit includes a wealth of resources like training guides for nurses and medical assistants who are administering RA outcome surveys (e.g., RAPID-3, PROMIS-PF or patient global assessments), copies of the ACR recommended RA outcome measures (including versions in Spanish and Chinese), sample workflows that rheumatologists are using in different electronic health record systems, and videos featuring best practices from rheumatologists with highly effective strategies for collecting RA measures.

The toolkit also includes guidance on utilizing the ACR’s RISE registry, a powerful tool for tracking performance on the collection of RA measures. With RISE, rheumatologists can effectively monitor and evaluate the implementation of RA measures in their practice and track performance in the Quality Payment Program (QPP).

The new toolkit is not just an asset when it comes to providing rheumatologists with standardized outcome measures for assessing disease activity and functional status for people with RA, it’s also helpful for training medical staff on collecting RA outcome measures.

“Collecting RA outcome measures accurately and efficiently requires a team-based approach that includes nursing staff. Proper training of staff ensures that surveys are administered accurately and consistently, leading to reliable data collection,” said Yazdany. “Moreover, training can help staff engage patients, emphasizing the importance of collecting this information. By providing training materials for staff, the toolkit saves time and allows rheumatologists to focus on taking care of patients.”

The authors of the toolkit interviewed dozens of rheumatologists and their staff to gather best practices and innovations for collecting RA outcomes.

“The information gathered in the toolkit allows our community to learn from both the successes and challenges encountered by our colleagues. The toolkit includes examples of successful clinic workflows, tips for efficient RA outcome measure collection, as well as interviews with high-performing practices,” said Dr. Yazdany.

The RA Measures toolkit e-book can be viewed in full online at https://ratoolkit.kotobee.com/#/.

Continuous Anakinra IV for a Patient With Macrophage Activation Syndrome

Continuous Anakinra IV for a Patient with MAS

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The following is from a research article published August 4, 2023.

Abstract

Macrophage activation syndrome (MAS) is a type of hemophagocytic lymphohistiocytosis (HLH), which occurs due to excessive stimulation of the immune system. Common precipitants of MAS include disseminated infection or underlying rheumatologic disorders such as adult-onset Still’s disease which is characterized as an inflammatory arthritis with daily fevers and a salmon-colored rash. We present a case of a patient with probable adult-onset Still’s disease and subsequent disseminated cytomegalovirus (CMV) infection, who met the criteria for MAS based on the presence of a fever, cytopenia in multiple cell lines, elevated ferritin, presence of hemophagocytosis on bone marrow, low fibrinogen, and mild splenomegaly on physical exam. The patient responded to treatment with continuous anakinra infusion and ganciclovir for treatment of CMV. Though cytotoxic medications such as etoposide have traditionally been considered first-line treatment for HLH/MAS, interleukin-1 inhibitors such as anakinra are emerging as a less cytotoxic alternative.

Key Quotes

MAS is often considered the most feared consequence of patients with AOSD and has been reported to occur in close to 15% of these patients. Treatment options for MAS currently include IV Igs, corticosteroids, etoposide, cyclosporine, tocilizumab, and anakinra. Though anakinra is generally administered via the subcutaneous route, the IV route may be preferred in critically ill patients due to increased absorption and a decreased risk of bleeding if an underlying coagulopathy is present.

Recent case series and reports have highlighted the role of IV anakinra infusion at doses of 1-2 mg/kg in the treatment of patients with macrophage activation syndrome.

What does this mean?

We have some new-ish options for treating MAS! Now, that isn’t new as in a new drug, but it is a new delivery method for Anakinra (Kineret) specifically.

This is important for a range of reasons, but especially considering that MAS — “the most severe form of cytokine storm” — has occurred in COVID-19 patients. Between happening during infection, we also know that COVID-19 causes major upset to the body’s systems, leading to the development of a host of conditions that may themselves cause MAS. And, in children, MIS-C may often appear — a condition somewhere between MAS and Kawasaki Syndrome.

In the rheumatology world, MAS is most common in AOSD and SJIA, happening in 10-15% and roughly 10% of patients respectively. It can occur in patients with other autoimmune and autoinflammatory arthritis types, most often Systemic Lupus Erythematosus (SLE). According to research, mortality in MAS cases is between 20-53%. It’s hard to know exact numbers when MAS may not be caught before a patient dies — if they even can access healthcare and try to do so.

This is also part of why it is imperative for AOSD, SJIA, and SLE patients to all know the early signs and symptoms of MAS — and to trust your gut if you feel something is really wrong.

Citation

Gullickson M, Nichols L, Scheibe M (August 04, 2023) A Novel Therapy for a Rare Condition: Continuous Anakinra Infusion for a Patient With Macrophage Activation Syndrome. Cureus 15(8): e42968. doi:10.7759/cureus.42968

ACR Significantly Concerned with MedPAC’s Latest Recommendations to Cut Part B Drug Reimbursement

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The following is a press release from ACR dated June 15, 2023:

The American College of Rheumatology (ACR) today expressed disappointment that the Medicare Payment Advisory Commission (MedPAC) has recommended yet another cut to physician reimbursement for infusing life-altering treatments as part of its June 2023 Report to the Congress: Medicare and the Health Care Delivery System.

In a letter to MedPAC, ACR took issue with the organization’s recommendation to Congress to keep a six percent add-on payment for the lowest-cost drugs, reduce the add-on payment for mid-to-high-level drugs, and add a payment cap for the costliest drugs.

“While we support efforts to rein in the cost of prescription drugs, we firmly believe that this policy would jeopardize provider practices and patients’ health by reducing access to life-changing provider-administered therapies,” said Douglas White, MD, PhD, president of the ACR. “We urge Congress to address the high cost of drugs at the root cause, like the opaque pharmacy benefit manager business practices, and not at the expense of providers.”

In its recommendation, MedPAC asserted that providers prescribe and administer the highest-price medications in order to receive higher reimbursement. ACR firmly rejects this premise. Administering Medicare Part B drugs in provider offices requires rheumatologists to buy the drug in bulk, maintain full-time staff to administer the treatment, and only bill Medicare after it has been given to the patient.

The six percent of the average sales price (ASP) add-on does not incentivize high-cost treatments, but rather offsets the costs of acquiring, storing, and administering treatments. As written, MedPAC’s recommendation would force providers to cut back on offering cutting-edge therapies or offer these medications at a loss, severely limiting patients’ access to medication and threatening practice viability.

“As providers, it is our job to prescribe the most appropriate treatment for our patients. The add-on payments are not considered in our clinical decision-making. We are concerned that policymakers are concentrating on these add-on payments rather than focusing on policies that address the true cause of drug prices,” said Christina Downey, MD, chair of ACR’s Government Affairs Committee. “The ACR will always express concern when provider payments to administer drugs are eschewed by widely supported policies. Hopefully, the bipartisan movement to reform the PBMs industry will yield meaningful change, and patients will see the benefits.”

EULAR: Systemic JIA, adult-onset Still’s disease ‘are the same disease’

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The following are excerpts of an article released by Healio Rheumatology on June 12, 2023.

Systemic juvenile idiopathic arthritis and adult-onset Still’s disease have been combined into one entity called Still’s disease in new recommendations document presented at the EULAR 2023 Congress.

“The first [overarching principle] was that systemic JIA and adult-onset Still’s disease are the same disease and should be called by the same name, and that is Still’s disease,” [Bruno] Fautrel said.

Read more.

disabled and pregnant? research opportunity

Are you disabled / chronically ill and pregnant?

Posted on by Grayson

The follow is a call for research participants to test a tool. I’m not involved, but it popped up on my radar, but I’m sharing here.

Brandeis University, the Cincinnati Children’s Hospital Medical Center, and others have worked together to develop a new tool – an Accessible Pregnancy Action Plan. This tool would help pregnant people with disabilities think about what they need during pregnancy, during birth, and after having their baby.

They are looking for people between 12 and 36 weeks along in their pregnancies who have disabilities / chronic illnesses and also use English or American Sign Language (ASL) to communicate.

Participants will work on their Action Plan with a peer facilitator, who is also a parent with a disability. These meetings will take place over Zoom. There will be between 2 and 4 meetings.

Most participants will complete the intervention in two sessions. They will be paid $50 per session. Participants who complete the program will also be compensated an additional $50.

Learn more about the study and take the screener here.

screener poster (has all the same information as in the Brandeis link)