FDA approves leukemia treatment developed by Celgene, Agios

(Reuters) – The U.S. Food and Drug Administration (FDA) on Tuesday approved Celgene Corp and Agios Pharmaceuticals Inc’s oral treatment for acute myeloid leukemia (AML) patients with a rare genetic mutation.

The drug, Idhifa, will have a monthly list price of $24,872, Celgene said in an email, noting that the median time on therapy for patients was 4.3 months in the trial to secure the FDA’s approval.

The list price of a drug is not necessarily what patients actually pay. Their ‘out-of-pocket’ cost is based on their individual healthcare insurance plans and duration of treatment.

AML is a cancer that originates in the bone marrow and progresses rapidly, resulting in an abnormal increase in white blood cells. It is generally diagnosed in older people, and is uncommon before the age of 45.

The drug secured approval for relapsed or refractory AML patients with an IDH2 mutation. It is to be used along with a diagnostic test, developed by Abbott Laboratories, designed to detect the mutation.

“While the product isn’t expected to be a big needle mover for Celgene, it does represent the first approval for a partnered product,” J.P. Morgan analyst Cory Kasimov said, pointing out that the drug targeted “an area of clear high unmet need”.

Novartis AG’s recently approved AML drug, Rydapt, has a list price of $7,495 for a 14-day treatment duration and $14,990 for a 28-day duration.

However, Novartis’ treatment is approved for newly diagnosed patients with AML carrying a specific genetic mutation called FLT3.

Idhifa’s approval comes with a boxed warning — the strongest mandated by the FDA — designed to call attention to the risk of differentiation syndrome, an adverse reaction that could be fatal if untreated.

Differentiation syndrome is characterized by fever, respiratory distress and multi-organ dysfunction.

Idhifa is the first and only FDA-approved therapy for patients with an IDH2 mutation, a group that accounts for 8 to 19 percent of all AML patients. In the United States, that translates to about 1,200 to 1,500 patients, Celgene said.

Oppenheimer’s Leah Rush Cann said in a client note that the drug could generate sales of $1.4 billion in 2021.

About 21,380 new cases of AML will be diagnosed in 2017 and some 10,590 patients will succumb to the disease this year, according to estimates by the American Cancer Society.

Shares of Agios were up 5 percent at $58.77 in afternoon trading. Celgene was little changed at $135.09.

Reporting by Tamara Mathias and Natalie Grover in Bengaluru; Editing by Shounak Dasgupta and Maju Samuel

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AstraZeneca gets breakthrough status for blood cancer drug

LONDON (Reuters) – AstraZeneca said on Tuesday that U.S. regulators had awarded its blood cancer drug acalabrutinib “breakthrough” status for the treatment of patients with mantle cell lymphoma, a rare type of blood cancer.

The U.S. Food and Drug Administration decision clears the way for a speedy regulatory review and comes a day after another of its drugs, Imfinzi, won breakthrough designation for non-metastatic lung cancer.

Both developments represent pluses for AstraZeneca’s cancer portfolio following the initial failure of the key Mystic trial in lung cancer, which triggered the biggest ever daily fall in the company’s shares last week.

AstraZeneca acquired acalabrutinib after buying Acerta Pharma in 2015. The drug is being developed for a variety of cancer types.

Reporting by Ben Hirschler; editing by Jason Neely

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Where Do MOC Fees Actually Go?

Maintenance of Certification (MOC) is a process by which the American Board of Medical Specialties ensures that physicians are keeping up with developments in their fields. A research letter appearing in the Journal of the American Medical Association calls into question where all the exam fees being collected actually go. In this 150-second analysis, F. Perry Wilson, MD, follows the money.

“Maintenance of Certification,” the process by which the American Board of Medical Specialties supposedly ensures that physicians keep up with the times in terms of medical knowledge, has more drama surrounding it than the cast party for a high school musical. Speaking from experience here:

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Is MOC a form of rigorous self-regulation to be emulated across other professions, or a corporate boondoggle, swindling innocent docs out of their hard-earned?

Full disclosure: I am board-certified in Internal Medicine and Nephrology. Every ten years, I have to spend several thousand dollars to take an exam to maintain that certification. Where does all that money go?

After reading this research letter appearing in the Journal of the American Medical Association, I’m not really sure.

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Researchers Brian Drolet and Vickram Tandon did the legwork of pulling the fees associated with maintenance of certification for 24 member boards within the ABMS. Then they did something clever. They pulled the IRS Form 990 records for each board — these are nonprofit organizations after all – to determine just how much money they are making from all of our fees. One caveat – this is all 2013 data.

Overall, the 24 boards brought in $263 million in 2013, and spent $238 million. Examination fees were the bulk of the revenue at 87% but only 21% of the revenue was spent on administering exams, certifying, and verifying diplomates.

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The rest was spent on staff compensation. FYI, I looked at the 2015 form 990 for the American Board of Internal Medicine to see that the CEO had compensation totaling $850,000.

All right, I’m not an accountant. I’ll let smarter people than me debate the finances of non-profit entities like this. The important question to me is whether the whole process of specialty certification matters – does it improve patient care?

The data here is actually mixed.

I love this study, appearing in JAMA in 2014.

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Basically, the researchers took advantage of the fact that individuals who were certified in internal medicine in 1989 never had to recertify, but those who certified in 1991 were subject to the every 10-year recertification process. This is as close to a randomized trial as we’ll get. The bottom line? Maintenance of certification was not associated with a reduction in hospitalizations – you can see these lines basically overlap.

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But 1989 was a long time ago. Medicine moves faster now.

We also have good data to suggest that patients really like this stuff. 79% of patients in a 2003 Gallup poll said re-certification is very important.

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The medical specialty boards exist to facilitate public trust. But studies like this which raise questions about their financial situation erode the trust of the doctors they are trying to serve. We need some way to ensure that we, as professionals, are keeping up with the rapid evolution in medical science, but we’re going to need greater transparency from the ABMS if they want to continue to be our default mechanism.

F. Perry Wilson, MD, MSCE, is an assistant professor of medicine at the Yale School of Medicine. He is a MedPage Today reviewer, and in addition to his video analyses, he authors a blog, The Methods Man. You can follow @methodsmanmd on Twitter.

2017-08-01T12:00:00-0400

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Revised Gout Guideline Stresses Patient Education

A guideline for gout management originally published in 2007 has been revised and updated. Strongly emphasized in the latest version of the British Society for Rheumatology/British Health Professionals in Rheumatology guideline are patient education and providing information about gout and its treatment.

Guideline authors Michelle Hui and colleagues in the United Kingdom offered four reasons for why there is a clear need for an update:

  • New drug treatment options have expanded efficacy and safety profiles
  • Gout incidence, prevalence, and severity have increased
  • Fewer than half of patients with gout receive urate-lowering treatment
  • The barriers to patients with gout receiving adequate care can be overcome

Following are the highlights of the update, which was published in Rheumatology.

Acute Gout Attacks

  • Educate patients to treat gout attacks as soon as they occur and throughout the attack
  • Advise patients that joints affected by gout should be rested, elevated, and cooled; bed-cages and ice packs may be useful
  • Use a maximally dosed non-steroidal anti-inflammatory drug (NSAID) with colchicine dosed at 500 μg two to four times a day, the drugs of choice, in the absence of contraindications. Drug choice depends on patient preference, kidney function, and comorbidities. Prescribe gastroprotective agents for patients receiving NSAIDs
  • Aspirate and inject a joint with corticosteroids for acute single-joint gout. This may be the best treatment in patients with acute attacks and comorbidities. A possible alternative: a short course of oral corticosteroid or a single injection of an intramuscular steroid. Systemic steroid therapy is also appropriate for single or multiple joint attacks
  • Combine treatments for patients with acute gout where monotherapy has failed
  • Consider interleukin-1 inhibitors for patients who do not respond to standard treatment

Modifying Risk Factors and Lifestyle

  • Consider an alternative blood pressure-lowering agent when diuretics are used to treat hypertension and not heart failure, provided the agent controls the hypertension
  • For every patient with gout, give verbal and written information on the causes and consequences of gout and hyperuricemia; how to manage acute attacks; dietary recommendations (e.g., alcohol consumption and obesity management); rationale, use, and goals of urate-lowering therapy; and plans specific to the patient for comorbidities and concurrent medications. Discuss patients’ perceptions of illness and barriers to care
  • In overweight patients with gout, encourage dietary changes aimed at gradual reductions in body weight and continued maintenance. Discuss well-balanced low-fat, low-sugar diets high in vegetables and fiber with all patients
  • Encourage patients with gout and a history of kidney stones to consume more than two liters of water daily and consider using potassium citrate (60 mEq/d) for alkalinization of the urine when stones are recurrent
  • Screen for all cardiovascular risk factors and comorbidities (e.g., smoking, hypertension, diabetes, dyslipidemia, obesity, and kidney disease) with at least annual reviews and appropriate management

Optimal Use of Urate-lowering Therapies

  • Explain to patients the option of urate-lowering treatment upon gout diagnosis and involve patients completely in the decision as to when to begin urate-lowering treatment. Emphasize the importance of taking their urate-lowering medications regularly. Support patients when they have increases in gout flares during urate-lowering treatment
  • Discuss urate-lowering therapy and offer it to all patients with gout, especially those who have recurring attacks, tophi, chronic gouty arthritis, joint damage, renal impairment, kidney stones, diuretic use, or gout that started at a young age
  • Start urate-lowering therapy after inflammation has settled, when the patient is not in pain
  • Use urate-lowering therapy initially to lower and maintain the serum uric acid level at or below 300 umol/L to prevent further crystal formation and dissolve existing crystals. The lower the serum uric acid level, the quicker the crystals dissolve. After years of successful treatment, when tophi have gone and the patient is free of symptoms, adjust the dose of urate-lowering therapy to maintain a level at or below 360 umol/L
  • Start allopurinol, the recommended first-line drug for lowering uric acid levels, at a low dose (50 to 100 mg/d) and increase it in 100-mg increments every 4 weeks until uric acid levels have fallen to target. In patients with kidney impairment, use increments of 50 mg every 4 weeks when increasing the dose
  • Prescribe febuxostat as a second-line alternative when allopurinol is not tolerated or when kidney impairment is present. Start with a dose of 80 mg/d and, if needed, increase to 120 mg after 4 weeks
  • Use uricosuric agents in patients resistant to or intolerant of xanthine oxidase inhibitors. These drugs and doses are recommended: sulfinpyrazone, 200-800 mg/d; probenecid, 200-2000 mg/d, suitable for patients with normal or mildly impaired kidney function; benzbromarone, 50-200 mg/d, suitable for patients with mild to moderate kidney impairment
  • Do not use losartan and fenofibrate primarily as urate-lowering therapy unless they are already used in lowering blood pressure and lipids, respectively, because they have a weak uricosuric effect. Vitamin C supplements also have a weak uric acid-lowering effect
  • When the target urate level has not been achieved with monotherapy, combine a uricosuric agent with an xanthine oxidase inhibitor
  • Consider colchicine at 500 μg once or twice a day as prophylaxis against acute attacks resulting from starting or increasing any urate-lowering drug and continue for up to 6 months. If colchicine is not tolerated and there are no contraindications, use a low-dose NSAID or coxib with gastroprotection

Management in Special Groups

  • Reduce colchicine in patients with glomerular filtration rates of 10-50 mL/min/1.73 m2 and avoid it for patients who have more severe renal failure
  • Do not use high-dose NSAIDs if any level of kidney disease is present
  • Corticosteroids may be used in patients with chronic kidney disease but have not been examined in randomized trials
  • Use great caution when doing flare prophylaxis with colchicine or NSAIDs in patients with kidney insufficiency and gout; colchicine is preferred to nonsteroidals
  • Refer patients who have severe symptomatic tophaceous gout with uncontrolled hyperuricemia to a rheumatologist for consideration of the polyethylene glycol modified mammalian uricase treatment, pegloticase
  • Although gout is rare in pregnancy, conservative measures may be used (e.g., ice, nonsteroidals in the second trimester, steroids, and lifestyle modifications)
  • Avoid colchicine in pregnancy

This article originally appeared on our partner’s website Rheumatology Network, which is a part of UBM Medica. (Free registration is required.)

2017-08-01T10:30:00-0400

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Protecting yourself from pneumonia is easy

Did you know that 674,000 Americans go to the emergency room with pneumonia every year? Pneumonia is a lung infection caused by pneumococcal disease, which can also cause blood infections and meningitis. The bacteria that causes pneumococcal disease spreads by direct person-to-person contact. There’s a vaccine to help prevent pneumonia, but only 64% of adults 65 and over have ever gotten it.

Medicare can help protect you from pneumococcal infections. The pneumococcal shot is the best way to help prevent these infections. Medicare Part B covers the shot and a second shot one year after you got the first shot.

You may be at a higher risk for these infections if you:

  • Are 65 or older
  • Have a chronic illness (like asthma, diabetes, or lung, heart, liver, or kidney disease)
  • Have a condition that weakens your immune system (like HIV, AIDS, or cancer)
  • Live in a nursing home or other long-term care facility
  • Have cochlear implants or cerebrospinal fluid (CSF) leaks
  • Smoke tobacco

Learn more about Medicare-covered vaccines by watching our video. Preventing pneumonia is easy—get your pneumococcal shot today.

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