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CLINICAL NEWS
increased to the highest point in 2016 , mainly because of rising health-care costs . “ Working Americans are using the same or less health care and are paying more and more for it every year ,” said HCCI President Niall Brennan , MPP .
Spending per person in 2016 grew 4.6 percent from 2015 to $ 5,407 , while in 2015 , spending grew 4.1 percent from the previous year . Health-care spending per person grew cumulatively by 15 percent between 2012 and 2016 .
HCCI based its findings on claims data for 39 million U . S . patients under age 65 who were enrolled in employer-sponsored health insurance from 2012 to 2016 . The data represent about one-quarter of all people covered by employer-based insurance and was obtained by insurance companies , including Aetna , Humana , Kaiser Permanente , and UnitedHealthcare .

“Working Americans are using the same or less health care and are paying more and more for it every year .”

— NIALL BRENNAN , MPP , president of the Health Care Cost Institute
In 2016 , this increased spending was driven mostly by the cost of outpatient services , such as emergency room ( ER ) visits and surgeries , and prescription drug prices . Per-person spending on outpatient services reached $ 1,507 in 2016 , representing an increase of 6.2 percent from 2015 . Spending on prescription drugs totaled $ 1,030 , which was up 5.1 percent from the previous year ; however , this was less than the 10.4 percent growth seen in 2015 .
Spending on professional services , including physician visits , drug administration , anesthesia , radiology , and pathology , rose 3.5 percent to $ 1,821 .
Despite these increases , the use of most health-care services has held steady or declined overall from 2012 to 2016 . Mr . Brennan attributed this to the effects of the economic recession or the migration of workers toward high-deductible health plans , which Americans may find difficult to afford . In addition , the prices of health-care services have risen enough to offset any decreases in health-care use , the researchers found .
The study results also showed large
price increases for administered drugs ( up 42 % to $ 581 on average ) and outpatient ER visits ( up 31 % to $ 1,917 on average ) during the study period .
Source : Modern Healthcare , January 23 , 2018 .

Clinical Hold Placed on BPX-501 After Patients Develop Encephalopathy

The FDA put a clinical hold on the investigational cellular immunotherapy BPX-501 , halting four U . S . studies assessing the drug ’ s use for cancer and orphaninherited blood disorders . The agency placed the hold after three patients developed encephalopathy that was deemed possibly related to treatment . BPX-501 is an adjunct T-cell therapy administered after allogeneic hematopoietic cell transplantation ( alloHCT ) and is designed to eliminate alloreactive BPX-501 T cells in the event of uncontrollable GVHD .
The clinical trials affected by this hold include the following phase I / II studies :
• BP-001 and BP-005 : adults with hematologic cancers receive BPX-501 after alloHCT
• BP-003 : children with orphaninherited blood disorders receive BPX-501 after alloHCT
• BP-008 : adults and children with blood cancers are treated after relapsing post-transplant and evaluated for the potential for a titrated dose of rimiducid to resolve uncontrolled graft-versus-host disease ( GVHD ), while preserving BPX-501 cells
The clinical hold does not affect the European BP-004 trial assessing BPX-501 after alloHCT in children with hematologic cancers or orphan-inherited blood disorders .
More than 240 patients have been treated with BPX-501 after alloHCT . The drug ’ s manufacturer , Bellicum Pharmaceuticals , said it is waiting for formal FDA communication to determine requirements for resuming the studies .
Results from the BP-004 study , in which researchers evaluated immune recovery and outcomes of 112 pediatric patients who underwent a haploidentical HCT ( haplo-HCT ) followed by treatment with BPX-501 , were presented at 2017 ASH Annual Meeting . At 24 months after haplo-HCT , BPX-501 cells expanded progressively , peaking at nine months after the allograft . Among 16 patients with two years of follow-up , the mean number of BPX-501 cells was 62 + 23 / μL .
BPX-501 received orphan-drug designation in February 2016 .
Sources : FierceBiotech , January 31 , 2018 ; Merli P , Bertaina V , Galaverna F , et al . Donor T cells genetically modified with a novel suicide gene ( inducible Caspase 9 , iC9 ) expand and persist over time after post-allograft infusion in patients given αβ T-cell and B-cell depleted HLA-haploidentical allogeneic stem cell transplantation ( αβ haplo-HSCT ) contributing to accelerate immune recovery . Abstract # 211 . Presented at the 2017 American Society of Hematology Annual Meeting , December 9 , 2017 ; Atlanta , GA .

FDA Examining Patient Deaths Associated With Extracorporeal Photopheresis Treatment

The FDA published a letter to healthcare providers noting that the CELLEX ® Photopheresis System is under review following reports of venous thromboembolism ( VTE ), including pulmonary embolism ( PE ), in patients who received autologous immune cell therapy . Two deaths were observed , although “ the link between PE and death cannot be made with certainty ,” the agency cautioned .
The treatment system is an extracorporeal photopheresis ( ECP ) device approved for use in the ultraviolet-A irradiation of extracorporeally circulating leukocyte-enriched blood . It is used for the palliative treatment of the skin manifestations of cutaneous T-cell lymphoma that is unresponsive to other forms of treatment . It also is used to treat GVHD and acute cardiac allograft rejection .

“ The timing of the events in these reports suggests that ECP therapy may increase [ the ] risk [ of venous thromboembolism ].”

— U . S . FOOD AND DRUG ADMINISTRATION
Since 2012 , the FDA has received seven reports of PE during or soon after ECP treatment ( mean = 1.2 days ). Four of these events occurred in patients with GVHD , which includes the two patient deaths . The FDA also received two reports of deep
vein thrombosis ( DVT ) that occurred during or soon after an ECP session ; both occurred in patients with GVHD .
“ The timing of the events in these reports suggests that ECP therapy may increase that risk ,” the agency noted in the letter .
The FDA recommends that healthcare providers alert patients , clinical staff , and technicians about the signs and symptoms of PE and DVT ; refer to device labeling for anticoagulation use with this system and use clinical judgment in adjusting heparin doses ; and report VTE events related to ECP procedures through the FDA Safety Information and Adverse Event Reporting program , MedWatch .
Source : U . S . Food and Drug Administration news release , February 5 , 2018 .

Ferumoxytol Injection Receives Expanded Approval for Iron- Deficiency Anemia

The FDA approved a supplemental new drug application for ferumoxytol injection to include all eligible adults with iron-deficiency anemia ( IDA ) who cannot tolerate or have not responded to oral iron . The drug was previously only indicated for patients with IDA and chronic kidney disease .
The decision was based on the results of two phase III trials evaluating ferumoxytol injection versus iron sucrose or placebo in patients with IDA and a randomized , double-blind , phase III trial comparing ferumoxytol injection with ferric carboxymaltose injection in 2,000 adults with IDA . In the third study , ferumoxytol injection was comparable to ferric carboxymaltose injection based on the primary composite endpoint of the incidence of moderate-to-severe hypersensitivity reactions and moderate-to-severe hypotension .
Researchers also found that ferumoxytol injection improved hemoglobin per gram of iron from baseline to week five ( 1.35 vs . 1.1 g / dL ).
AE rates were similar across treatment groups , but the incidence of severe hypophosphatemia ( defined as blood phosphorous of < 0.6 mmol / L at week 2 ) was lower in the patients receiving ferumoxytol injection , compared with ferric carboxymaltose injection ( 0.4 % vs . 38.7 %; p value not provided ). The most common AEs associated with ferumoxytol were diarrhea , headache , nausea , dizziness , hypotension , constipation , and peripheral edema . ●
Source : AMAG Pharmaceuticals press release , February 5 , 2018 .
ASHClinicalNews . org ASH Clinical News
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CLINICAL NEWS increased to the highest point in 2016, mainly because of rising health-care costs. “Working Americans are using the same or less health care and are paying more and more for it every year,” said HCCI President Niall Brennan, MPP. Spending per person in 2016 grew 4.6 percent from 2015 to $5,407, while in 2015, spending grew 4.1 percent from the previous year. Health-care spending per person grew cumulatively by 15 percent between 2012 and 2016. HCCI based its findings on claims data for 39 million U.S. patients under age 65 who were enrolled in employer-sponsored health insurance from 2012 to 2016. The data represent about one-quarter of all people covered by employer-based insur- ance and was obtained by insurance com- panies, including Aetna, Humana, Kaiser Permanente, and UnitedHealthcare. “Working Ameri- cans are using the same or less health care and are paying more and more for it every year.” —NIALL BRENNAN, MPP, president of the Health Care Cost Institute In 2016, this increased spending was driven mostly by the cost of outpatient services, such as emergency room (ER) visits and surgeries, and prescription drug prices. Per-person spending on out- patient services reached $1,507 in 2016, representing an increase of 6.2 percent from 2015. Spending on prescription drugs totaled $1,030, which was up 5.1 percent from the previous year; how- ever, this was less than the 10.4 percent growth seen in 2015. Spending on professional services, in- cluding physician visits, drug administra- tion, anesthesia, radiology, and pathology, rose 3.5 percent to $1,821. Despite these increases, the use of most health-care services has held steady or declined overall from 2012 to 2016. Mr. Brennan attributed this to the effects of the economic recession or the migra- tion of workers toward high-deductible health plans, which Americans may find difficult to afford. In addition, the prices of health-care services have risen enough to offset any decreases in health-care use, the researchers found. The study results also showed large ASHClinicalNews.org price increases for administered drugs (up 42% to $581 on average) and outpatient ER visits (up 31% to $1,917 on average) during the study period. Source: Modern Healthcare, January 23, 2018. Clinical Hold Placed on BPX-501 After Patients Develop Encephalopathy The FDA put a clinical hold on the investigational cellular immunotherapy BPX-501, halting four U.S. studies assess- ing the drug’s use for cancer and orphan- inherited blood disorders. The agency placed the hold after three patients devel- oped encephalopathy that was deemed possibly related to treatment. BPX-501 is an adjunct T-cell therapy administered after allogeneic hematopoietic cell trans- plantation (alloHCT) and is designed to eliminate alloreactive BPX-501 T cells in the event of uncontrollable GVHD. The clinical trials affected by this hold include the following phase I/II studies: • BP-001 and BP-005: adults with hematologic cancers receive BPX-501 after alloHCT • BP-003: children with orphan- inherited blood disorders receive BPX-501 after alloHCT • BP-008: adults and children with blood cancers are treated after relaps- ing post-transplant and evaluated for the potential for a titrated dose of rimiducid to resolve uncontrolled graft-versus-host disease (GVHD), while preserving BPX-501 cells The clinical hold does not affect the European BP-004 trial assessing BPX-501 after alloHCT in children with hemato- logic cancers or orphan-inherited blood disorders. More than 240 patients have been treated with BPX-501 after alloHCT. The drug’s manufacturer, Bellicum Pharma- ceuticals, said it is waiting for formal FDA communication to determine require- ments for resuming the studies. 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