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CLINICAL NEWS

The World Health Organization ( WHO ) published a new list of 113 diagnostic tests that it considers “ essential ” to every health-care system . Called the Essential Diagnostics List , this list represents the first time that the agency has labeled diagnostic services essential , with the intention of ensuring that all people have access to these tests .

“ An accurate diagnosis is the first step to getting effective treatment ,” WHO Director-General Tedros Adhanom Ghebreyesus , MD , noted . “ No one should suffer or die because of a lack of diagnostic services , or because the right tests were not available .”

The Essential Diagnostics List includes tests needed to diagnose common conditions worldwide , as well as many “ global priority diseases ” like HIV , tuberculosis , malaria , hepatitis B and C , human papillomavirus , and syphilis . Most tests on the list are in vitro ( e . g ., using blood and urine samples ) and can be administered in limited-resource areas .

The list of diagnostic tests is similar to the WHO ’ s Essential Medicines List , which was first published in 1977 , the agency explained in a press release announcing the list ’ s publication . “ The Essential Diagnostics List is intended to serve as a reference for countries to update or develop their own list of essential diagnostics ,” according to the WHO . “ To truly benefit patients , national governments will need to ensure appropriate and quality-assured supplies , training of health-care workers , and safe use .”

The WHO will update the Essential Diagnostics List regularly and pledged support to countries as they adapt the list to the local context .

Source : World Health Organization press release , May 15 , 2018 ; The New York Times , May 21 , 2018 .

The U . S . Food and Drug Administration ( FDA ) has approved avatrombopag for the treatment of thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure . This is the first drug approved for this indication .

The FDA ’ s decision was based on data from two randomized , double-blind , placebo-controlled trials ( ADAPT-1 and

ADAPT-2 ). A total of 430 patients were enrolled in the trials : 274 received avatrombopag and 156 received placebo daily for five days prior to a scheduled procedure .

The primary efficacy endpoint in each trial was the proportion of patients who did not require a platelet transfusion or any rescue procedure for bleeding after randomization and up to seven days following an elective procedure .

Among patients with the lowest baseline platelet counts , 66 percent and 69 percent of avatrombopag-treated patients responded in the ADAPT-1 and ADAPT-2 trials , respectively , compared with 23 percent and 35 percent of placebo-treated patients . Among those with the highest baseline platelet counts , 88 percent of avatrombopag-treated patients in both trials responded to treatment , compared with 38 percent and 33 percent of placebo-treated patients in ADAPT-1 and ADAPT-2 .

The most common adverse events ( AEs ; reported in ≥3 % of patients ) were pyrexia , abdominal pain , nausea , headache , fatigue , and peripheral edema .

Source : U . S . FDA news release , May 21 , 2018 .

Researchers have developed an artificial bone marrow niche , in which hematopoietic and progenitor cells are able to multiply and remain functional for several days , according to research published in the Proceedings of the National Academy of Sciences .

Scientists from the University of Basel , University Hospital Basel , and ETH Zurich were able to reproduce natural bone marrow in the laboratory by combining human mesenchymal stromal cells with a porous , bone-like 3-dimensional scaffold made of a ceramic material . They then used a perfusion bioreactor to combine the biologic and synthetic materials .

This process created a structure covered with a stromal extracellular matrix with embedded blood cells that mimicked the molecular structure of natural bone marrow niches .

The artificial tissue will help researchers to better understand the mechanisms of blood formation and to develop new therapies for hematologic malignancies and other conditions . “ We could use bone and bone marrow cells from patients to create an in vitro model of blood diseases such as leukemia , for example ,” study investigators Ivan Martin , PhD , and Timm Schroeder , PhD , explained in a press release . “ Importantly , we could do this in an environment that consists exclusively of

human cells and which incorporates conditions tailored to the specific individual .”

Sources : University of Basel press release , June 4 , 2018 ; Bourgine PE , Klein T , Paczulla AM , et al . In vitro biomimetic engineering of a human hematopoietic niche with functional properties . Proc Nat Acad Sci . 2018 June 4 . [ Epub ahead of print ]

The FDA has approved pegfilgrastim-jmdb , a biosimilar version of pegfilgrastim , which is indicated to decrease the duration of neutropenia in patients receiving chemotherapy .

A product is considered a biosimilar if it is similar but not identical to a biologic already approved by the FDA . The FDA ’ s approval of pegfilgrastim-jmdb is based on its review of structural and functional characterization , animal study data , human pharmacokinetic and pharmacodynamic data , clinical immunogenicity data , and other clinical safety and effectiveness data .

— SCOTT GOTTLIEB , MD

The agency also noted that pegfilgrastimjmdb is approved as a biosimilar , not as an interchangeable product .

The most common AEs associated with either pegfilgrastim or its biosimilar are bone pain and pain in the extremities . Other AEs include spleen rupture , acute respiratory distress syndrome , serious allergic reactions including anaphylaxis , glomerulonephritis , leukocytosis , capillary leak syndrome , tumor growth , and

fatal sickle-cell crises . In a press release announcing the approval , FDA Commissioner Scott Gottlieb , MD , reiterated the agency ’ s commitment to biosimilar approval and his desire for new policies to promote biosimilar development : “ We ’ ll continue to prioritize reviews of these products to help ensure that biosimilar medications are brought to the market efficiently and through a process that makes certain that these new medicines meet the FDA ’ s rigorous standard for approval .”

Sources : Reuters , June 4 , 2018 ; FDA news release , June 4 , 2018 ; Mylan press release , June 6 , 2018 .

The FDA has issued final guidance documents outlining the types of information that drug companies may share with insurers , including information not expressly described in the drug ’ s FDAapproved label . The policy is part of a broad shift toward value-based health care ; providing insurers with more access to economic and off-label information will allow insurers to better assess the true value of a medication , the agency argued .

Under the new rules , pharmaceutical companies will be permitted to share information about how the drug impacts a patient ’ s overall health-care spending , as well as whether there are additional unapproved uses or dosages for the drug . The document also states that drug companies will be obligated to provide “ a conspicuous and prominent statement describing any material differences between the health-care economic information and the labeling approved for the drug .”

The move expands a policy developed under the previous FDA administration to clarify the link between the value of a drug and its cost , allowing insurers to make more informed decisions about which treatments to cover and how much to charge for therapies .

FDA Commissioner Scott Gottlieb , MD , described the new guidelines as a step toward a more competitive and efficient health-care market . “ Already , the ability to harness this information and use it to help establish the value of medicines is providing the foundation for a shift toward innovative , value-based payment arrangements .” ●

Sources : STAT News , June 12 , 2018 ; FDA news release , June 12 , 2018 .