CLINICAL NEWS
• clarify its review and approval of NGS-based tests for germline diseases
• include its regulation of other types of NGS-based tests ( e . g ., tumor genome sequencing ) that are valuable for the identification of structural genetic alterations
• address variant interpretation
• develop a complementary guidance for somatic diseases
“ ASH commends the FDA for developing recommendations that will foster the effective design , development , and validation of NGS-based tests for hematologic malignancies ,” wrote ASH President Charles S . Abrams , MD , in the recommendation letter . “ These recommendations would facilitate the improvement in overall quality control of testing laboratories , improve the tests ’ clinical benefits , and , more importantly , advance diagnosis and treatment outcomes .”
The recommendations were developed with input from various committee members and built on previous comments that ASH has submitted on this topic .
Source : American Society of Hematology press release , September 29 , 2016 .
CMS Releases Final Rule on MACRA
On October 14 , 2016 , the Centers for Medicare and Medicaid Services ( CMS ) released its final rule outlining the new payment system for Medicare clinicians , implementing the Merit-Based Incentive Payment System ( MIPS ) and Alternative Payment Model ( APM ) payment provisions of the Medicare Access and CHIP Reauthorization Act of 2015 ( MACRA ), collectively called the Quality Payment Program ( QPP ).
MACRA repeals the Medicare Sustainable Growth Rate methodology for updates to the Physician Fee Schedule and replaces it with the QPP . The ruling covers such issues as data reporting , new practice models , evolving clinical standards , and physician evaluations . According to the U . S . Department of Health & Human Services , the new payment system creates two pathways for clinicians :
1 . The first provides an opportunity to be paid more for better care and for investments that support patients , while reducing existing requirements . It also provides a more flexible performance period during the first year to allow physicians extra time to prepare .
2 . The second allows physicians to participate in organizations , such as accountable care organizations . When better health results and reduced costs are demonstrated , physicians receive a portion of the organization ’ s savings .
Key provisions of the final rule include :
• Clinicians are able to report as individuals or at the group level ; that same identifier should be used for all four performance categories ( quality , cost , clinical practice improvement activities , and advancing care information ).
• The number of measures physicians will report is reduced from nine to six .
• The reporting threshold was finalized at 50 percent for claims , clinical registry , electronic health records ( EHR ), and qualified clinical data registry mechanisms for the 2017 transition year , with graduation implementation of the higher thresholds in 2018 and beyond .
• The benchmark for resource use assessment is set on the measurement year as opposed to the prior year ’ s benchmark , and will include episode measures .
• The definition of Clinical Practice Improvement Activities is broader and more flexible . It includes any activity that a MIPS-eligible clinician , organization , or other stakeholder identifies as improving clinical practice or care delivery , and that the Secretary determines is likely to result in improved outcomes when effectively executed .
• CMS has moved away from the “ allor-nothing ” measurement standard used in the current Meaningful Use program to one that awards partial credit for certain components of EHR use and reduces the total number of required measures from eleven in the proposed rule to five in the final rule .
CMS will accept public comments on the final rule until December 19 , 2016 .
Sources : U . S . Department of Health & Human Services press release , October 14 , 2016 ; American Society of Hematology press release , October 14 , 2016 .
FDA Awards 21 Grants to Facilitate the Development of Orphan Drugs
The FDA has awarded 21 new clinical trial research grants over the next four years to encourage the development of drugs to treat rare diseases . These grants , totaling more than $ 23 million , were awarded to principal investigators of studies taking place at both national and international clinical sites .
In response to the Cancer Moonshot Initiative ’ s call to accelerate cancer research , 24 percent of the FDA grants were awarded to studies enrolling patients with cancer , and 43 percent went to studies enrolling pediatric patients , including newborns .
Grants related to hematology included :
• a $ 1.7 million , four-year grant awarded to Wolfgang Oster , MD , PhD , chief executive officer of the pharmaceutical company Oncoceutics , for a phase I / II study
FDA Approves HEA Test for Sickle Cell Trait
The FDA approved a new molecular assay ( PreciseType Human Erythrocyte Antigen [ HEA ] test ) to screen for sickle cell trait ( SCT ) in blood donors . The test was previously approved by the FDA for use in determining blood compatibility between donors and transfusion recipients . Screening for SCT helps to avoid the transfer of SCT-positive red blood cells to neonates or patients with sickle cell disease . Blood from SCT donors also can cause complications when performing the required filtration of white cells from the blood donation . The new HEA test allows these units to be identified prior to filtration .
Source : Immucor press release , October 26 , 2016 .
of ONC201 for the treatment of multiple myeloma
• a $ 99,630 , one-year grant awarded to Leslie Kean , MD , PhD , associate director of Ben Towne Center for Childhood Cancer Research at Seattle Children ’ s Hospital , for a phase II study of abatacept combined with calcineurin inhibition and methotrexate for prophylaxis of graft-versus-host disease
• a $ 500,000 , one-year grant awarded to Stanley Lewis , MD , MPH , chief medical officer of St . Hope Foundation , for a phase III study of ibalizumab for patients with multidrug resistant human immunodeficiency virus
Source : U . S . Food and Drug Administration news release , October 17 , 2016 .
FDA Grants Orphan Drug Designation for TLC178 for Cutaneous T-Cell Lymphoma
The FDA granted orphan drug designation to the investigational drug TLC178 , a liposomal-encapsulated formulation of the chemotherapy drug vinorelbine for patients with cutaneous T-cell lymphoma ( CTCL ). This formulation applies a nanotechnology platform to decrease the toxicity of the drug . A phase I / II open-label , doseescalation study of the safety , tolerability , and pharmacokinetics of intravenous TLC178 in patients with CTCL and other lymphomas is planned for sites in Taiwan and the United States .
Source : TLC press release , October 26 , 2016 .
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