HHS Secretary Recommends Screening for Spinal Muscular Atrophy

On July 2, 2017, Health and Human Services (HHS) Secretary Alex Azar approved a recommendation for genetic screening of all newborns for spinal muscular atrophy (SMA). The approval follows a recommendation from the Advisory Committee on Heritable Disorders in Newborns and Children to add screening for SMA to the Recommended Uniform Screening Panel (RUSP). The RUSP is a list of disorders that HHS recommends states screen for as part of their state universal newborn screening programs.

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Topics: Spinal Muscular Atrophy

FDA’s Guidance for Genetic Testing: What are the implications?

In April of this year, the FDA finalized guidelines on two topics related to genetic testing: (1) how to address clinical validity through the use of databases; and (2) how to address analytical validity for next-generation sequencing (NGS)-based diagnosis of germline diseases. The guidelines make recommendations and outline processes that may increase the speed of innovation and create a more efficient path to market for genetic tests.

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Rising Obesity Rates in U.S. Children and Adolescents

Posted by The Evidence Blog on July 10, 2018

The percentage of children and adolescents affected by obesity has more than tripled since the 1970s, according to the CDC; nearly 1 in 5 American youth are obese. According to data from the National Health and Nutrition Examination Survey, around 18% of youth ages 2 to 19 years were obese and nearly 6% were severely obese in 2015-2016. Rates of obesity and severe obesity in adolescents (ages 12 to 19 years) are even higher.

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FDA Approves First Cannabis-Derived Drug for Treatment of Seizures

Posted by The Evidence Blog on July 5, 2018

The Food and Drug Administration (FDA) last week approved Epidiolex (cannabidiol; GW Pharmaceuticals) for the treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome in patients 2 years of age and older. This is the first FDA-approved drug that contains a purified drug substance derived from marijuana; it’s also the first FDA approval of a drug for the treatment of patients with Dravet syndrome.

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Topics: FDA approval, Epidiolex

FDA’s Efforts to Support Value-Based Healthcare

Posted by The Evidence Blog on June 27, 2018

Innovative medical treatments are being developed at an astonishing pace. However, rising medical costs are a roadblock to patient access to many of these innovations. Last month, Secretary of Health and Human Services Alex Azar rolled out a blueprint to address this challenge. The blueprint highlights the importance of linking payments for medical treatments to their value and removing regulatory obstacles to value-based purchasing.

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Topics: FDA, value based healthcare

FDA Approves Palynziq for Treatment of Phenylketonuria

Posted by The Evidence Blog on June 15, 2018

Recently, the FDA approved Palynziq (pegvaliase-pqpz; BioMarin Pharmaceutical Inc.), a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations on existing management. 

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FDA Expands Indications for Senhance Surgery System

Posted by The Evidence Blog on June 15, 2018

With the promise of shorter hospital stays, less blood loss, less pain, fewer complications from surgery, and a quicker return to normal activities for most patients, many hospitals and healthcare systems are embracing the use of robotic surgery systems. 

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FDA Approves New Drug to Treat Thrombocytopenia in Chronic Liver Disease

Posted by The Evidence Blog on June 14, 2018

The FDA has approved Doptelet (avatrombopag; AkaRx Inc.), a drug used for the treatment of thrombocytopenia in adult patients with chronic liver disease who have low blood platelets and are scheduled to undergo a medical or dental procedure. This is the first drug approved by the FDA for this use.  

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FDA Approves First-in-Class Medicine for Migraine Prevention

Posted by The Evidence Blog on June 14, 2018

The FDA has approved Aimovig (erenumab-aooe; Amgen and Novartis) for the preventive treatment of migraine in adults. Aimovig is an injectable calcitonin gene-related peptide (CGRP) antagonist.

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Adoptive Immunotherapy the Focus of New Hayes Report

Posted by Paula Goines, PhD, Medical Research Analyst on October 2, 2017

Chimeric antigen receptor (CAR) T cells and genetically engineered T-cell receptor (TCR) T cells have been topics of increasing interest as of late for both payers and providers alike. T cells are targeted, personalized therapies for:

  • solid tumors
  • hematological cancers
  • lymphoproliferative diseases
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