PUBLIC RELEASE DATE: 9-Sep-2014 Contact: Karen Kreeger karen.kreeger@uphs.upenn.edu 215-349-5658 University of Pennsylvania School of Medicine http://www.twitter.com/PennMedNews PHILADELPHIA - Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this month's issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments. "Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit," says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. "Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. We're proposing a different approach that spreads payments out and only keep coming if the patient continues to do well." Wilson and co-author Troyen A. Brennan, MD, JD, MPH, … Continue reading →

PUBLIC RELEASE DATE: 9-Sep-2014 Contact: Evy Vierstraete info@vib.be 32-924-46611 VIB (the Flanders Institute for Biotechnology) For the first time the Jeffrey Modell Foundation is giving a research grant to a Belgian laboratory. The team of Adrian Liston from VIB-KU Leuven will use the grant to develop a gene therapy to cure children that suffer from IPEX syndrome, a rare and fatal autoimmune disorder in which the immune system attacks the body's own tissues and organs. At the moment, the only successful therapy to treat the syndrome is a bone marrow transplantation, which is not available for all children. "This is a real chance for a cure", said group leader Adrian Liston. "The gene responsible for this disease was identified 13 years ago, but for the first time we may have learned enough about the basic biology to solve it. We should know within a year whether the gene therapy works in mice, after which we can move to patients at top speed." The Jeffrey Modell Foundation (JMF) JMF is a global non-profit organization for patients who suffer from Primary Immunodeficiency (PI) and their relatives. The organization is devoted to early and precise diagnosis, meaningful treatments and, ultimately, cures. Through clinical … Continue reading →