drug development

When planning began for the 2020 Annual Conference on Vaccinology Research sponsored by the National Foundation for Infectious Diseases (NFID), the novel coronavirus (SARS-CoV-2) that causes COVID-19 had yet to emerge. Since then, the world has changed dramatically. There have been more than 1.8 million cases and more than 100,000 deaths due to COVID-19 in the U.S. alone since December 2019, with nearly 1,000 new deaths reported each day. COVID-19 has devastated economies across the globe, and here in the U.S., more than 36 million individuals have lost their jobs. Lives have been disrupted, and cultures have had to adapt. Meetings, weddings, and graduation ceremonies are now held virtually...
Ella Murray is a happy, inquisitive elementary school student. However, because she was born without collagen factor VII, she suffers from a disease called epidermolysis bullosa (EB). This causes her to develop blisters all over her body, and she has to endure 3 hours of bandage changes multiple times a week. There are currently no therapies for EB, in part due to low prevalence of the disease and low enrollment in clinical trials. Joe Murray, Ella’s dad, told her story and called on researchers to inform communities about existing clinical trials during the Coalition for Clinical Trials Awareness panel discussion on child inclusion in clinical trials held in Washington, D.C. on May 1. This...
Partnerships between patient groups, industry, government and academia are accelerating the research and development of new treatments for rare diseases, many of which have no effective therapies. As a result of the Food and Drug Administration’s (FDA) Orphan Drug Designation, eligible companies receive incentives for product development such as tax credits for qualified clinical testing and new drugs are reaching patients sooner. Since 1983, more than 400 drugs and biologic products for rare diseases have been developed, far outpacing the number of products that were available some 40 years ago. Amyotrophic lateral sclerosis (ALS) is among the nearly 7,000 rare diseases where very few...
"There's not one fix" to ensuring patient access to innovative medicine. That appeared to be the consensus of moderator Christi Shaw, president, Novartis Pharmaceuticals Corporation and panelists, discussing the opportunities and challenges in getting new therapies to patients with complex and chronic conditions like cancer, multiple sclerosis and rheumatoid arthritis during a super session today at the BIO International Convention. Is the cost of some drugs a barrier? Are we adequately pricing the value of innovation? To ensure breakthroughs continue to reach patients, Scott Gottlieb, resident fellow, American Enterprise Institute, suggested that we need an approach to financing care that'...
Is the patient voice adequately represented in the drug development process? The situation has improved but the patient community is not yet recognized as a co-equal in drug development or regulatory review according to Marc Boutin, executive vice president and chief operating officer at the National Health Council , a panelist at a BIO International Convention session on Wednesday. ’€œWe scare you,’€ declares Boutin who says there is an adversarial connection between patients, regulators and industry but the relationship has evolved over the last decade. Patient advocates have developed strategies to amplify their voice and demand access, providing the data and evidence to help transform...