Expanded Access to Investigational Drugs – Not Just About Being “Compassionate”

Expanded access use of drugs in development (also commonly known as “compassionate use”) is an issue that has come to the forefront recently with some high-profile cases involving the news media and social media. Many may recall the news of a 7-year old boy who was suffering from a severe adenovirus infection and whose family fought hard for access to a drug still being studied in clinical trials, but which presented one of the only hopes for successful treatment. After the manufacturer of the experimental drug denied the request for access to the drug by the boy’s family, a major social media campaign ensued which mounted significant public pressure on the drug company and resulted in a reversal of the decision. The drug company worked with the US Food and Drug Administration (FDA) to grant access to the drug for the boy and other patients like him.
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Automation Helps Pharmacies Meet Compliance Standards

The passage last year of federal legislation to regulate pharmacy compounding was not only necessary but long overdue. Compounding pharmacies had historically been regulated at the state level, even though some produced drugs that were being sold in other states. Meanwhile, the public remained largely unaware of the compounding industry, confident that their health care providers would never inject them with anything that was not safe.
All that changed in the fall of 2012 when people began falling ill – and dying – from steroid injections produced by the New England Compounding Center (NECC) in Farmingham, Massachusetts. The NECC tragedy not only reaffirmed the risks inherent in manual compounding, but the scale at which the pharmacy operated. The tainted injections it produced were linked to more than 750 cases of illness and 64 deaths across 20 U.S. states.
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Investigations and Audits: Developing an Effective Game Plan

Monday Morning Quarterbacking

On Monday mornings during pro football season in offices around the country, win or lose, there’s the inevitable water cooler discussion about the team’s performance and what they should have done during the game in critical situations. The discussion, or post game review, is generally referred to as “Monday morning quarterbacking” and is analogous to the kind of after the fact analysis that occurs in a highly regulated industry.
In the business environment, this kind of hindsight, and post event regulatory scrutiny, heightens the need for effective governance, risk and compliance (GRC) programs as “getting it wrong” often comes with penalties, fines and sanctions. Everything companies do on the global playing field is viewed microscopically by regulators in the unique position to assess day to day operational performance after the fact.
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Integrating RWE into Investigator Initiated Trials

Expanding government and payer requirements for additional data on products, whether for safety monitoring, efficacy confirmation or economic and clinical value evaluation, underscore the importance of an integrated strategy for investigator initiated trials (IIT) and real world evidence (RWE) research. In developing an IIT/RWE strategy, companies need to be clear up front about why specific research should be supported, and use this criteria to decide which studies have the potential to be valuable. Determining how and where to use IITs and RWE requires clear objectives for the research and coordinated organizational capabilities.
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Preparing for Crisis Communications in a Hyper-Connected World

When you work in healthcare, you are trained to expect a crisis whether it is a failed clinical trial, a life-threatening side effect for a marketed drug, or a decision by a government agency to require additional data before granting approval.  Professionals must understand how to prepare for these inevitable regulatory crises and the best ways to manage them.
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Change is in the Wind for Biopharma Deal Making

Through 2013 and the first half of 2014, the biopharma industry experienced a breakout year. The NASDAQ Biotechnology Index ended 2013 at a record high, with US investors embracing biopharma IPOs and follow-on financings at record levels. The total amount of capital allocated within M&A/licensing increased, and the bull market in biopharma continued its surge in the first half of 2014.
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