Everything You Would Ever Want to Know about the New FDA Gene Therapy Guidances on Bastille Day Eve.

by

FDA issues new gene therapy guidances FDA’s Center for Biologics Evaluation and Research (CBER) issued a suite of six scientific guidance documents intended to serve as a framework for gene therapies. The agency hopes the guidances will “serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness.” The documents are being issued in draft form so that the FDA can solicit public input on the new policies. The first three guidance documents focus on gene therapy Read More

CMC and Regulatory Affairs See the forest and the trees

by

The Forest: Broadly written guidelines are subject to interpretation and raise so many half-truths and misconceptions that it is difficult to know with any certainty what constitutes a recommendation, as opposed to what is an actual requirement at any given phase of clinical development. FDA regulations in 21 CFR Section 312.23(a)(7)(i) states, “that an IND for each phase of investigation include sufficient CMC information to ensure the proper identity, strength or potency, quality, and purity of the drug substance and drug product” and goes on to say, “The type of information submitted will depend on the phase of the investigation, Read More

Was Your Rooster on Fire in 2017?

by

The Chinese Zodiac declared 2017 to be the year of the Rooster (the famous fire rooster, to be exact) promising Trustworthiness, with a strong sense of Timekeeping and Responsibility at work!  Looking back, did the Rooster come through for you? I sincerely hope so. He was definitely crowing, almost constantly, for DSI… hardworking, resourceful, courageous, confident, talkative, frank, open, honest, and loyal partnering. Trustworthy 100% IND, NDA, BLA and DMF approvals for our clients Engaged in several new Breakthrough Designation Status programs Welcomed 11 new warriors to our Staff, making us an EVEN DEEPER department! The LEADER in Regulatory Drug Read More

Meetings Between the FDA and Sponsors Aren’t Quite What You Think They Are?

by

FDA Meetings aren’t what many people think they are especially when it comes to the CMC focused discussions. Beliefs surrounding one’s CMC compliance strategy run the gamut from the pessimistic “What if the FDA wants much more to be done than is currently scheduled?” to the euphoric “Our manufacturing process for preparing and controlling clinical product for earlier phases is also considered good enough to go forward into later-stage clinical trials and even commercial manufacturing.”  Let’s look at seven common opinions about formal meetings between the FDA and Sponsors or Applicants of PDUFA Products when it comes to CMC compliance. Read More

Eclipses, Hurricanes and Wildfires?

by

The Path of Totality 2017 began with no clear picture of who would be assuming leadership of the Agency. In early May, Dr. Scott Gottlieb was sworn in as the 23rd Commissioner of FDA and he moved quickly to shift policy priorities in almost every area that the Agency regulates, a goal that in some ways was made more efficient due to the concurrent timing of modernization mandates. Increase in Generics It will come as no surprise to our regular readers here – or to anyone who reads anything for that matter – that high drug prices are a “thing” Read More

7 Reasons to Hire Regulatory Consultants

by

“Why should I hire a consulting firm? I can just do it myself!” This statement is often made without a complete understanding of the real value provided by Regulatory Drug Development Consultants (RDDC). An effective consulting firm is there to supplement your business proceedings, never to imply that they know better than you do. In fact, there are a number of misconceptions that exist so let’s re-evaluate some of the often forgotten facts relating to effective regulatory drug development consultants. RDDCs have deep industry expertise. Consultants have the rare privilege of working with a plethora of different companies on a Read More

Bill Buckner’s Encore: When a Miscalculation Decides the Outcome

by

Bill Buckner Revisited: When an Error Decides the Game (When a Decision Decides the Outcome) Beginning with spring training through the dog days of summer to the stretch drive, after eight months of baseball, the Astros and Dodgers are putting their mark on the 2017 World Series. One lesson of the postseason however may be miscalculation in the field. The ball that trickled through Bill Buckner’s legs in Game 6 of the 1986 World Series between the Boston Red Sox and New York Mets felt as if it would carry a never-ending stigma, well at least until 2004, when the Read More

12 Reasons You Should Invest in Biotech Consulting

by

Effective usage of biotech consulting allows you to maintain your focus on the pharmaceutical you’re creating. Biotechnology firms are cropping up all over the place, and growing at an unbelievable rate. California alone is home to over 3,000 life sciences companies, employing over 287,000 people[1] and the global industry is expected to be worth $727.1 billion by 2025.[2] One way to ensure you stay ahead of the competition is to truly understand the value provided by biotech consulting. Effective usage of biotech consulting allows you to maintain your focus on the pharmaceutical you’re creating while consultants provide specific and complementary Read More

Breakthrough Therapy Designation Tips: Invest early in impurity control and comparability

by

Drug development has always walked a bit of a tightrope between the need to get safe, efficacious and profitable new products approved and doing so in a way that is both timely and cost effective. When it comes to decisions about investing resources in the drug development process, timing is just as important to ultimate success as it is in investments in the stock market. Invest when there is high risk, at the top of the market, and you are doomed. Pick the turning point at the bottom of the market and you are set for a big profit.   Read More

What The SUPERBOWL Can Teach Us About CMC Strategy?

by

EVER CHANGE YOUR PLAYBOOK IN A CROWDED HUDDLE?   “Clinical Phase Appropriate” assignment of CMC activities can serve as relief from the pressure of limited resources for carrying out your CMC regulatory compliance strategy. Phase Appropriate is defined as “matching the extent of completion for a CMC activity, as well as its timing for completion, to the patient safety risk associated with each clinical development phase.”   What are you trying to accomplish? You may not be trying to win a game, but it’s likely you’re considering how to make decisions to drive development, satisfy commitments with increasing expectations, appease Read More