As Pfizer, Moderna, and other pharma
companies prepare to seek emergency authorization for their SARS-CoV-2
vaccines, the FDA has laid out a roadmap designed to ensure appropriate
scientific rigor and help engender public trust. That plan was the subject of a
special meeting of the Vaccines and Related Biological Products Advisory
Committee (VRBPAC) on October 22, 2020, where experts discussed 2 critical FDA
guidance documents that provide a blueprint for development and approval of
SARS-CoV-2 vaccines. That blueprint is at the center of a massive government
effort to quickly and safely speed vaccines to the American public and bring
the pandemic to an end.
Members of the VRBPAC, with expertise in
infectious disease, epidemiology, and vaccine development, focused on issues
around the FDA standards for safety and effectiveness that will support
Emergency Use Authorization (EUA) of vaccine candidates. They discussed the
need to continue the phase 3, randomized, placebo-controlled trials to
completion after an EUA is granted. They considered how the vaccines will be
rolled out to the American public, and they raised concerns about whether the
public will embrace the vaccines and roll up their sleeves.
When utilized to its full potential, the Target Product Profile (TPP) is a dynamic, living document that ensures all stakeholders—clinical, regulatory, quality and manufacturing, commercial, market access, and medical affairs—are working from the same blueprint. Unfortunately, the TPP often has a bad rap within industry because many people think it is too rigid for today’s drug development environment. But often that reflects a failure to truly collaborate or a tendency to let the TPP get stale. To be effective, the TPP must be continually updated based on changes in the data and the competitive landscape. When companies take a balanced approach to developing the TPP and have a dynamic process that allows them to monitor and adapt it, as needed, they build agility into their drug development program that allows them to make critical go/no-go decisions or course corrections when necessary.
By using the TPP to ensure everyone is on the same page, drug developers can avoid costly delays when, for example, manufacturing isn’t ready to scale up to commercial production when the phase 3 data comes in ahead of schedule. Keeping a close eye on the evolving therapeutic landscape helps the development team anticipate what data will be needed to support labeling claims that may serve as a key differentiator from the competition and provide added value in the marketplace. So let’s look at how a dynamic TPP—one that is proactively updated—can help achieve the critical success factors introduced in the last installment. Continue reading
Bringing a drug to market is a long and expensive process. An analysis by the Tufts Center for the Study of Drug Development estimated the total cost of development from discovery to commercialization at $2.6 billion over the course of about 10 years (based primarily on big pharma companies). This represents more than a 10‑fold increase since the 1970s, when a drug could be developed from bench to bedside for less than $200 million (Figure 1).1 Others estimate the cost to commercialize a drug to be much lower (< $1 billion when they consider small biotech companies),2 yet it is generally accepted that the cost of drug development is on the rise. A major driver of those rising costs is the money spent on drug candidates that never make it to market because of safety concerns or lack of efficacy. The bottom line is that there is no room for costly mistakes, miscalculations, or inefficiencies in the drug development process.
One of the fundamental responsibilities of the US Food and Drug Administration (FDA) is to approve effective medicines for people who need them, while upholding high standards for safety. That mission also demands that the FDA work efficiently and not delay approval of life-saving medical advances. Today, the FDA is reviewing applications for approval of new medicines faster than ever, and that’s a welcome change from the status quo 25 years ago. In the era from 1962 (immediately following the thalidomide recall) to the early 1990s, FDA review times for a New Drug Application (NDA) or Biologic License Agreement (BLA) were often measured in years rather than months. In 1993, the standard review time (from NDA or BLA submission to decision) for a new molecular or biologic entity (parlance for a drug not previously approved for any other use) was about 28 months,1 and in some cases, approvals were delayed for many years.
On January 20, members of the International Committee of Medical Journal Editors (ICMJE) announced a proposal that would require the authors of clinical trial publications to share the deidentified individual patient data that support their published results within 6 months of publication. Announced in an editorial published simultaneously in multiple medical journals, this proposal is based on the belief that authors have an “ethical obligation to responsibly share data generated by interventional clinical trials.” It also reflects the broader agenda of the ICMJE to foster greater transparency and reduce the potential for bias. This new requirement will likely go into effect in 2016 and will affect any clinical trial that enrolls patients beginning 1 year after ICMJE adopts the requirement.
This proposal makes a lot of sense in the interest of transparency, but what does it mean for clinical investigators involved in research and the companies that sponsor that research? To quote the ICMJE authors, “enabling responsible data sharing is a major endeavor that will affect the fabric of how clinical trials are planned and conducted and how their data are used.” Continue reading
When President Obama’s White House Task Force meets for the first time this week, it faces a lofty charge: “Let’s make America the country that cures cancer once and for all.” His proposed Cancer Moonshot 2020 initiative will provide much needed funding for research, will encourage collaboration between industry and academia, and will undoubtedly lead to important advances in our understanding of cancer and how to treat it.
Although this initiative is noble and well intentioned, calling for a “cure for cancer” oversimplifies the challenge and is based on an outdated understanding of the disease.
The public needs to understand that cancer is not one disease; it’s hundreds of different diseases, and it’s more complex than anyone imagined back in 1971 when President Nixon first declared war on cancer. Those involved in cancer research know that conquering cancer, in all its varied forms, is truly a global endeavor that will require a sustained commitment for decades to come. While a challenge to be the “country that cures cancer” stirs our patriotic spirit and conviction that American biomedical know-how can render cancer irrelevant in our lifetime, the reality is that meaningful progress will require international collaboration.
Are we, as a nation, prepared to take on that challenge and lead such a charge? The President has taken the first step, appointing Vice President Joe Biden to lead the White House Task Force. But now the difficult work begins. Can we balance competing priorities and sustain both our political will and financial commitment for the many years that such an effort will undoubtedly take? Continue reading