Overall, by job type by function, Regulatory/Quality and Clinical outpaced all others at 33%; followed by: IT-Finance General Executive Administration, 27%; and R&D 12%. The last two functions—Sales and Marketing and Manufacturing were down by 16.5% and 38% respectively.

We will follow ZRG Partners Global Life Sciences Hiring Index for the rest of the year, and use it as a guide to inform our 2012 Salary Survey, which will publish in December of this year. We again will partner with CenterWatch on this important industry survey.

During the panel, Joseph Kim, Director of Clinical Operations for Shire, asked the patients their views on different topics concerning both trials and drug research. It was a very honest and frank discussion from the panelists, who have truly suffered and have come to grips with their diseases through medications, procedures, lifestyle changes, and reaching out to others. One panelist, Carol Levy, a patient health activist, has had trigeminal neuralgia, a severe and oftentimes disabling facial pain disorder, for over 30 years. Carol is the founder of “Women in Pain Awareness” an online community and has written a book A Pained Life – A Chronic Pain Journey. After the conference, she wrote two blogs. In her first blog, “Yesterday was the Best Day in a Long Time,” Carol talks about the conference, the conversations she had, and what a great experience it was. Carol’s second blog, “Clinical Trials, Info and What do You Think” talks about what she learned at the conference about clinical trials. She posts links for patients to learn more about trials, what the different phases are, where to find trials.

I’d like to thank Carol for sharing these experiences with her readers, and with the conference attendees. It was a truly valuable experience for all.

I may seem to write about cardiac safety a lot, but it is extremely important in clinical trials and overall drug development. So many drugs have been flagged with cardiac safety issues, it is a wonder that all drugs are not required to have some kind of cardiac safety evaluation. However, we know the reason is that it would not be feasible, economical, nor necessary for any regulatory authority to go that route.

Instead, pharmaceutical sponsors rely heavily on their specialized ECG-related vendors for their medical knowledge to perform these tests on certain therapeutic areas, certain drug classes, and certain populations.

Oncology and cardiac signaling

One therapeutic area gaining attention for the cardiac safety issues is oncology. Safety issues and benefit risk evaluation in oncology is different than other therapeutic areas obviously because of the complexity and severity of many cancers. Because of that two of the most recently approved oncologic drugs have a REMS requirement from the FDA—Nilotinib for Chronic Myelogenous Leukemia and Vandetanib for Medullary Thyroid Cancer—the former has a medication guide only, the latter an Element to Assure Safe Use (ETASU), which is the most restrictive of REMS requirements.

CardioCore recently posted a white paper “Major Cardiotoxicity Biomarkers During Oncology Development” to our web site. Some white papers can be overly promotional, however, this white paper an informative account of the issues around cardiac safety issues in oncology, as well as offering a round-up of biomarker activity for all cardiac-related events.

Dr. Kelsey’s reaction to thalidomide exemplifies the FDA’s mission: protecting and promoting the health of the American people, using science for regulatory decision-making.
Now I know that in some circles regulation is viewed as a roadblock to innovation and economic growth. But in actuality, when done right, regulation isn’t a roadblock; it’s the actual pathway to achieving real and lasting innovation.

Smart, science-based regulation instills consumer confidence in products and treatments. It levels the playing field for businesses. It decreases the threat of litigation. It prevents recalls that threaten industry reputation and consumer trust, not to mention levying huge preventable costs on individual companies and entire industries. And it spurs industry to excellence.

The tragedy of thalidomide led to changes that strengthened both the regulatory and scientific environment for medical product development and review.

In response to the public uproar, in 1962 Congress enacted the Kefauver-Harris amendments to the Federal Food, Drug and Cosmetic Act. Thanks to these new amendments, manufacturers had to prove that a drug was not only safe, but also effective. Approvals had to be based on sound science. Companies had to monitor safety reports that emerged postmarket and adhere to good manufacturing practices that would lead to consistently safe products. And there were new protections for patients.

The amendments not only benefited patients, they helped industry, raising scientific standards that eventually ushered in today’s sophisticated, science-based life sciences industry.

For the very first time, many companies put in place research and development programs, including the design and implementation of controlled clinical trials. Major therapeutic breakthroughs resulted, including the use of beta blockers in patients after a heart attack and angiotensin-converting enzyme inhibiters to improve survival in patients with heart failure. All of these were good news for public health and for corporate bottom lines. The best drugs and treatments rose to the top, not simply those that were most heavily marketed.

The Harris-Kefauver Amendments created a culture of quality and innovation that laid the foundation for our current regulatory environment which fosters a domestic pharmaceutical industry that is second to none.

Going forward, smart regulation requires regulatory flexibility that responds to changing situations, new information and new challenges. It also demands that we advance regulatory science: the knowledge and tools necessary for the meaningful and timely review of products for safety, efficacy, quality and performance.

Thalidomide, once again, is a good example. It came back on the US market in 1998 after data showed it was safe and effective to treat a complication of leprosy. In an appropriate balancing of benefit and risk, FDA required strong safety monitoring and a strict dispensing plan before approving the drug.

Regulation such as this requires a strong, robust FDA, one endowed with the necessary resources to ensure smart, sound, science-based regulation.

Written by Margaret Hamburg, MD, Commissioner of the US Food and Drug Administration, for FDA Voice.

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Remembering the Thalidomide Tragedy at ICPE 2011

A new study published in the Archives of Internal Medicine has found that close to half of the AEs in the database have led to label changes.

Strictly from a consumer point of view: if the reported AE is serious enough to warrant a label change in 50 percent of the cases, how can the FDA advise prescribers and users to take no action?

At the very least, doctors should be encouraged to inform their patients of the new information, so the patient can make a better decision concerning their use of the product. Obviously there are situations where the end user has no choice but to continue using the medication in question due to the lack of any other viable options, but they should, at a minimum, be informed of the possible risk.

By telling doctors to take no action, the FDA is in essence encouraging physicians to withhold valuable information from their patients: a thought no doubt disturbing to anyone placing their trust and health in the hands of another.

But once again, European regulators are under the harsh glare of scrutiny, and—once again, as in the recent Mediator case, when questions were raised over patient injury from Servier’s anti-diabetic medicine, it is a French company that is the focus of the criticisms.

To be fair, Poly Implant Prothèse, the company under the spotlight now, for supplying non medical grade silicone in breast implants, received its product authorization from Germany. But the essential issues are the same—questionable business practice, and insufficiently stringent control by health authorities.

In mid-February, the European Commissioner for health, John Dalli, announced a new crackdown on the devices industry in Europe. It is necessary, he said, to tighten controls and increase surveillance, so as to protect citizens and to restore confidence in the regulatory system.

“Patients’ health is the priority in this situation”, he intoned—rather obviously—as he made his announcement. But although obvious, it needed saying. And he admitted that the case had exposed “major loopholes” in the legislation. Equally obvious, but equally important as a starting point for reform.

Dalli’s admissions won immediate support from the European body representing consumer associations, known by its French initials as BEUC. “The existing EU medical devices legislation is weak and the overall system is flawed,” said a spokesman. “Until the system is improved we cannot be sure that the medical devices we are using are safe and effective.” The spokesman concluded that “restoring consumer’s confidence needs to be top of the agenda.”

Even the European medical technology industry has had to recognize “the need to improve the effectiveness of the current European regulatory framework for medical devices.” Eucomed, the industry’s European association, said it “appreciates the European Commission’s swift reaction,” and considers “the measures are balanced and appropriate and will have a positive effect on patient safety.”

From a technical point of view, the parallels between marketed breast implants and clinical trials for medicines yet to be launched are indeed indirect. Breast implants fall under European legislation on medical devices, which requires manufacturers themselves to ensure that their products will not compromise patient safety. Control for high risk devices, such as breast implants, is supposedly ensured by a third party conformity assessment body, so-called “notified bodies.” The crucial weaknesses identified in this case are that the notified body was not operating a very effective conformity assessment, and the company was taking advantage of the laxity by fraudulently using industrial silicone instead of the approved medical grade silicone.

But from a consumer perspective, a patient perspective, a safety perspective, a confidence perspective, there is plenty of overlap with clinical trials. Without adequate rules and proper enforcement, things can go wrong, and patients—or subjects—suffer. The key point in Dalli’s recipe for remediation is to ensure that notified bodies do their job properly—including conducting unannounced inspections. “These are an important aspect of what can already be done now,” he emphasized.

The answer to the challenge is not, Dalli said, to ban all implants—any more than safety would be improved by banning all clinical trials because a problem emerges with one trial. He underlined that the implant case was essentially a matter of fraud. This, he went on, can be tackled only by better market surveillance and controls. It happened with these implants because “inspections of the notified body were always announced two months in advance,” Dalli accepted—adding “This is not the proper control procedure to adopt.”

And what is sauce for the goose of devices is sauce for the gander of clinical trials. Complaints are frequently raised about the frequency and intensity of inspections of clinical trials for GCP or GMP—and they can of course be onerous and time-consuming and occasionally appear unduly intrusive. But they are guarantee that is needed by the public, the patients, the subjects, that regulations are not only in place, but are being implemented and enforced. And that, for confidence to be maintained in the regulatory apparatus, is what has to happen. It is the price of ensuring not only safety, but ensuring just as importantly that safety is seen to be secured.

The documents describe a step-wise approval pathway, starting with extensive analytical, physico-chemical and biological characterization data that can demonstrate a high degree of similarity. FDA will evaluate that data and then provide advice to the sponsor on the extent and scope of animal and human testing needed to show biosimilarity. FDA will consider multiple factors in making study determinations, including product complexity, formulation, stability, structure-function relationships, manufacturing process and clinical experience with the reference product.

Unlike European regulators, FDA decided not to issue guidances for developing biosimilars in different drug product classes. If a specific issue arises with a specific class, we may write guidance, explained Rachel Sherman, Director of the Office of Medical Policy in FDA’s Center for Drug Evaluation and Research. But so far, she said at a press briefing, “this has not been seen at the most efficient route.”

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