Clinical News Roundup: Senate Confirms Gottlieb As FDA Commissioner

By Ed Miseta, Chief Editor, Clinical Leader

The Senate on Tuesday afternoon voted along party lines to confirm Dr. Scott Gottlieb as the next U.S. FDA commissioner. Gottlieb, who faced limited resistance during his Senate committee hearing for his close financial ties to the life sciences industry, will fill a position most recently held by Dr. Robert Califf, who served under President Barack Obama until last January.

In his confirmation hearing, Gottlieb made it clear that he would not be afraid to offer conflicting views with the administration. He also pledged his support for the agency’s mission, calling for a "bottom up" approach from career staff and guided by "the science and what’s good for patients."

"There is one standard for safety and effectiveness, and no commissioner can change that," he added, noting his previous work from 2005 to 2007 as FDA deputy commissioner and before that, from 2003 to 2004 as a senior adviser to FDA Commissioner Mark McClellan and as the FDA’s Director of Medical Policy Development.

Gottlieb received his doctor of medicine at Mount Sinai School of Medicine of New York University in 2002, is a former resident fellow at the conservative American Enterprise Institute and assistant professor at New York University School of Medicine.

INC Research And inVentiv Health To Merge

INC Research Holdings, Inc. and inVentiv Health, Inc. announced their Boards of Directors have unanimously approved a definitive merger agreement under which their businesses would combine in an all-stock transaction, creating a leading global biopharmaceutical solutions organization.

Upon closing of the transaction, INC Research shareholders are expected to own approximately 53 percent and inVentiv shareholders are expected to own approximately 47 percent of the combined company. Advent International and Thomas H. Lee Partners, two preeminent private equity firms, are currently equal equity owners of inVentiv and will remain investors in the combined company upon closing of the merger.

The announcement will make the combined company a top three CRO globally with more than 22,000 employees spanning more than 60 countries and serving customers in more than 110 countries. The new organization’s combined clinical scale, therapeutic depth, and expertise are expected to allow it to partner with biopharmaceutical companies of all sizes to navigate an increasingly complex biopharmaceutical development and commercialization environment.

Alistair Macdonald, Chief Executive Officer of INC Research, said, “Today marks a significant milestone for INC Research. Customers are increasingly seeking simultaneous approvals and product launches in multiple markets worldwide. Through this strategic combination we are bringing together two of the most innovative and respected players in the field to create a leading global biopharma solutions organization with a full suite of clinical and commercial solutions to address the needs of biopharmaceutical companies, patients, physicians, and payers.”

Michael Bell, CEO of inVentiv Health, said, “As biopharmaceutical companies of all sizes face increasingly complex challenges to bring products to market, they are seeking comprehensive outsourced solutions across the clinical and commercial spectrum. The new company is purpose-built to address market realities where clinical and commercial must work together, sharing expertise, data and insights, to improve client performance.”

Cancer-Stricken Researcher Helps Patients Navigate Trials

Researcher Tom Marsilje has made a name for himself by helping others learn about new clinical trials. He has scoured the database ClinicalTrials.gov, circulated a weekly spreadsheet, and served as a clearinghouse for patients desperate for a chance to beat back their disease. 

For Marsilje, it has been a labor-intensive process, and it has been hard to escape a troubling reality. He is waging his own fight against stage 4 colon cancer, and if he ultimately succumbs to the disease, his community of fellow patients could lose the kind of information they need most (the kind he provides).

On Wednesday, in partnership with a New York-based health technology startup and the advocacy group Fight Colorectal Cancer, Marsilje launched a free online site that largely automates the clinical trials search for patients with his disease, known as microsatellite stable (or MSS) colon cancer.

The idea is to radically simplify the process of digging through ClinicalTrials.gov, the government’s compendium of tens of thousands of clinical trials, which is designed more for researchers and physicians than patients. Marsilje said he hopes the service can one day be expanded to simplify the way people search for clinical trials for other diseases. As he put it, he wants to focus on “changing the face of how many types of patients search for and find clinical trials.”

Marsilje, 45, said profit-driven technology companies have tried before to build apps to filter ClinicalTrials.gov, but they generally rely on automated keyword searches, like “colorectal cancer.” Such an approach can miss important clinical trials that are designed for “solid tumors,” for instance, without mentioning colorectal cancer by name.

His results are more curated. With the exception of immunotherapies, which harness the body’s own immune system to fight cancer and which offer the possibility of long-term benefit, Marsilje excludes Phase 1 trials for colorectal cancer because he views the potential risks and benefits as too speculative. Since only a few new trials appear weekly for his type of cancer, he can manually update his list within an hour.

Should Trial Participants Help Determine Drug Pricing?

People who volunteer to participate in clinical trials of new drugs provide a valuable service to pharmaceutical companies and patients. In return, should they have a say in how much these drugs will cost when they hit the market? Spencer Phillips Hay, a research fellow in the Program On Regulation, thinks they should. He notes this would not only honor their service, but also provide a patient-centered mechanism to lower the price of new drugs.

The high cost of prescription drugs is a problem. It was a central topic in last year’s presidential election and continues to remain a pressing issue for legislators, policymakers, and the public. Hay notes one of the major challenges is that it is often unclear exactly what drugs cost. Currently, there is kind of a shell game taking place between pharmaceutical companies (who set the initial price), insurance companies (who pay the bill and set premiums), and pharmacy benefit managers (who negotiate secret rebates on behalf of the insurance company). Each party is now pointing fingers at the others for concealing the true price of medications and putting profits ahead of patient benefits and the public good.

But one group that has a large stake in the cost of drugs has been largely overlooked in this discussion: Those individuals who participate in drug trials. Although our system of research oversight and regulation has long emphasized the vulnerability of trial participants, Hay believes they are also in a potentially powerful position.

No new drugs can be developed and brought to market without people agreeing to participate in company-sponsored drug trials. “They are essentially donating their bodies, and in some cases their lives, for the benefit of future patients — not to mention for the financial enrichment of drug companies,” he states. “I propose that every drug company should have to work with its clinical trial participants to set a price limit on the new medication, should it come to market, and to put this limit into a binding contract.”

Funders And NGOs Implement WHO Standards On Trial Results

Some of the world’s largest funders of medical research and international non-governmental organizations (NGOs) have agreed on new standards that will require all clinical trials they fund or support to be registered and the results disclosed publicly.

In a joint statement, the Indian Council of Medical Research, the Norwegian Research Council, the UK Medical Research Council, Médecins Sans Frontières and Epicentre (its research arm), PATH, the Coalition for Epidemic Preparedness Innovations (CEPI), Institut Pasteur, the Bill & Melinda Gates Foundation, and the Wellcome Trust agreed to develop and implement policies within the next 12 months that require all trials they fund, co-fund, sponsor or support to be registered in a publicly-available registry. They also agreed that all results would be disclosed within specified timeframes on the registry and/or by publication in a scientific journal.

Studies note about 50 percent of clinical trials go unreported, often because the results are negative. These unreported trial results leave an incomplete and potentially misleading picture of the risks and benefits of vaccines, drugs, and medical devices. This situation can also lead to the use of suboptimal or even harmful products.

"Research funders are making a strong statement that there will be no more excuses on why some clinical trials remain unreported long after they have completed," said Dr Marie-Paule Kieny, assistant director-general for Health Systems and Innovation at WHO. The signatories to the statement also agreed to monitor compliance with registration requirements and to endorse the development of systems to monitor results reporting.