Drug Development Clinical Trial Data – Public or Private?

Posted by Judi Space on Thu,Jan 23, 2014 @ 06:00 PM

Should drug development information from clinical trials be made public or kept private? In the past, data from clinical trials of new drugs and medical devices have been the property of the companies that designed them, and they were allowed to control how and what data was released. The FDA and international equivalents research new products before granting approval, so data the company generated during drug development was up to the company to disseminate as it wished. Now, with the widely held perception that companies have only been releasing results that favored their products, the pendulum is swinging toward regulation making all data on approved drugs and devices available to the public.

The Transparency Campaigners

In Britain, the transparency initiative has been headed by the British Medical Journal, which now requires all authors of drug or device clinical trials who publish in the Journal to provide full scientific study data to anyone with a reasonable request. Also, the Cochrane Collaboration (an international non-profit) began campaigning for increased transparency when it was attempting to verify the efficacy of TamiFlu and was unsatisfied with the available data. 

These major players are joined by doctors, researchers, medical journals, and scientific societies who advocate more transparency in publishing detailed results of clinical trials and as much data about the specifics of the trials as possible. They have been pressing for this data not only going forward, but also for all medicines currently on the market.

The Drug Regulators: EU, EMA, FDA

In December 2013, the EU drafted a clinical trials regulation that will require clinical trials to be registered and results uploaded to a database that can be accessed by the public. This legislation is expected to be formally approved this year. Also, the Public Accounts Committee (of British Parliament), has issued a report this monththat calls for “full methods and results” to be released from all clinical trials, future and past.

Meanwhile, the European Medicines Agency (EMA), which is the European equivalent of the FDA, is considering a new policy on proactive disclosure of trial results, but further discussion of the issue has been tabled until March. Last year, The EMA received injunctions from both AbbVie and InterMune received injunctions against the EMA to prevent it from releasing trial data to competitors, but these injunctions were lifted last month.

In the United States, basic results of all clinical trials must now be registered in a national database that is accessible to the public (www.clinicaltrials.gov). The FDA also publishes staff reviews and other documents when it approves a new drug. However, federal laws restrict what kinds of information can be released, especially data that could reveal personal or commercially confidential information.

Self-regulation by the Pharmaceutical Industry

The European Federation of Pharmaceutical Industries and Associations (EFPIA) and the Pharmaceutical Research and Manufacturers of America (PhRMA) are fighting back by drawing up “principles for responsible data sharing” for pharmaceutical companies to voluntarily abide by. Some companies have already promised to release years of detailed clinical data upon request, in response to public relations issues.

The ABPI (Association of the British Pharmaceutical Industry) has pointed out that a peer-reviewed study it commissioned in 2013 showed a “positive trend” of increasing levels of disclosure – almost nine out of ten of all industry-sponsored trials were disclosed as of January 31, 2013.

Balancing Economic Interests with the Public’s “Right to Know”

PhRMA has argued that proposals for mandates to release all trial information go too far. “If you dump onto the sidewalk all the data, and you include commercially protected information,” he said, “then you’re essentially giving to competitors what we invested billions of dollars in.”

PhARMA claims that if the medical research industry is forced to disclose data that allows competitors to replicate their products, companies will be deterred from developing new drugs and devices that could improve quality of life for many people. On the other hand, patent protection prevents this kind of product replication. At the same time, publication bias can be used by unscrupulous members of the industry to mislead the public.

When the EFPIA/PhRMA pact was announced last year, EFPIA acknowledged that the industry was slow in affecting change. Given the exposure this topic has received recently, efforts to self-regulate may be too late to affect the groundswell of opinion in favor of legislating more transparency in drug development.

This post is based on an article by Ian Schofield – “The rising tide of trial transparency.” 


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Drug Developers, EMA, Drug Development, Clinical Drug Trials

New Cancer Drugs Fuel Debate over Drug Testing Process

Posted by Judi Space on Thu,Oct 17, 2013 @ 06:00 PM

Question:
If the results of initial testing on a drug are amazing – and could mean the difference between life and death – should pharmaceutical companies be required to go through the current long and costly process of drug testing before making it available to the public? 

Is the FDA's quick drug approval process better, or is NICE's cost/benefit approach the right way to go? Read and decide.If a drug is fine-tuned to treat a specific genetic profile, does it make sense to do randomized controlled trials, even if it means delaying the availability of the drug and possibly increasing the cost, thereby rendering it unattainable to certain patients?  

The Renewed Debate over Drug Testing

Two drugs that were recently approved by the United States Food and Drug Administration (FDA) have helped renew this debate. In 2011, Zelboraf, a drug that targets specific mutations in cancer cells, was approved by the FDA after an early-stage trial showed eight out of ten patients experienced significant tumor shrinkage.  Earlier this year, another drug – Tafinlar, a drug targeting the same mutant genes as Zelboraf - was approved by the FDA based on a single clinical trial of just 250 patients. 

Tafinlar is available at 30 percent less the cost of Zelboraf. 

Are Randomized Controlled Trials Becoming Obsolete?

For many years, randomized controlled trials have been the most effective way of seeing if a drug works.  However, according to Richard Pazdur, M.D., the FDA expert who wanted to shorten the Zelboraf trial, “The types of drugs that we’re seeing now are different.  They are just simply better in terms of efficacy.”    Scientists have a better understanding of molecular changes that promote cancer growth.  For example, an estimated 50-60 percent of melanoma patients - the people Zelboraf and Tafinlar target - have a specific genetic mutation. For drugs that focus on patients with specific genetic mutations, randomized controlled trials may not make sense anymore.

Will Faster Drug Approval Impact Pricing?

Eleven of the twelve cancer drugs launched in the United States last year cost more than $100,000 a year per patient.  In April, more than 100 leukemia specialists from around the world spoke up in the American Society of Hematology’s journal Blood that cancer drug prices were “too high, unsustainable, may compromise access of needy patents to highly effective therapy, and are harmful to the sustainability of our national healthcare systems.”

Accounting for 36 percent of the total research expenditure in 2012, clinical trials are the biggest single cost in drug company R&D.  Many contend that if researchers were permitted to test certain treatments on just people with a specific targeted mutation, researchers could work more quickly, and with fewer patients, to determine if the treatment is effective - bringing these treatments to patients more quickly and, possibly, more cheaply.  Unfortunately, there has not been a significant decrease in medications with the most recent changes in testing procedures.  Paul Workman, head of drug discovery for Britain’s Institute of Cancer Research says, “The costs should be coming down tremendously.  What’s disappointing is that we haven’t seen it happen yet.”

European Drug Regulators Vs. FDA

Part of the issue is that European drug regulators and particularly government funding agencies, such as the National Institute for Health and Clincial Excellence (NICE), may not be willing to accept products based on the FDA’s more flexible clinical trial standards.  In Europe, even if a drug is approved by the European regulatory agencies, if the governments don’t approve funding for it, it will still not be available to many people.

It will be interesting to see what happens.  As Workman said, “We are in a fascinating but frustrating period of transition.”

This post is based on Insight: How new cancer drugs can skip randomized trials by Julie Steenhuysen and Ben Hirschler published in Reuters on September 26, 2013.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Cancer Drug Development, Drug Development, NICE, Clinical Drug Trials

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