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

Is NICE Denying Patients the Best Treatments Available Today?

Posted by Judi Space on Sat,Oct 05, 2013 @ 08:00 AM

NICE’s Policies Under Fire

Will NICE's stringent drug approval process discourage drug developers in the UK, or is their cost-benefit approach likely to entice other countries to follow suit?The National Institute for Health and Care Excellence (NICE), an organization given the responsibility of setting the standards for high quality healthcare in the UK, has come under fire for being too conservative and resource-intensive.  In a recent opinion piece in The Times of London, business editor Ian King cautioned that the pharmaceutical industry could abandon drug development in the UK if NICE doesn’t ease its practice of restricting access to drugs based on expense or side effects.

Quoting Jonathan Emms, Pfizer UK managing director, King said that NICE turned down 40 percent of new medicines last year, advising the National Health Service (NHS) that it cannot use them or restricting their use.  Enns pointed out that “19 out of 20 attempts fail before they reach the patient – even before any NICE review,” and added, “NICE is denying patients access to some of the best treatments available today.”  

Drug Developers and the Cost of Bringing a Drug to Market

NICE CEO Andrew Dillon said that his agency is chartered with providing the NHS the best medicines available at affordable prices.  He added, “NICE is, quite properly, scrutinized closely on its decisions and the methods we use to arrive at them. We have changed and improved over the decade and more that we have been advising the NHS.  We are not perfect, but we are respected throughout the world for our quality of work.”

Dillon added, “Companies are entitled to expect a return on their investment, but health services have to be confident that the extra benefit to patients justifies the price.  It mostly does so, though sometimes at a stretch.  If we are not sure, we have to say so, in the interests of all those of us who expect the NHS to apply its resources equitably across all of the demands we make of it.”  Using the widely-quoted estimate that it now costs 1.2 billion pounds (approximately $1.9 billion) to bring a new drug to market, NICE CEO Andrew Dillon countered, “if it really does cost 1.2 billion pounds to develop a new drug, the question the pharmaceutical industry must be able to answer is this: Are you absolutely confident that it needs to.”   Dillon contends that the “research and clinical environment (in the UK) holds too many advantages for companies” to abandon the country. 

Are the UK Market Advantages Enough to Keep Drug Developers Interested?

Stephen Whitehead, CEO of ABPI, the industry body negotiating on behalf of the branded pharmaceutical industry, warns that this “advantage” in biopharmaceutical R&D is “fragile and under constant challenge,” citing that UK’s share of global clinical trials fell from 6% to 1.4% in just 10 years.  He says, “The consequences of this is the closure of six research sites in the UK over the past six years.”

Whitehead concluded, “The biopharmaceutical industry has proven that it delivers solutions to healthcare challenges but the conservative and resource-intensive approach taken by NICE adds significant cost to the development of medicines.  Through its decisions, NICE has a role in repeatedly denying patients in the UK access to medicines that are routinely available in other countries.”

Clearly, drug companies are not going to abandon the development of new drugs just because a small segment of the market will not pay for them. There is plenty of incentive from the rest of the globe. NICE is the bellwether, far out in front of other countries that are trying to impose some control over the run-away cost of new drugs. What Big Pharma really fears is that other countries, particularly in Europe will take on the NICE attitude.

What do you think will happen?  


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: Drug Developers, Drug Development, NICE

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