At a recent conference of biotech executives, frustration seemed to be the order of the day with venting the inevitable result. Both are understandable. The remarks (dutifully reported by Lisa Richwine of Reuters and reprinted below) show an industry and an agency engaged in a life-or-death battle. Actually, two life-or-death battles. The first is corporate survival, the second, patient survival. And while saving lives is more important than making money (is it even necessary to say that?) you can’t have one without the other. No biotech industry, no new lives saved, extended, or enhanced.

The general frustration is the on-going dichotomy of predictability versus ambiguity at the FDA. One reviewer deals with a sponsor one way while two doors down data is addressed in an entirely different manner. Predictability is power in pursuit of the public health. And while that’s always been true, today it’s become an urgent public health imperative.

Reform is crucial. Or should I say “critical” … like in Critical Path.

Here’s the Reuter’s article. Read beyond the frustration and the venting and you’ll see an opportunity for greater collaboration between regulators and regulated. A high-wire balancing act? Certainly. But one well worth the effort.

Business as usual is not an option.

Biotechs say FDA ranges from good to ‘horrifying’
By Lisa Richwine

BOSTON (Reuters) — Biotechnology companies trying to get new drugs to the market have had experiences with U.S. regulators that range from productive to “horrifying” as they craft early development plans, senior executives told Reuters this week.

Interactions can vary vastly between Food and Drug Administration divisions that review products and provide guidance to companies long before they seek approval to sell a drug, company officials said.

“We have some very good experiences and we have had some horrifying experiences, where you wonder, ‘Where did that come from?’” Genzyme Chief Executive Henri Termeer said at the Reuters Biotechnology Summit in Boston. “What is reasonable and what is not reasonable differs by individual … It’s quite frustrating,” he added.

FDA officials have acknowledged inconsistencies, or a lack of clarity, from some reviewers during early stages, when companies are seeking input on how to design clinical studies of experimental medicines. They have pledged improvements and launched major efforts to work with manufacturers earlier in the process to help more drugs make it to the market.

The agency holds more meetings than ever with drugmakers, more than 2,000 annually.

“We believe more and better interactions will lead to better outcomes and have invested a lot of resources in these efforts,” Scott Gottlieb, FDA deputy commissioner for medical and scientific affairs, said via e-mail. The FDA also is providing guidelines on how to develop better applications, he said. Officials will announce other measures to improve the review process soon.

Biotechs can face more challenges than traditional pharmaceutical companies. Many biotechs are small, inexperienced and testing their first product. Their technology may be new and unfamiliar to regulators.

Alnylam Pharmaceuticals Inc. CEO John Maraganore said his company has had “remarkably good” dealings with the FDA on efforts to develop treatments based on a new “gene silencing” technology.

“You can engage them in a science-based discussion … People that run into troubles don” engage them in that type of dialogue,” he told the Reuters Summit.

FDA staff are very responsive on potential breakthrough drugs, said Ariad Pharmaceuticals Inc. CEO Harvey Berger. Having “fast-track” status for promising medicines helps insure regular communication, he said. His company’s employees talk often with the agency in person and on the phone and get answers to questions within days. “I think the key is you have got to have a drug that they see a real opportunity for,” Berger said.

Cell Therapeutics Inc. CEO Jim Bianco agreed FDA staff will move quickly for drugs that are “sexy,” but said less exciting, though still important, medicines can get left behind. His company has been frustrated in its development of a cancer drug, Xyotax, which he described as a traditional chemotherapy medicine with fewer side effects.

European regulators are open to clearing the drug based on evidence it is equivalent to other chemotherapy, rather than superior, because it has less toxicity, but U.S. regulators are not, Bianco said.

FDA staff “are not equipped to handle that,” he said. To the agency, “standard chemo is not what moves the field forward,” he said.

Genzyme’s Termeer said it has become easier to deal with European regulators, who routinely approve his company’s products before the FDA does.

“The Europeans are willing to delegate much more of the process … to expert opinions. Much more of the work is done outside the bureaucracy,” he said.

Remember the January 30th Federal Register notice where FDA said they were going to investigate whether or not coupons in DTC ads were legit because they might “foster consumer misperceptions about the advertised prescription drug product.” Well, they’ve withdrawn it — quietly. When asked why the agency replied, “The agency withdrew the notice because the matter required additional consideration.” Well said. Translated that means (and this is my best guess) that cooler heads in the agency (most likely in the Office of Chief Counsel) pointed out that FDA has no authority to regulate this area.

Good decision. Good press statement. Face is saved.

Plastics.

It was good advice in “The Graduate,” but it’s bad news for internet profiteers masquerading as pharmacists.

Both Visa and MasterCard rules prohibit the processing of any illegal transactions such as those from illegal online pharmacies. Both card companies have issued fines for doing so. Several online pharmacy accounts, in fact, were recently terminated as a result of card Association and member bank pressure.

To do business with online pharmacies it is required that the member bank conduct an investigation for every online pharmacy. This will include providing a copy of the pharmacy’s license, a statement that the merchant’s sales activity is in compliance with all applicable laws, proof that valid prescriptions are required before medications are dispensed and proof that only prescriptions from licensed physicians are accepted.

Any questions?

The Ol Perfessor (in this case, the young and vibrant Dr. Bob Goldberg) tells it like it is …

The study put out by the Democrat minority staff of the House Government Reform committee, “Medicare Drug Plan Prices Are Increasing Rapidly,” is misleading and inaccurate in many respects.

It deliberately leaves out drug plans that allow patients to pay a fixed low price for all the drugs they survey. There are many drug plans that allow consumers to pay $30 for a month’s supply of each drug (or $25 a month if purchased through mail order). All these prices are substantially lower than any of the average prices cited by the Democrat staff. For example, the Democrat House staff quotes average prices of about $150 for a month supply for Aricept and Advair whereas most plans quote about $27.

Nor does the study does control for different co-pays and prices for drugs depending on its tier placement. Zocor, Prevacid and Lipitor have been shifted into higher co-pay and price tier by some companies and not others. At the same time, some companies have pushed Celebrex off their formulary and raised prices accordingly while others have kept it on formulary.

Even when these errors and oversights are taken into account, a significant number of companies offer drug prices lower than those quoted by House Democrat staff. In many cases the prices quoted by the various plans are about 75 percent lower than the average price cited by the House Democrat staff.

Like Casey said, you can look it up.

I recently had the privilege of a private meeting with Nobel Laureate Joshua Lederberg. The topic of conversation was the future of the FDA and the agency’s Critical Path initiative. We talked about the state of applied research and “the texture” of the agency, the prioritization of development science, biomarkers and a host of other future-oriented issues. He talked. I took a lot of notes. At the end of the meeting he put everything into perspective in a single sentence. He leaned over the table and said, “The real question should be, is innovation feasible?”

I hope so.

Smart thinking from Bob Goldberg, CMPI’s new Vice President for Strategic Initiatives …

Meet the challenge
By Robert Goldberg
Published February 21, 2006
The Washington Times

Democrats are gearing up to make health savings accounts and Medicare battering rams against the Republican Party in in the midterm Congressional elections. Liberal think tanks and congressional Democrats first attacked the Medicare drug benefit as a “a complex program that does nothing to bring down the costs of soaring drug costs.” Now, they are trashing health savings accounts as a tax shelter for the rich without offering an alternative of their own.

They don’t have to. The Bush administration and congressional Republicans have done a poor job responding to criticisms of the Medicare drug benefit and HSAs. Conservatives and think tanks have largely stood on the sidelines as the beating has taken place. Many think they are standing on principle, but in fact it reflects a lack of vision on their part.

The fact is, most conservative think tanks and conservatives frame Medicare and health insurance in terms of reducing health-care costs. As a result, they cede the moral high ground on the health-care issue that is all about coverage and quality of care. Similarly, as I have argued before, conservative griping about the cost of the Medicare drug benefit has blinded Republicans to the fact that Medicare modernization is counterweight to single payer health care.

Liberals complain that even if HSAs become the norm, they won’t slow health-care costs because 80 percent of all health-care spending is incurred after the high deductible ($2,100) is met and would still be generated by consumers using insurers’ money instead of HSAs. That’s true. Then again, the same thing is even more true about single-payer systems or managed care plans where the out-of-pocket cost are nearly hidden.

In either case, it’s the 20 percent of people with chronic illness that generates 80 percent of the cost. Medical progress, not administrative machinations, will reduce the cost of disease.

Conservatives are doomed to lose the HSA debate because they regard them largely as cost cutting tools. To the extent that anxiety about health care coverage is at the heart of uneasiness about a vibrant economy, it behooves the Republican Party to retool their health-care message in general and their HSAs policy in particular.

Republicans must commit to expanding and protecting medical insurance for all Americans. HSAs should be hailed as one way for achieving that goal. Last year, people with incomes of $35,000 or less per year were the fastest growing segment of HSA-eligible health insurance plan purchasers. Sales to this segment grew 104 percent over 2004. MIT economist Jonathan Gruber estimates that more than 1 million working class people will lose their insurance as a result of HSAs being offered. He doesn’t explain why. Maybe the 1.5 million lower-and middle-income people, who were previously without insurance were too stupid to follow an MIT economist’s theory. They actually obtained obtain insurance that included well baby visits, prescription drugs, ob-gyn care and other medical care for about $125 a month, less than what they paid last year.

To continue to boost and maintain insurance coverage among low and middle income Americans, the Republicans should introduce legislation that allows employers to put the full amount of the deductible into HSAs in order to eliminate any out of pocket cost. Working-class and poor families should receive pre-funded debit cards for HSAs with the full deductible amount as well.

The Department of Health and Human Services could help people comparison shop by posting Medicare prices for every single service. It could work with WebMd and other private health-information services to share news about who is doing a good job delivering medical care. Further, since most people rarely spend more than a small percentage of their account, the legislation should allow unused funds — as well as any additional monies contributed by the individual to be used tax free to pay for health care premiums during periods of unemployment as well as any deductibles or co-pays.

Third, HSAs must be compared to liberal efforts to prop up a single-payer and an employer-based health system that is providing less coverage for more money each year. Liberals are responsible for the loss of health care coverage in America. Now they shriek that HSAs would leave less money for subsidizing the increasingly expensive health insurance demands of labor unions, demands that send jobs overseas. Meanwhile, HSAs are giving more working class people more coverage they can keep and call their own for less money.

HSAs are not for everybody or a panacea. They’re only part of a long-term effort to expanding access to health insurance. But if Republicans frame the HSA debate in terms of coverage, they can stop playing defense on health care. First, they have to get passionate about the value of medicine and not what it costs.

Robert Goldberg is vice president for strategic initiatives for the Center for Medicine in the Public Interest.

According to an article in today’s edition of The New York Times, psychiatrists say they have been getting panicked phone calls from patients worried by an FDA advisory panel’s recommendation this month that drugs for attention-deficit disorder carry a prominent warning about heart risks.

Can you say “unintended consequences?”

The calls are coming not just from parents of children who take the drugs but from adult users, who the panel warned might be at the highest risk for heart problems.

Can you say, “irresponsible advisory committee causing increased non-compliance?”

“Every single adult patient I saw today, the first thing out of their mouth was, ‘Am I going to drop dead on this?’ Every single one of them,” said Dr. Timothy Wilens, a psychiatrist at Massachusetts General Hospital in Boston.

Can you say “fear replacing caution?”

Can you say “Precautionary Principle?”

Because if you can’t say it, you can’t fight against it.

And fight against it we must.

The just completed WHO meeting on counterfeit prescription medicines is over. And what took place in Rome should have a chilling effect on us all.

Consider these few facts (direct from WHO, so nobody can say they’re “industry” numbers) —

Between 1998 and 2004 there has been a 1000% increase in seizures of counterfeit prescription drugs

That is not a typo. A 1000% increase.

Between 2001 and 2004 there were 170 official cases of counterfeit medicines originating from an illegitimate supply chain

And most frightening …

Between 2001 and 2004 there were 27 official cases of counterfeit medicines that originated from the legitimate supply chain.

Sound frightening? It is.

Action is required.

While some in the US still don’t believe that counterfeit prescription drugs are a real problem (“Show me the dead Canadians!”) Europe is taking it dead serious.

On Wednesday, the Center for Medicines in the Public Interest (home to this blog) and the Brussels-based Centre for the New Europe co-sponsored a conference titled, “A False Sense of Security — The Growing Threat of Counterfeit Prescription Products.”

Shortly I will post all of the presentations from that event. A book will follow.

The next day (last Thursday) the World Health Organisation and the international pharmaceutical industry launched a global taskforce aimed at stemming “the growing epidemic” of counterfeit drugs that has become a magnet for organized crime.

Here’s what the Financial Times wrote on the subject …

On some estimates, fake drugs account for more than 10 per cent of the medicines market worldwide, worth nearly 40bn US dollars (34bn euros, 23bn pounds) a year. But in developing countries that proportion is more like 25 per cent and in some countries it may be as high as 50 per cent.

Howard Zucker, WHO’s top official on pharmaceuticals, said: “People donç©° die from carrying a fake handbag or wearing a fake t-shirt. They can die from taking a counterfeit medicine.”

The taskforce will focus on strengthening national laws and enforcement, raising awareness by consumers and health professionals, improving international co-operation and developing “innovative technology solutions” including electronic tagging to track fakes.

However, WHO is no longer pressing for work to start on an international treaty to tackle counterfeit medicines, which critics feared could simply serve as an excuse for delay and inaction.

“Clearly what’s needed here is action and we don’t need a convention to take some action,” said Harvey Bale, director general of the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), which is co-sponsoring the Rome conference.

The Centre for Medicines in the Public Interest has predicted that counterfeit drug sales could nearly double to $75bn by 2010. While the problem is worst in poor countries with weak regulation and enforcement, where counterfeiters fake medicines for life-threatening diseases such as malaria, tuberculosis and Aids, much of the growth is fuelled by internet sales into western markets.Counterfeiters are now focusing not just on “lifestyle” drugs such as hormones and remedies for erectile dysfunction, but also expensive treatments for cancer and drugs in high demand, most recently the anti-viral Tamiflu.

The absence of criminal laws against counterfeiting in many countries has encouraged organised crime rings to enter the business.

“Organised crime is gravitating towards the industry because the risks are a lot less than forging currencies or trafficking heroin,” said Mr Bale. “You can make a fortune, at no risk.”

Dr Zucker said: “International police action against the factories and distribution networks should be as uncompromising as that applied to the pursuit of narcotic smuggling.”

WHO said it planned to extend to more regions a web-based system for tracking the activities of drug counterfeiters it set up last year to link national health authorities and other agencies in the western
Pacific.

As I arrived back in the US my mobile phone rang. It was a radio station in South Korea wanting to interview me on the fact that North Korea is now in the business of counterfeiting prescription drugs for profit. The world is paying attention. So must we.

It’s time for US regulators to join this working group and address the problem from a truly transatlantic perspective.

The lives we save may be our own.

Rep. Gil Gutknecht (R-Minn) and Rep. Rahm Emanuel (D-Ill.), the dumb and dumber of Congressional voices calling for drug importation, demanded an explanation on Wednesday for increased government seizures of drugs mailed to U.S. customers by Canadian pharmacies.

They don’t want an explanation — because they know the answer — uninspected, unregulated foreign drugs are unsafe and illegal. This is a recording.

“We believe this unannounced policy of increased enforcement is irresponsible,” the two said in a letter to the Food and Drug Administration and to U.S. Customs and Border Protection.

“There is a growing chorus of outraged Americans concerned that access to affordable prescription drugs is being denied to them,” reads the letter.

It is a chorus of two backed up by a supportive media echo chamber. But the music is out of tune and increasingly out of touch.

Here’s what these two elected representatives of the people are complaining about — recently the FDA launched an investigation confiscating thousands of drug shipments headed for the U.S. Some of them were headed for Minnesotans who ordered them over the state’s Web site. When opened, nearly half claimed to be of Canadian origin but, according to FDA investigators, 85 percent of them were from 27 other countries such as China, Iran, and Ecuador. And 30 of the drugs were counterfeit.

Why did FDA do what they did? Because it’s their job and it’s the right thing to do.

This is a recording. If you are looking for media attention, please leave your name and congressional district at the sound of the beep.

Bob Goldberg on the price-tag for survival …

It is both remarkable and tragic that at a time when we are finally winning the war against cancer, actually reducing the numbers of deaths due to that dreaded disease, the New York Times is editorializing against the value and use of the very medicines that have made such advances possible. Do we really need enemy combatants against medical progress?

The NYT claimed recently that Avastin at a retail price of $8000 for a month of treatment is too expensive for an average of 6 months of more life, without pain or toxic side effects for cancer patients that have not responded to other forms of cancer care. Here’s how I would have put the question: Is it worth having your insurance company pay about $5000 to keep you alive for 6 months or longer? What’s it worth to have drugs like Avastin around to keep tens of thousands of cancer patients alive longer and longer so they can live free of caregivers, can go to work, parent their kids, etc? The Times never asks that question.

Similarly, medical journals are now abandoning real science and are simply becoming collections of editorials by Monday morning quarterbacks. Most recent is the assault on the right of multiple sclerosis patients to use Tysabri to treat the devastating relapses associated with the disease. The British Medical Journal published “Lessons for Clinical Trials from Natalizumab in Multiple Sclerosis” by Abhlijt Chaudhuri, a physician known for believing that no drug for MS should be used without any rebuttal.

Unfortunately, his “lesson” seems to “grin and bear it.” Not acceptable.

The term ‘relapse’ sounds innocuous. It means losing balance, one’s sense of taste, eyesight, memory, etc. without warning. It can make walking impossible. The attack on Tysabri is simply an old saw applied to a new drug: since the drug doesn’t actually stop MS from progressing the possible long term safety problems aren’t worth the risk of returning to market. The article spews the same accusations made of every drug that is experimental: we don’t know what the optimal dose should be and how long people should take it. The fact that only clinical experience can help answer that question is ignored in favor of a standard of evidence that, if applied to the early release of penicillin, the measles vaccine, HIV drugs, and other breakthroughs, would have cost millions of lives.

That is like saying that since some drugs don’t cure a disease, don’t make them available at all. Again, the value of these medicines to people and their ability to give them back lives of productivity, dignity and independence means nothing.

On 2/4, blogging on a report that there was a backlog of generic drug applications at the FDA, I suggested that the agency seriously consider user-fees for this important area of responsibility. And it looks like that’s going to happen.

Scott Gottlieb, the FDA’s feisty and forward-looking deputy commissioner for medical and scientific affairs plans to tell the generic drug industry today that it is time for the government to charge its member companies fees so the agency can hire more staff and exprdite the processing of applications to market new generic medications.

In a speech he is scheduled to deliver to the Generic Pharmaceuticals Association in Florida, Gottlieb plans to say that user fees — like those paid by the makers of brand-name drugs, medical devices and animal drugs — are needed to keep up with generic reviews.

“From our perspective at FDA, we have seen user fee programs applied to our medical device and new drug programs with great success,” a copy of his speech says.

Gottlieb is scheduled to say in his speech that raising funds from generic-drug companies would allow the agency to review applications faster, to have more scientific data analyzing whether proposed new generics are equivalent to established brand-name drugs, and to address potential safety issues once the drugs are on the market.

Per Scott, “It’s not fair to compare our work in our generic drug office to our work in the new drug office, as some have done, without acknowledging that our new drugs program has benefited from funding tools that are not available to us when it comes to generic drugs.”

Use of generic drugs — which must be equivalent to the branded products that they duplicate — has been growing steadily, with more than 53 percent of prescriptions now filled with generics. That percentage could increase
quickly because an unprecedented number of major branded drugs will lose their patent protection in the next few years.

In addition to raising the user-fee issue, Gottlieb is set to outline a number of agency initiatives to speed generic reviews. One is a formal lifting of a ban on direct telephone conversations between FDA reviewers and generic drug makers. Another involves grouping bioequivalence review applications for the same or similar drugs.

You tell em Scott.

Talk about being a Nabob of Negativism! Families USA (the political lobbying group that masquerades as a “citizens advocate”) is downright gleeful in it’s grossly premature burial of Medicare Part D. According to its new report (a love note to the Intelligensia of Interference issued on Valentine’s Day) the Bush Administration has “significantly lowered” its enrollment projections for the new drug benefit.

Really? I don’t recall anyone from the White House saying that? Maybe Ron Pollack, the Families USA “Godfather,” meant White Castle? And I certainly didn’t hear Mark McClellan say anything remotely like that during his recent, highly publicized congressional testimony. And I didn’t read it in a Robert Pear column. So could it really be true?

No. I think that the Godfather must be hearing voices — and that those voices are “interfering” with his cognitive processes.

Giving a new and draconian meaning to “family-friendly,” Godfather Ron also concludes that a vast majority of those enrolled in Part D already had drug coverage before the benefit began and that only “a very small fraction” of beneficiaries eligible for the low-income subsidy have entered the program.

Methinks that the Godfather would like the truth to sleep with the fishes. This new report is just another example of reality non-interference.

I suppose that Families USA expected 100% sign-ups with zero problems after almost a month and a half! Can’t this administration do anything right!

Proving that they are most decidedly living in a Pelosi-induced fantasyland, Godfather Ron commented, “It’s truly disappointing to see such a large number of seniors not get the benefits to which they are entitled.” He further added that the drug benefit has so far “produced rather meager results.”

I guess he thinks that if he keeps saying that enough times people may actually start believing him.

CMS spokesman Gary Karr politely suggested that Families USA spend its time and energies more productively by helping to expand the number of seniors enrolled in Part D instead of taking “ill-informed partisan potshots.”

Good for you Gary.

Karr then said that CMS has not downgraded their enrollment estimates. “The numbers that [Families USA] cite come directly from us. We’ve been open and above board about exactly what those numbers are and where we get our estimate of the 28-to-30 million.” Families USA, citing a January 28, 2005 CMS Federal Register notice, said that the administration had been projecting that 39 million beneficiarieswould enroll.

But it’s the Godfather of Garbage and his “Family” who are doing the projecting. For them it’s all about political spin and vituperative spittle. Anything for the cause.

Well, isn’t this wonderful? The Wall Street Journal reports today that “… the G-8 nations [propose] to subsidize the purchase of new vaccines—-for between $800 million and $6 billion—-if pharmaceuticals companies develop ones that meet standards of efficacy and safety. Once the G-8 spends the pledged amount, the drug companies would sell the vaccine at a set discount in the developing world.”

So: Having damaged the vaccine sector seriously with a combination of price controls, regulatory hurdles, and absurd tort liabilities, the bureaucrats and politicians now propose to undo the damage using taxpayer resources, all the while, of course, congratulating themselves for their compassion. Well, how is it that the private sector ever produced vaccines at all? To say the same thing differently, we now are reaping the fruits of decades of destructive policies; now taxpayers will have to assume investment risks that the private sector is in a far better position to evaluate and bear. Moreover, the risk allocation issue is only the beginning; which vaccines will receive favor? The ones that offer the biggest health bang for the buck? Or the ones that are most favored among the politically corrrect? And will “profits,” however defined, be limited while losses are not? Will the various governments attempt to use profits from other investments implicitly to subsidize these favored vaccines? Etc. Just asking.

A recent inspection by the U.S. Food and Drug Administration found some of the drugs Minnesotans order from Canada may not be from the country at all.

Wallace Greenfield discovered one of his “Canadian” drugs came from Greece, and another came from Vanuatu, a small island in the South Pacific.

“I never heard of the place,” Greenfield said.

The U.S. government says it happens all the time and is a growing concern.

“We were beginning to see a pattern of products coming that were purporting to be of Canadian origin coming from various countries from throughout the world,” said Steve Niedelman of the FDA. “We wanted to determine how widespread this was.”

The FDA launched an investigation confiscating thousands of drug shipments headed for the U.S. Some of them were headed for Minnesotans who ordered them over the state’s Web site.

When opened, nearly half claimed to be of Canadian origin, but “85 percent of them were from 27 other countries,” Niedelman said.

“We saw product coming through from Germany, from Australia, from China, from Iran, from Ecuador,” Niedelman said.

The FDA said 30 drugs were counterfeit.

The Minnesota Senior Federation says 25 prescription orders from Minnesota were among those that were confiscated in recent months.

According to Tom Sheck of Minnesota Public Radio (a media never accused of slanting to the right), “When Gov. Pawlenty announced the MinnesotaRXConnect program two years ago, he predicted that it could cover 700,000 Minnesotans and save millions of dollars for consumers. The actual numbers are well short of those projections, and demand for the program has been declining sharply in recent months.”

It’s time for Governor Pawlenty to relenty and stop his unlawful, unsafe and (fortunately) seldomly used state-sponsored program.

Because today it’s just folly, but it could very well turn into state-sponsored health care terrorism.

During my tenure at the FDA I was the senior official in charge of advisory committees. I was proud to oversee a transparent, collegial, and scientific program crucial to the agency’s mission of protecting and advancing the public health.

Today I am upset and worried about the future of this process.

In the wake of FDA bashing for political and personal gain, the advisory committee process is spinning out of control. Witness yesterday’s unexpected chest thumping by members of the Drug Safety and Risk Management advisory committee

The panel voted 8 to 7 to propose a ‘black box’ warning for methylphenidate drugs, sold under the brand names Ritalin, Concerta, Methylin and Metadate, and on the amphetamines Adderall and Adderall XR, stimulants used to treat attention deficit hyperactivity disorder. The warnings could be rescinded if future studies fail to definitely establish any risk.

But the harm to physicians (worried about law suits) and parents (worried about their children), and the children (who aren’t being appropriately treated) would have already happened. What makes this so very frustrating is that the committee didn’t make its recommendation for a black box based on the available data, members of the board said the recommendation was driven as much by worries that the drugs are being overused in the United States as by the possible side effects.

The FDA advisory committee process in the Age of Grassley seems to be “Science? We don’t need no stinking science.”

And even more frightening is Precautionary Principle creep.

According to the Associated Press, “The surprise recommendation has caught the Food and Drug Administration off guard.”

To say the least.

“You don’t want to overscare people with data that aren’t very solid,” said Robert Temple, director of the FDA’s office of medical policy. He said the drugs carry real benefit for some patients. Before the committee’s vote, Thomas Laughren, who heads the FDA’s division of psychiatric drugs, told the committee he didn’t “think we are there yet with this cardiovascular risk” in terms of justifying a black box.

Science? We don’t need no stinking science.

Here’s a headline from a story by Marla Cone in today’s Los Angeles Times …

“Mercury readings high in state.”

Here’s the lead paragraph:

“Californians have among the worst mercury contamination in the nation, with nearly one-third of those volunteering in a nationwide study exceeding the concentration of the potent neurotoxin deemed safe, according to a study organized by two national environmental groups.”

Here’s the 10th paragraph (of an 11 paragraph story):

“The new study, which is ongoing, is the largest test of mercury exposure in the nation. But the results are not statistically representative of the United States because participants were self-selected volunteers. They joined the study by visiting the Greenpeace or Sierra Club Web sites and sending $25 with each hair sample.”

Sloppy, slanted, spurious reporting.
Please pass the tuna.

I’m sure that Senator Charles Grassley and others who think the FDA should view the pharmaceutical industry in a strictly one-dimensional adversarial fashion will be upset to learn that collegial two-way communications is — gasp — good for the public health.

According to a new report, new medicines reach the U.S. market sooner when regulators meet with drug makers before the final phase of human testing and made sure they were addressing potential pitfalls. The report, by consulting firm Booz Allen Hamilton (undertaken at the request of the FDA), said fifty-two percent of manufacturers that consulted the Food and Drug Administration at that time won approval after an initial review, according to an analysis of 77 drug applications submitted from 2002 to 2004. Only 29 percent of companies that did not have such meetings received clearance for their products during the original cycle. (Medicines that fail to win FDA clearance after the original review of typically six to 10 months may go through multiple evaluations before reaching the market.)

A top FDA official said the agency agreed the early meetings with drug makers were productive but said more staff would be needed if the number increased substantially. (This is code for “we need more money!”)

“We have seen a pretty dramatic growth in the number of meetings we’re having with sponsors in the past several years. Any additional workload for meetings is going to have to be supported by additional staffing,” said Dr. John Jenkins, director of the FDA’s Office of New Drugs.

“Early and open communication with the sponsors will allow sponsors to address/resolve issues in a timely manner, potentially within the first review cycle,” the report said.

Once more with feeling everybody, let’s do the budget season chant — “Show me the money!”

Dr. Bob Goldberg pulls no punches in response to Stephanie Saul’s article (“Record Sales of Sleeping Pills Are Causing Worries”) in today’s edition of the New York Times …

Stephanie Saul fits the mold of NY Times reporting on the drug industry like a glove: Big bad companies market lifestyle meds with horrible side effects when simple changes in how we live could easily take care of the problem. Instead of marketing erectile dysfunction drugs, the drug companies are pushing sleeping pills of all things.

Here’s the crux of her article, “… some experts worry that the drugs are being oversubscribed without enough regard to known, if rare, side effects or the implications of long-term use. And they fear doctors may be ignoring other conditions, like depression, that might be the cause of sleeplessness.”

Rare side effects and implications of long-term use? What are the side effects? Sleepiness? That is fairly standard and well known. How do we know they are being over “subscribed?” Saul gives us no benchmark since there is nothing in the article about the extent of sleeping disorders in the United States. Along those line, her claim that experts fear doctors are ignoring related conditions flies in the face of a campaign to make doctors aware that sleep disorders are a component of other illnesses. If she had done a little research, just five minutes of Googling, she could have found that out.

But she doesn’t give us context because Saul wants us to believe sleeplessness was a problem invented by drug companies to sell a useless product. Has she read anything about the increased risk for stroke and sleep apnea? What about the studies reporting up to 80 percent of people on dialysis suffer from insomnia? The fact that most primary care doctors have failed to treat sleeping disorders among people suffering from mental illness, arthritis and fibromyalgia?

A good article would have provided context, history, risks and benefits. As it stands, it is another example of the rotting standards of medical journalism that dwells on the fear factor.

FDA released its Fiscal Year 2007 budget request to Congress totaling $1.95 billion, a 3.8 percent increase over FY 2006.

Nearly $6,000,000 ($5,940,000 to be precise) is new funding for the Critical Path Initiative. This is the first time Critical Path funding has been included in the Administration’s proposed budget.

It’s money well spent and I certainly hope members of Congress embrace this as an important initial investment in the future of American health care.

An initial investment.