Pfizer spokeswoman Gwen Fischer told Pharm Exec that the deal came together in a matter of weeks, and that FoldRx will be fully incorporated into Pfizer’s research division. All of FoldRx’s employees are expected to become Pfizer employees when the deal is completed.

FoldRx made its name—literally—by discovering compounds that treat diseases related to protein misfolding, using a proprietary yeast-based target discovery platform. Its pipeline leader is tafamidis meglumine, a treatment for TTR amyloid polyneuropathy, which is a genetic neurodegenerative disease.

“Over the past five years the FoldRx team has successfully developed tafamidis from the bench stage to MAA submission,” stated Richard Labaudiniere, president and CEO of FoldRx, in a release. “Pfizer’s strong clinical and regulatory resources, global marketing reach, and commitment to the treatment of rare diseases will significantly enhance the ability to pursue the goal of efficiently bringing tafamidis to all patients affected by this devastating neurodegenerative disease.”

According to Pfizer, the deal is a reflection of the company’s new business unit structure. The original intent of the business unit was to target specialized expertise in specific research areas.

“This particular transaction will add another important component to the specialty care business unit and investigation medicines for rare and orphan diseases,” Fischer said. “The company also compliments our neurosciences disease area, which is currently a part of Pfizer’s specialty care.”

Earlier this year, Pfizer created an orphan and genetic disease research unit charged with discovering diseases affecting fewer than 200,000 patients in the United States and fewer than 10,000 patients in the European Union.

Pfizer has been upfront with the general public in stating that one of the key ways it will drive growth in the next few years will be through strategic business development, focusing on small to mid-size business deals such as the acquisition of FoldRX.

In the last two years, FDA has twice cited Baxter for similar violations. In April 2009, it sent the company a warning letter indicating that select promotional materials created in support of Tisseel (a fibrin sealant) were false or misleading. In July 2008, the Agency’s Office of Compliance and Biologics Quality (OCBQ) issued an untitled letter to the company over claims about its anti-inhibitor coagulant complex, Feiba VH. The letters are not legally binding, but FDA can follow them up with litigation if they are ignored.

FDA told Baxter in the latest letter that it is “very concerned by your continued violative promotion of your products.” The agency requested that Baxter cease dissemination of the Araplast brochure and respond in writing about how it intends to bring its promotional material in line with the Federal Food, Drug, and Cosmetic Act.

Baxter reports that it has since sent a letter of response to FDA and put its promotional material for Araplast on hold. In a statement issued Aug. 31, the company said it “has confidence in the strength of its promotional review policies and procedures and a history of collaborating with FDA. [Baxter] will continue to work with FDA to ensure the company is in compliance with all relevant rules and regulations.”

“Without exception, each member of the Genzyme board believes this is not the right time to sell the company, because your opportunistic takeover proposal does not begin to recognize the significant progress under way to rectify our manufacturing challenges or the potential for our new product pipeline,” Genzyme CEO Henri Termeer wrote.

The letter goes on to state that the company outlined a laundry list of improvements it plans to make in its manufacturing facilities—an area that Genzyme has been chastised for recently—as well as future earnings from its much lauded multiple sclerosis treatment alemtuzumab, currently in development.

The board feels that Sanofi is low-balling the company with its $69-per-share offer, and should be willing to boost the price since Sanofi has openly talked about how much they are willing to pay for mergers and acquisitions as recently as last year.

According to analysts interviewed by Bloomberg, Sanofi should expect to pay upwards of $77 per share for Genzyme, but might not bump up the offer until its had time to thoroughly examine Genzyme’s financials.

Sanofi CEO Chris Viehbacher originally offered the same price for Genzyme in early July when the genetic firm’s stock hovered around $50.

“Notwithstanding this information and assistance, you have not increased your price above $69 per share,” Termeer stated. “You and your advisors claim you are willing to pay more but that you are unwilling to ‘bid against yourself.’ The Genzyme board is not prepared to engage in merger negotiations with Sanofi based upon an opportunistic proposal with an unrealistic starting price that dramatically undervalues our company.”

This is the first time AARP has measured the retail price of Rx drugs. In the past, the lobby group only tracked the wholesale manufacturers’ price of generic, branded, and specialty drugs, which is traditionally considered to be much higher than the retail price.

The brand name pharma firms had criticized those reports for not taking into account rebates and couponing offered to pharmacies and hospitals.

“What we found was that the prices are still trending way up, and for the most part they are relatively similar increases that we received when we were measuring the manufacturer prices,” AARP spokesperson Jordan McNerney told Pharm Exec in an interview. “Those discounts and rebates aren’t making much of a difference on the bottom line as far as the consumer is concerned.”

The concern was that if the wholesale price of drugs was increasing, then the cost had to be passed along to the consumer.

Boehringer Ingelheim’s prostate drug Flomax saw the biggest jump in price, increasing nearly 25 percent. McNerney pointed out that the treatment’s price appears to have jumped as the drug prepared to go off patent.

In response, the pharmaceutical lobby group Pharmaceutical Research and Manufacturers of America (PhRMA), released a diatribe on its website, supporting drug companies for innovating new treatments. PhRMA also supported industry against catcalls from AARP and another study released at the meeting of the American Sociological Association that claimed that few of the new treatments released in recent years offer significant improvements to patients’ lives.

“AARP’s report is misleading because nearly half of the drugs on its top 25 brand-name drug list were filled as generics in the first part of 2010, but AARP counts these drugs as if they were brand-name drugs,” stated PhRMA Senior Vice President Rick Smith in a release. “The report calculates costs in this inaccurate way even though it acknowledges that brand-name drugs typically lose about 90 percent of their sales after going generic.  The result is an overstatement of consumers’ actual costs for these medicines and there is a tremendous disparity between AARP’s report and the numerous independent analyses showing drug costs growing slowly.”

McNerney explained that the patients most likely to feel the spike in price are those in the Medicare Part D “donut hole.” Once a patient reaches a certain threshold of drug treatment, they hit a cap where they have no coverage and must pay out of pocket.

“We are looking at Congress and industry to control these prices,” McNerney said. “We would love to see Medicare negotiate directly with drug companies so patients can get the best prices on drugs and we would like to see any legislation to get generic drugs on the market faster.”

Like it or not (and frankly, I don’t), stakeholders are here to stay in European pharmaceutical affairs. Don’t get me wrong. I’ve no objection to democratic engagement, or to giving people with a legitimate interest a legitimate voice. It’s the word I object to. Partly on aesthetic grounds, because it is an ugly coining. But much more because it is one of these words that has now been appropriated by everyone to mean exactly what they want it to mean, irrespective of what it means to anyone else. Consequently, despite its almost mandatory inclusion in every discussion of contemporary pharmaceutical affairs, it has largely lost its meaning.

The upcoming symposium of The Organization for Professionals in Regulatory Affairs (TOPRA) offers a fine example. One of the sessions, entitled ‘Better access to medicines in a changing regulatory environment,’ will look, says the programme, at “the important contribution of all stakeholders” in achieving better health outcomes and improved access. The session will doubtless provide a valuable investigation of some of the genuine challenges identified in the title. But why ‘stakeholders’?

In fact the programme goes on to spell out who TOPRA consider

s as the constituents of this group: “specifically patients, healthcare professionals, academia, pharmaceutical industry, EU institutions and regulatory agencies.” That seems a perfectly reasonable list of interested parties to debate the subject. But when it then indicates that the discussion will cover “the experiences of interaction between stakeholders and working parties at national and EU level,” the question immediately arises as to who sits on these working parties. Not stakeholders, since stakeholders are interacting with them. That — according to TOPRA’s own definition — would rule out patients, healthcare professionals, academia, pharmaceutical industry, EU institutions and regulatory agencies. So what looked like a useful exchange on an important subject is suddenly revealed as an exploration of what can only be phantom working parties consisting of people who have nothing at all to do with pharmaceuticals.

To offer only the most obvious example of the risks to the pharma industry from lazy use of words like ‘stakeholder,’ look at how the term is used by the European Public Health Alliance (EPHA) — an admirable, influential and respectable civil society organization, but no automatic supporter of the pharmaceutical industry. Its mission statement includes the pursuit of “European insti

tutions that are accountable and accessible, policy-making that is transparent and with real opportunities for stakeholder input.”

The stakeholders that EPHA are thinking of certainly do not include the pharmaceutical industry or EU institutions. For EPHA, and many of the other organizations that maintain cautious vigilance over the operations of the pharmaceutical industry, stakeholders are the public, or patients, or consumers, or organizations representing them — not, in other words, the uncritical allies of pharmaceutical companies.

So if the European pharmaceutical industry continues to use the term uncritically, it is at best muddling its message, and at worst playing into the hands of some of its most vociferous opponents. The point is not a simple question of semantics. If the pharmaceutical industry wants to win the battles for hearts and minds that it inevitably and constantly must fight across so many fronts, it is going to increase its chances by being more rigorous in its approach to debate — and it is going to decrease them by sloppy talk.  One stakeholder’s heaven may prove to be another stakeholder’s hell.

It used to be so easy. Patients listened to doctors, doctors listened to pharma and everyone was happy. There was even a time when payers listened to pharma, reimbursing their prices with very few questions asked. Now everyone is asking questions and no-one listens to anyone else.
No-one, that is, except pharma. Branded as the bad guy of healthcare, companies are nonetheless trying to learn the art of dialogue, enlisting medical liaison officers in place of reps, engaging social media experts to navigate the minefields of Facebook and YouTube, and creating whole new departments to become more conversant in health economics and patient reported outcomes.

On Facebook, where it can seem the entire world is engaged in conversation, pharma companies are seriously outcast.

But with every step they take in this new patient-centric world, their handicaps become more apparent. On Facebook, for example, where it can seem the entire world is engaged in conversation, pharma companies are seriously outcast. According to the Dose of Digital website, there are around 50 Facebook pages sponsored by pharma and healthcare companies but the vast majority of these don’t allow comments from the public, thereby scuppering any hope of dialogue before its even started.
Dose of Digital founder Jonathan Richman cites three reasons for pharma companies’ reticence to fully engage:
1. They don’t want people to post adverse events.
2. They don’t want people to post off-label information about their products.
3. They don’t want to deal with negative comments.

All good reasons but a fourth — incurring the FDA’s wrath — is arguably the most potent. This happened when the FDA sent a letter to Novartis on July 29, warning that using Facebook’s share button (ie, allowing dialogue) on its Tasigna page amounted to promotion of the leukemia drug. This should have been expected, because the regulations as they stand simply can’t handle sharing technology, where information about a drug can whizz round the net losing, at countless different junctures, the balance it must maintain in terms of risks and benefits.

Why all the talk about the benefits of social media when real dialogue is impossible until the rules change, if indeed they ever will?
So, why all the talk about the benefits of social media when real dialogue is impossible until the rules change, if indeed they ever will? One reason is the clear evidence that patients are the way forward and that they gather to talk about their conditions in ultra-convenient communities.
Patients are indeed the way forward.

In a recent international survey conducted by the Economist Intelligence Unit, the overwhelming conclusions were that patients are speaking up as never before and changing how healthcare systems work. The respondents, healthcare professionals and people from the life science industries, came from the US, the UK, Germany and India. Specifically, 52% of them said patients expect higher standards of care, 57% said they want more information about their treatment; 49% said they wanted more involvement in relevant decisions about their care; and 49% said they wanted access to the latest treatments.
But patients don’t exist exclusively on the web. Even when they choose to converse in patient communities, they usually do so on sites sponsored by patient groups, where their views and concerns are collated by people who are accessible by email, phone, even, dare I say it, in person.
Pharma could start its forays into meaningful dialogue with patients by engaging with these bodies. But the evidence suggests otherwise. Another international survey, this time conducted by PatientView into patient groups’ views of pharma, found that just 22 percent of the 665 respondent groups maintained routine contact with a pharma company.

Moreover, although many of these groups said they were opposed to working with pharma, many more told the survey they wanted to forge ties with industry but believed they were not sufficiently attractive to companies for them to be interested.
Whatever the reasons for this belief, patients are emerging as strong and natural allies of pharma and not only in terms of access to drugs. Call me old-fashioned, but given the rules effectively banning real dialogue on the web and the growing importance of patients, pharma could do a lot worse than simply picking up the phone and asking patient groups what their members want.

FDA on Monday announced that it was looking to withdraw approval for the low-blood-pressure treatment midodrine hydrochloride because the companies manufacturing the drug failed to provide data from post-approval studies.

The kicker: The drug was approved 14 years ago.

FDA gave midodrine hydrochloride—branded by Shire as ProAmatine and produced by a half-dozen generics firms—the green light in 1996 as part of the fast-track approval program designed to speed to market drugs for diseases with no current treatments.

The catch is that FDA requires post-market clinical trials to ensure that the drug is meeting risk/benefit endpoints. In other words, the regulatory body wanted to make sure no hiccups occurred with the treatment when it hit the general population.

In response, Shire—who acquired the drug when it bought Roberts Pharma in 1999—chose to withdraw the drug as of September 30. The drug firm made it clear that the withdrawal had nothing to do with any safety concerns. In addition, Shire stated that it had conducted post-market trials in conjunction with Roberts, but FDA felt the results were “inconclusive.”

According to a release by FDA, none of the companies selling the drug have provided any data to prove that the treatment is beneficial. That said, some 100,000 people were treated with the midodrine hydrochloride last year alone.

“We’ve worked continuously with the drug companies to obtain additional data showing the drug’s clinical benefits to patients,” stated Norman Stockbridge, director of the Division of Cardiovascular and Renal Drugs in the FDA’s Center for Drug Evaluation and Research. “Since the companies have not been able to provide evidence to confirm the drug’s benefit, the FDA is pursuing a withdrawal of the product.”

FDA stated that patients currently on midodrine hydrochloride should not stop taking the medication. Shire now has 15 days to respond to FDA’s inquiry and provide some data supporting the drug. Shire did not respond to calls as of Monday afternoon. In addition, it’s unclear as to why FDA waited so long to ask for more data and whether any adverse reactions have been reported pertaining to the drug.

A research team in Belgium discovered a new test that has proven successful at identifying patients that are developing Alzheimer’s disease, according to a report published in the Archives of Neurology on Tuesday.

The study took spinal fluid from patients with varying levels of cognitive memory and looked for a specific protein signature or biomarker that has been attributed to Alzheimer’s.

Researchers tested more than 300 seniors—some with the disease, some suffering with recall difficulty, and some with no sign of Alzheimer’s. The study proved that 90 percent of the patients already diagnosed with the disease had the particular protein characteristic. Seventy-two percent of patients with some memory problems tested positive for the protein, and only 32 percent of patients in the normal cognition group had the biomarker.

Even more fascinating were the results of a second test of deceased patients confirmed to have Alzheimer’s. In that study, 64 out of 68 patients tested positive for the protein.

Healthcare professionals hope that the test can be used to detect Alzheimer’s way before the disease takes hold. Presently, the only way physicians can prove that a patient has the disease is post-mortem, making it very hard to isolate and treat it. Instead, doctors diagnose the disease through process of elimination—not the most reassuring method.

The downside to the test is that the spinal tap used to capture the fluid is notoriously painful, so it’s doubtful that healthy adults would elect to undergo the procedure—especially if insurance doesn’t cover it. There is also a fear of depression due to early detection or a false positive. The fact is, there are no medicines on the market to stop or slow the disease, so treatment—at this time—is fairly futile.

For the pharma industry, the new test could help researchers isolate trial participants by those most likely to have the disease, rather than test a broad range of people with cognitive problems, many of which might not have Alzheimer’s.

“We have to go very early to patients who have just the beginnings of Alzheimer’s in their brains—those are the people we need to identify to test the treatments,” Stephen Ferris at New York University’s Alzheimer’s Disease Center told CBS News. “That’s why these spinal fluid tests are going to be extremely important over the next few years.”

Novartis got hit with an untitled letter from FDA’s Division of Drug Marketing, Advertising, and Communications (DDMAC) on July 30 (posted August 4) for including a “Share on Facebook” widget on its branded website for Tasigna. This the first known enforcement by FDA in regards to Facebook—the massively popular social network.

The agency argued that the trackback link that the button generates is in violation of its marketing code because the link represents the benefits of the drug and not the adverse reactions.

To clarify, “Facebook Share” widgets are plug-ins added to blogs or websites that allow visitors to a site to repost a link to that site onto their Facebook profiles. What appears on the user’s Facebook page (or Wall) is a short excerpt from the site, a link to that page, and usually a thumbnail photo generated automatically. 

According to Digitas Health, the letter concerned the content provided by Novartis to the user, not about comments or messages left by random Facebook users. A major concern by industry and regulators has been how to control user-generated content on social media sites. For example, should a pharma company be held responsible if a banner ad for a drug appears on a public online forum where adverse reactions to the drug are being discussed—even if it’s a third-party site not associated with the drug firm?

“FDA seems more explicitly to be endorsing the view that it will hold pharmaceutical companies accountable solely for the material they provide for use in social media venues, not for any comments made about that material,” Digitas stated in a release. “DDMAC applied the principle of holding pharmaceutical companies accountable for filing only those materials generated by the pharmaceutical company.”

Digitas suggests that pharma companies eschew using the brand name on sites with a Facebook Share widget enabled or simply avoid making claims about the drug’s effectiveness.

FDA stated that the information in the Facebook link was pulled from the website’s metadata description, which Novartis has full control over. Users cannot modify the message; only add additional comments below the link. Digitas recommends that pharma companies planning on including a Facebook Share button should submit the proposed shared content as well as the site’s metadata for FDA review prior to a site’s launch.

“This repeal effort was started by legislators concerned that the law is hurting the state’s economy,” explained PhRMA’s Senior Assistant General Counsel Marjorie Powell in a prepared statement. “And it is certainly true that it adds an extra level of administrative complexity for companies in the state. Pharmaceutical marketing is already effectively regulated by such federal government agencies as the Food and Drug Administration.”

The Massachusetts ban was part of the larger Health Care Cost and Quality Act. Although the bill is best known for extending health insurance almost universally in the state, it also created a new code of conduct for sales reps and established penalties for wayward reps of up to $5,000 per violation. It also created the Massachusetts Public Health Council, which has the power to create rules further limiting marketing practices.

Last year the Council passed new rules for both pharma and medical device companies, making Massachusetts the state with the most comprehensive marketing and disclosure code. The rules specifically mandate disclosure of fees, payments and other compensation to doctors; ban promotional items such as pens and mugs; and restrict meals to those provided at training or educational events. Payments for research and clinical trials, rebates and discounts, prescription drugs provided for patient use and demonstration units for charity care are exempt. All other payments of $50 or more must be disclosed.

Currently, Vermont and Maine have bans on all food service “gifts” to physicians. Minnesota limits gifts to under $50, while California, Louisiana, Maine, Massachusetts, West Virginia and the District of Columbia all require pharmaceutical and biotech companies to report marketing spending on physicians, but do not ban food service outright.