Latest Drugwonks' Blog
A judge in the U.S. District Court for the District of Columbia has dismissed KV Pharmaceuticals suit against the FDA over the company's preterm birth drug Makena hydroxyprogesterone caproate.
After developing an FDA approved version of a drug that was in danger of becoming obsolete, KV promptly – and without warning – decided to charge $1500 per dose. The company was rightly criticized for the sudden jump in price and did the right thing by cutting the retail price of the drug to $680 and offering the drug at a reduced price to a wide range of organizations.
And yet the FDA decided to allow pharmacists to continue compounding even though KV developed the drug to put an end to the risk associated with compounding products. (FYI -- The FDA is currently Investigating bacteria that sickened 19 people at Alabama hospitals and may have killed nine and has turned up at compounding pharmacy in Alabama. And there are many other examples.)
Is this a victory for the public health?
KV filed suit against the FDA, alleging that the agency's policy of non-enforcement for compounded hydroxyprogesterone caproate products violated KV's right to market exclusivity for Makena under the Orphan Drug Act.
No dice.
Judge Army Berman said KV's allegations relate to FDA's discretionary enforcement activities and are therefore "unreviewable claims." She said in her ruling that the "case is fundamentally an effort to get the court to direct and oversee the FDA's enforcement activities, and that it cannot do." We agree with Judge Berman -- but it's a dangerous precedent.
Last month, KV filed for Chapter 11 bankruptcy because it has been "unable to realize the full value" of Makena, citing FDA's lack of enforcement of the market exclusivity.
Is this a victory for pregnant women?
Certainly the FDA has the authority to ensure the availability of products and indirectly considers affordability. It did so when it kept generic asthma inhalers on the market for several years rather than forcing them off to comply with an EPA requirement to remove inhalers powered by CFCs. I was at the agency at the time and the issue of inner city access was hotly debated.
That was (IMHO) a considered and organized action based on much thought and many meetings with stakeholders. The KV action (IMHO) was a reaction to media hype and political pressure. The FDA even got involved in a little demagoguery when it cited the fact that the NIH had provided KV with support to conduct clinical trials as the agency’s reason to allow the compounding. Given that logic, should every drug developed in cooperation with the NIH or based on NIH research be denied market exclusivity?
If the White House mandates the sequestration of the FDA budget the hurt will be shouldered by the public health.
PDUFA dollars cover reviews – but what about all of the other non-user fee items the FDA has on its plate – like advancing regulatory science and combatting counterfeiting, and developing standards for biosimilars and enhancing the safety and security of our food supply – to name only a few?
Sequestration = Castration of the FDA's mission.
Less is not more.
Here we go again. Thomas J. Moore and Curt D. Furberg (in a new JAMA article) accuse the FDA of compromising safety for speed.
As usual, Janet Woodcock places the matter in the appropriate perspective. "I'd like to stress that where there are unmet medical needs, the public has told us they are willing to accept greater risks," Dr. Woodcock said. "The cancer community in particular says we haven't used accelerated approvals enough."
Here’s the truth -- there is no such thing as a "safe" drug. It's the patient who must understand the risks required to achieve the benefit. That’s why the patient voice must be heard during all phases of the regulatory review process.
The Observational Medical Outcomes Partnership (OMOP), a public-private research project aimed at evaluating and validating methods of using observational health care databases for active drug safety surveillance, is considering closer collaboration with FDA’s Mini Sentinel and the Patient-Centered Outcomes Research Institute.
The OMOP executive board proposes working with Mini Sentinel and PCORI in a tentative three-year plan posted recently on the project’s website for public comment.
The OMOP board proposes to “coordinate OMOP’s research with the research and practice of others in the fields of risk and effectiveness identification and interpretation.”
Mini Sentinel is a pilot project for FDA’s Sentinel Initiative, which is envisioned as a national post-market safety surveillance program drawing from a network of medical claims data and electronic health records.
PCORI was established by the Affordable Care Act to sponsor outcomes research, including comparative effectiveness research, for drugs and other medical treatments.
OMOP’s other proposed activities include developing a framework for incorporating observational data and stakeholder perspectives into decision making, as well as methods to enable active surveillance and risk identification for newly marketed medical products.
The problem is that, after this important joint action, we're still hungry for more.
The China State Food and Drug Administration (SFDA) has joined forces with the FDA to shut down 18 Chinese-language web sites selling illegal drugs.
According to China Daily's report on Tuesday, the Sino-U.S. collaboration was launched in June this year and targeted Web sites in the U.S. advertising counterfeit drugs and health food. The 18 sites take down were run and hosted by Chinese speakers and aimed for Chinese consumers including Chinese Americans around the world, said Christopher Hickey, director of the U.S. FDA's China office.
"We welcome and appreciate the role played by the China State Food and Drug Administration to initiate the campaign and hope to continue combined efforts in the global fight against counterfeit drugs," he added.
The Sino-US collaborations was also part of a national clampdown on fake and illegal drugs within mainland China, said SFDA spokesperson Wang Lianglan in the report.
The operation saw police seizing 205 million tablets designed to look like branded drugs for treating male impotence, hypertension, diabetes and cancer, it noted. Wang added the SFDA will step up operations in the rest of the year with other law enforcement agencies and government departments to curb counterfeit medicince.
The next phase will see them enhance inspection of clinical trial for drugs made in China and meant for export to the United States, Hickey said.
For all the talk about a predictable and appropriate FDA pathway for biosimilars, where’s the thinking about what happens after approval?
Since patient safety and product efficacy will be a justifiable cause of angst to wary physicians, how will biosimilars be detailed? And who will do the detailing? Will biosimilar manufacturers need to develop patient assistance programs? What about REMS issues (both IP and implementation)?
Can you say, “authorized biosimilars?”
(And what will Congress and the FTC have to say about that?)
I am an EMR geek who isn’t so thrilled with the direction of EMR. So what, I have been asked, would make EMR something that is really meaningful? What would be the things that would truly help, and not just make more hoops for me to jump through? A lot of this is not in the hands of the gods of meaningful use, but in the realm of the demons of reimbursement, but I will give it a try anyhow.
Read his list here.
The bad news is that solanezumab (Eli Lilly’s experimental Alzheimer's treatment) failed to meet its primary endpoints in two late-stage clinical trials in patients with mild to moderate levels of the disease. The good news is that investigators saw positive signs in some analyses of the studies.
According to Lilly, after combining the results from the total of 2,050 participants in both trials, it found statistically significant signs that the drug slowed memory loss in some patients, many with mild cases.
"We're encouraged by the signals we see here," said David Ricks, president of Lilly Bio-Medicines. He said the solanezumab findings validated the company's heavy investment in Alzheimer's treatments. Lilly plans to discuss the findings with regulators before determining how to proceed with the drug.
Happy talk or hopeful hypothesis? Generally, combining the data from the two trials—aren't considered conclusive but are used to generate hypotheses that require further study and validation.
Importantly, the results from the pooled solanezumab data are to the first from any late-stage Alzheimer’s studies to suggest a benefit from an anti-beta amyloid compound.
Full data from the studies, based on an independent analysis by an academic national research consortium called the Alzheimer's Disease Cooperative Study, will be presented in October at scientific conferences in Boston and Monte Carlo, the company said.
As the Wall Street journal reports, “Much will depend on how regulators interpret the data. Usually the U.S. Food and Drug Administration makes decisions based on how experimental therapies perform in meeting the primary goals of pivotal trials.”
The Journal continues, “In the case of solanezumab, the FDA will have to weigh the potentially limited cognitive benefits against the lack of effective treatments, experts said. The agency could ask Lilly to do additional studies exploring further whether solanezumab does indeed slow down Alzheimer's progression in certain patients.”
True – but an even more important issue is understanding – and rewarding – incremental innovation. “Limited cognitive benefits” is in the eyes of the beholder, but the impact is anything but limited when it comes to our nation’s healthcare wallet.
As the prevalence impact of Alzheimer’s grows, so does the cost to the nation.
One in eight people age 65 and older (13 percent) has Alzheimer’s disease. Nearly half of people age 85 and older (45 percent) have Alzheimer’s disease. Of those with Alzheimer’s disease, an estimated four percent are under age 65, six percent are 65 to 74, 44 percent are 75 to 84, and 46 percent are 85 or older.
If Alzheimer’s Disease was a tradable stock, the demographics of the Baby Boom generation would make it a “must buy.”
The direct and indirect costs of Alzheimer’s and other dementias amount to more than $148 billion annually, which is more than the annual sales of any retailer in the world excluding Wal-Mart.
Is there value in slowing the progression of Alzheimer’s Disease? Certainly.
According to a study in Health Affairs (“Alzheimer's disease care: costs and potential savings”), monthly savings of $2,029 in formal services are possible if disease progression can be slowed. Annual institutional cost savings of $9,132 also are achievable if alternative residential settings are used.
Sometimes the biggest success stories rest on the foundations of failure. The solanezumab trials may have missed their primary endpoints – but they have succeeded in advancing our knowledge of Alzheimer’s Disease and provided a possible new and innovative option for patient’s (and family members) of this draconian disease.
Alas, solanezumab will not be the magic bullet that eradicates Alzheimer’s Disease from our lexicon of suffering – but it may yet be an important addition to our pharmacological armamentarium.
As FDA and then CMS decide on the value of solanezumab, it’s important to remember that innovation is slow. As any medical scientist will tell you, there are few "Eureka!" moments in health research. Progress comes step-by-step, one incremental innovation at a time.
Harvard University health economist (and Obama healthcare advisor) David Cutler has noted: "Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost."
The battle for the heart and soul of 21st century health care is the battle over innovation. And nothing short of victory is acceptable.
To borrow an over-used adjective from the world of global climate change -- we must protect "sustainable" innovation.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
