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But is this decline really due to the FDA’s ability to demand earlier information about potential shortages from manufacturers?
“I am both amazed and delighted to see the progress that’s been made, said Hamburg. Key word: “Amazed.”
While the FDA’s new authorities are both timely and important, there are many pieces to the drug shortages problem – not the least of which is that (when it comes to hospital injectables) 30% of manufacturing capacity is off-line due to FDA inspection issues. That’s a lot of capacity. In fact, according to the agency, 43% of reported potential shortages were due to manufacturing issues.
Greater cooperation between agency and manufacturers is required so that this gap can be corrected before problems arise. A 30% hole in manufacturing is more than a hint that something’s amiss on both sides.
Is it a victory when you are solving the problems you create?
Peggy Hamburg: Calendar Girl
Big hat tip to http://www.fdalawblog.net for the excellent reporting.
GAO Report Says That FDA Has Met Most PDUFA Performance Goals; Agency Plans to Take Steps to Address Lingering Stakeholder Concerns
A report released earlier this week by the Government Accountability Office (“GAO”) says that FDA has met most of the Agency’s PDUFA performance goals for priority and standard original NDA and BLA submissions and for priority and standard original efficacy supplements to approved NDAs and BLAs, although in each case FDA review times have increased slightly. The GAO’s analysis covers applications in the Fiscal Year 2000 to 2010 cohorts, as well as preliminary information for applications submitted in Fiscal Year 2011.
The GAO report was sent to Senators Richard Burr (R-NC) and Tom Coburn (R-OK). Both Senators have long criticized FDA, saying that the Agency‘s “regulatory malaise” harms patients and manufacturers. Senator Burr has also threatened to delay the passage of PDUFA and other user fee legislation unless FDA speeds up application approval times. In addition, Senator Burr was successful in getting an amendment added to the FDA appropriations bill passed last year that seeks to “improve the transparency and accountability of the FDA in order to encourage regulatory certainty and innovation on behalf of America’s patients.” That amendment requires the submission of information to Congress on, among other things,
- “the average number of calendar days that elapsed from the date that drug applications (including any supplements) were submitted to such Secretary under [FDC Act § 505] until the date that the drugs were approved under such section 505;” and
- “the average number of calendar days that elapsed from the date that [BLAs] (including any supplements) were submitted to such Secretary under [PHS Act § 351] until the date that the biological products were licensed under such section 351.”
The GAO’s analysis shows that except for Fiscal Year 2008, FDA met PDUFA goals in all of the Fiscal Year 2000 to 2010 cohorts. (FDA recently provided similar statistics in testimony before Congress.) Moreover, the GAO found that an average of 44% of all original NDAs and BLAs submitted to FDA in Fiscal Years 2000 to 2010 were approved during the first review cycle and 75% were ultimately approved. FDA and industry stakeholders the GAO interviewed suggested that FDA failed to meet Fiscal Year 2008 goals as a result of implementation of the Risk Evaluation and Mitigation Strategy (“REMS”) requirements added to the FDC Act by the 2007 FDA Amendments Act.
Although FDA met most PDUFA goals for the Fiscal Year 2000 to 2010 application cohorts (and is on track to meet Fiscal Year 2011 goals for the applications submitted in that cohort), the GAO’s analysis (reflected in the tables below) shows that average FDA review times (i.e., the time elapsed from when FDA received a submission until it issued an action letter) hve increased slightly from Fiscal Year 2000 through Fiscal Yeat 2010 for both priority and standard NDAs and BLAs and priority and standard original efficacy supplements to approved NDAs and BLAs.
With respect to Senator Burr’s request for the average number of calendar days that elapsed from the date of NDA or BLA (including supplement) submission to final FDA action, the GAO says that it was unable to calculate average FDA review times in any meaningful way because most cohorts were still open; that is, “fewer than 90 percent of submissions had received a final action such as approval, denial, or withdrawal.” Specifically, for priority original NDAs and BLAs, only four cohorts had at least 90% of submissions closed (Fiscal Years 2001, 2002, 2005, and 2006), and for standard original NDAs and BLAs, only one cohort had at least 90% of submissions closed (Fiscal Year 2002). For priority efficacy supplements, only four cohorts had at least 90% of submissions closed (Fiscal Years 2000, 2001, 2004, and 2007), and for standard efficacy supplements, only one cohort had at least 90% of submissions closed (Fiscal Year 2005).
Stakeholders the GAO interviewed identified some issues that they believe hamper the NDA and BLA approval process, including REMS implementation, the use of outside expertise for reviewing applications, insufficient communication between FDA and stakeholders, and a lack of predictability and consistency in FDA reviews. FDA commented in the Agency’s response to the GAO report that it is taking or has agreed to take steps (as part of PDUFA V) that may address these issues, including issuing new guidance, establishing new communication-related performance goals, training staff, and enhancing scientific decision making.
The American Recovery and Reinvestment Act (aka, “the stimulus package”) provided AHRQ with $29.5 million for a program on academic detailing and the “communication of CER results to physicians.”
One contract, for $11.7 million, went to Total Therapeutic Management (TTM) and is specifically intended for physician outreach and education.
(TTM is a company that focuses on chart abstraction, data mining, and physician and patient education for a predominantly commercial client base -- health plans, pharmacy benefit managers, employers, and pharmaceutical companies, etc.)
The goal of this contract is to integrate AHRQ’s comparative effectiveness research, products, and tools into clinical practice through 9,000 on-site, face-to-face visits with clinicians, nurses, health plan formularies, benefit managers, and other healthcare professionals.
I recently interviewed Barry Patel, the president of TTM. Here are some snippets from our conversation:
How will the government decide which doctors are to be visited? Will ‘‘high prescribers’’ of on-patent medicines be on a priority list?
TTM’s top priority is ‘‘high volume’’ practices across 150 Metropolitan Statistical Areas (MSAs). So, rather than focusing on offices with disproportionately high negative patient outcomes, the government is directing its efforts against those doctors who are high prescribers—which is a pretty good indicator about what government detailing is all about—decreasing cost rather than improving care.
When it comes to government detailing (at the taxpayers’ expense), what are the metrics for success?
According to Mr. Patel, the only metrics are whether or not a physician says the sessions have been useful and asks the detailer to come back to discuss other topics. In other words, the metrics are subjective and anecdotal -- not clinical.
Interestingly, Mr. Patel doesn’t even agree with either the term academic detailing or counter detailing. ‘‘We aren’t counter anything. We’re not there to undo anything. It’s not good versus bad. Our visits aren’t details, they’re the beginning of a process.’’ And, as far as ‘‘academic’’ goes, Mr. Patel uses that term because “that’s the phrase AHRQ uses and placed in the contract. Our people are patient-centered outcomes consultants, PCOCs.” And “his people” are largely pharmacists and nurses.
A former Merck employee, Patel likens his PCOCs more to pharmaceutical company Medical/Science Liaisons (MSLs) than field representatives. ‘‘They’re not discussing product-specific information, but the findings of comparative effectiveness studies.”
How does TTM schedule their appointments with targeted physicians?
According to Mr. Patel, when his ‘‘outreach experts’’ phone physicians to request appointments, the fact that the meeting will result in CME credits is always mentioned. Would a pharmaceutical company be permitted to offer such an enticement? Would such an offer be ‘‘sunshine-able’’ under state and federal guidelines? And, if so, why don’t government detailers have to share the details of their valued benefactions?
Interestingly, according to the Accreditation Council for Continuing Medical Education (ACCME), government is exonerated from having a commercial interest. (A commercial interest is any entity producing, marketing, re-selling, or distributing healthcare goods or services consumed by, or used on, patients.)
Our nation’s single largest payer, Uncle Sam, is not deemed to have a conflict of interest when it comes to designing and providing physician CME.
What’s wrong with this picture?
At a time when more government agencies have been created to regulate and redistribute economy activity, with a focus on finance, energy production and of course health care, it is useful to reflect upon how this fundamental shift in power and direction will shape our future. Most important in my mind is the way in which the administrative state is being used to create ideological no-fly zones for those who disagree or challenge the guiding principles of the agency and the professional experts who feed off it.
Bob Moffitt's lecture is an important contribution to this discourse. Harry Truman noted" Once a government is committed to the principle of silencing the voice of opposition, it has only one way to go, and that is down the path of increasingly repressive measures, until it becomes a source of terror to all its citizens and creates a country where everyone lives in fear.” Keep Truman's warning in mind as you read Moffitt's lecture.
http://www.heritage.org/research/reports/2012/03/why-congress-must-confront-the-administrative-state?query=Why+Congress+Must+Confront+the+Administrative+State
BioCentury reports:
IOM: FDA should consolidate benefit-risk data for each drug
The Institute of Medicine said FDA should consolidate benefit-risk information for approved drugs into publicly available repositories created for each product. In a report released Tuesday, IOM said the agency already collects much of the information needed but that it is spread across multiple records. The report recommended the creation of a publicly available document containing information for each product from its approval throughout its entire time on market, including safety issues, regulatory actions and any restrictions. FDA said in a statement it supports the general concept, but added that it would be "very challenging to implement this recommendation within our current resources without seriously compromising other critical regulatory activities."
IOM also noted in the report that there is no universal set of criteria to determine when FDA should require a postmarketing study to evaluate a drug's safety. However, the report noted that the agency should require additional post-marketing safety research when a drug's benefits or risks are particularly uncertain, including first-in-class drugs approved based on surrogate endpoints, drugs for which endpoints provide conflicting evidence about risk or drugs with a strong biological rationale for a particular side effect. IOM, which conducted the review at FDA's request, issued a preliminary report in 2010.
http://www.nejm.org/doi/full/10.1056/NEJMoa1109333?query=featured_home#t=articleBackground
The Treatment Options for Type 2 Diabetes in Adolescents and Youth (TODAY) study found that metformin and Avandia were superior to other treatments in controlling the disease in kids.
I have two questions: Will Steve Nissen admit he was wrong, again? Also, will doctors start to use existing diagnostics that identify those who do not respond to metformin, at least in kids. Isn't that choosing wisely?
Patient, diagnose thyself -- and then reach a little deeper into your wallet.
Physician, step aside.
Pharmacist, step up to the plate.
Maybe.
The FDA may soon permit Americans to obtain some drugs used to treat conditions such as high blood pressure and diabetes without obtaining a prescription.
The FDA says over-the-counter distribution would let patients get drugs for many common conditions without the time and expense of visiting a doctor, but medical providers call the change medically unsound and note that it also may mean that
Under the changes that the agency is considering, patients could diagnose their ailments by answering questions online or at a pharmacy kiosk in order to buy current prescription-only drugs for conditions such as high cholesterol, certain infections, migraine headaches, asthma or allergies.
By removing the prescription requirement from popular drugs, the Obama administration could ease financial pressures on the overburdened Medicare system by paying for fewer doctor visits and possibly opening the door to make seniors pay a larger share of the cost of their medications.
The change could have mixed results for non-Medicare patients. Although they may not have to visit a doctor as often, they could have to pay out more money for medications because most insurance companies don't cover over-the-counter drugs.
How will this impact patient compliance? How will this effect one condition masking another, more serious one?
How will this change the role of the pharmacist?
Comments on the FDA proposal are due by May 7.
“Cozy Deal” (New York Times, April, 29, 2012) argues,
“The best approach would be for the government to fully finance the F.D.A. That is unlikely to happen. So before it ratifies any new deal on user fees, Congress must ensure that patient safety is the first priority.”
Sure, but unlike the Gray Lady we must all live in the real world. User fees are the cost of predictability -- not just for innovators who wants to know if and when they can commence marking new products but also for patients and physicians who want to know if and when these new treatments will be available. Predictability is also an issue of how. And this rests on the ability of the FDA to create and communicate a thoughtful and patient-centered model of risk/benefit analysis. Thankfully this is a central part of the current reauthorization debate.
Talk about positive outcomes!
It pays to have friends on PCORI’s Methodology Committee. Five of the seven grantees selected for PCORI funding are directly affiliated with members of that panel. In dollar terms its even more lopsided. Of the $799,524 awarded, $721,940 went to institutions with direct representation on the conclave.
If those committee seats are pro bono somebody should ask -- Cui bono?
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.
