Latest Drugwonks' Blog
BioCentuty reports:
House moves closer to Senate on PDUFA
The U.S. House Energy and Commerce Committee posted a new draft PDUFA reauthorization bill that is more closely aligned with the Senate PDUFA bill than previous versions. Changes that reflect the Senate bill include dropping sections that would add jobs creation and economic development language to FDA's mission and adding a section that would create an expedited development pathway for breakthrough drugs.
However, several items in the House draft bill are still not in the pending Senate version, including provisions to increase public participation in crafting guidance documents and creation of a new priority review voucher system to encourage development of therapies for rare pediatric cancers. The House draft also requires that FDA provide Congress a 60-day notice prior to issuing guidance on the regulation of laboratory-developed tests. The Energy and Commerce Committee's health subcommittee is scheduled to meet Tuesday (today) to deliberate on the draft bill.
Per today’s Washington Times, “Health insurers gave a tentative thumbs-up Monday to the Food and Drug Administration’s proposal to make drugs treating chronic conditions available without a prescription by classifying them in an all-new category.”
Insurance companies cautiously support the idea but warned Monday in a letter to the FDA that a host of complications could arise.
Without the right safeguards in place, patients could obtain drugs they don’t need and it’s unclear who would be held liable if they do, said Robert Zirkelbach, spokesman for America’s Health Insurance Plans.
Insurance companies also would have to figure out whether and how they would cover drugs that fall into the new category.
“Expanding access is something that we support, but these other issues would have to be addressed for this to work if they decide to move forward with that,” Mr. Zirkelbach said in an interview.
If the FDA decides to move ahead with the plan, it would create a third category for classifying drugs.
Called “safe use” drugs, patients wouldn’t need a prescription but neither could they obtain them over the counter. Instead, people could only buy the drugs after diagnosing their ailments online or in the pharmacy.
Seeking a way to expand access to drugs for Americans who struggle with common conditions such as high cholesterol, migraines and diabetes, the FDA has raised the idea several times over the past decade and brought it up again in March, asking the public to weigh in.
It’s uncertain whether the agency will sign off on the new policy since the idea has faltered in the past under opposition by doctors and other medical providers. The American Medical Association has said it could open the door to drug misuse and expects that out-of-pocket costs will rise for patients.
When it comes to addressing and solving the drug shortages, what are the most important variables to consider?
According to Healthcare Intermediaries: Competition and Healthcare Policy at Loggerheads? -- a new white paper by the American Antitrust Institute, a key question “is whether high levels of consolidation in intermediary markets and potentially exclusionary conduct have caused or exacerbated shortages.”
“One major protection against shortages is a stable supply chain, which is largely determined by the number and diversity of suppliers. The concept of supply chain “fragility” is increasingly relevant in operations research, marketing, economics, and even sociology. Supply chains featuring only a few competitors and high entry barriers at critical junctures are excessively exposed to the risk of disruption and collapse following an exogenous shock … but a fragile supply chain can also be inefficient when it “fails” because of excessive consolidation that leaves few suppliers.”
Intermediary conduct can, “threaten to impair the achievement of healthcare policy goals such as affordable healthcare, choice in medical products, a stable supply chain, and diversity of supply.”
The paper points out that sterile injectables. In 2010, 60 percent of sterile injectables (which accounted for 80 percent of the drugs in shortage) were sole-sourced. Markets for specific drugs are likely to be even more concentrated because only one or two firms produce them. Markets for generic drugs – which accounted for 60 percent of sterile injectables and 50 percent of all shortages – are also concentrated. In 2010, the top three firms accounted for about 70 percent of the generic sterile injectable market and 90 percent of the generic sterile injectable oncology segment of the market.
Indeed, the FDA notes that while demand in the generic and oncology segment of the market is robust, the supply system is “vulnerable to drug shortages because a large supply disruption is difficult to make up with alternative suppliers.” This is compounded by low demand and supply elasticities for certain drugs, stringent product manufacturing quality controls, dedicated production lines, and “just-in-time” manufacturing and inventorying practices.
Among other suggested next steps, the paper recommends that regulatory initiatives designed to address drug shortages “should focus less on reporting requirements and more on the analysis of competition in intermediary markets and upstream markets for drugs, and medical devices and supplies.”
A paper worth reading with conclusions worth debating.
" The division shall have the power to design and to revise, consistent with this chapter, a basic schedule of health care services that enrollees in any health insurance program implemented by the division shall be eligible to receive. Such covered services shall include those which typically are included in employer-sponsored health benefit plans in the commonwealth. The division may promulgate schedules of covered health care services which differ from the basic schedule and which apply to specific classes of enrollees. The division may promulgate a schedule of premium contributions, co-payments, co-insurance, and deductibles for said programs, including reduced premiums based on a sliding fee, and other fees and revise them from time to time, subject to the approval of the division of insurance; and provided, however, that such schedule shall provide for such enrollees to pay one hundred per cent of such premium contributions if their income substantially exceeds the non-farm poverty guidelines of the United States office of management and budget."
Compared to this bill, Obamacare is free market reform.
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.
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.
