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This Week: House Republican Working Group Outlines King v. Burwell“Off-Ramp” Plan… FDA Approves First Biosimilar Product… CMS Approves NHMedicaid Expansion Waiver
House of Representatives
- House Republican Working Group Outlines King v. Burwell “Off-Ramp” Plan
- House Appropriations Subcommittee Holds Hearing on FDA Budget
- Bipartisan Legislation Introduced to End Surprise Charges for Medicare Beneficiary Colonoscopies
- Bipartisan Bill Introduced to Require Part D Plans to Include Six Protected Categories of Drugs
- Upcoming: HELP Committee Hearing on Medical Innovation for Patients
- FDA Updates Guidance Agenda to Include Generic Opioids
- FDA Approves First Biosimilar Product
- FDA Launches Drug Shortages Mobile App
- CMS Announces Release of 2015 Impact Assessment of Quality Measures Report
- MedPAC March 2015 Public Meeting
- CDC Announces New Director of Public Health Preparedness and Response
3. State Activities
4. Regulations Open for Comment
- FDA Solicits Comments on New Methodologies for Generic Drug Clinical Studies
- FDA Reopens Comment Period for Generic Drug Labeling Rule
- FDA Releases Five Draft Guidance Documents on Drug Compounding
- FDA Releases Draft to Streamline Experimental Drug Applications
- FDA Reopens Comment Period for Certain Provisions within Generic Drug User Fee Amendments
- Healthcare.gov: CMS Has Taken Steps to Address Problems, But Needs to Further Implement Systems’ Development Best Practices
- Drug Shortages: Better Management of the Quota Process for Controlled Substances Needed; Coordination Between DEA and FDA Should Be Improved
The House Republican Health Care Working Group hasreleased an outline of policies they would support if the Supreme Court rules this summer that subsidiesprovided to individuals in federally facilitated health insurance marketplaces are illegal. The outline centers on allowing states to “opt-out” of many ofthe ACA’s requirements. However, the plan would retain several popular components of the ACA, including protections for individuals with pre-existingconditions, and extending dependent coverage to children up to age 26. In addition, the outline contains traditional Republican policies, such as allowingfor purchase of health insurance across state lines, expansion of health savings accounts (HSAs),permitting small businesses and other groups to band together to increasetheir bargaining power with insurers, and bolstering the use of high-riskinsurance pools for individuals with costly medical conditions.
On March 4 the House Appropriations Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies held a hearing todiscuss funding priorities within the President’s FY 2016 budget for the Food and Drug Administration (FDA). The Obama administration’s budget proposal,released Feb. 2, seeks $2.7 billion in budget authority and $4.9 billion in total resources for the FDA. The overall FY 2016 request is $425 million, or 9percent, above the FY 2015 levels, according to the proposal. This increase consists of $148 million in budget authority and $277 million in user fees.Appropriations Committee Chairman Hal Rogers (R-KY) said the President’s ask for a nearly $150 million increase in budget authority for FDA “will be toughto swallow.” Most of the President’s request for additional FDA funding is slated to go toward food safety as the agency gears up to begin implementing andenforcing the 2011 Food Safety Modernization Act (FSMA), with $109.5 million in budget authority and $191.8 million in requested new food user feesallocated for food safety efforts. Outgoing FDA Commissioner Margaret Hamburg made her last appearance before the committee since she announced she wasstepping down from her position at the end of the month. Other topics touched on during the hearing were the recommendations of the 2015 Dietary GuidelinesAdvisory Committee and a discussion of whether FDA should be given the authority to evaluate dietary supplements at a higher standard due to concerns aboutcontaminants and false and misleading claims.
Dr. Margaret Hamburg
Commissioner, Food and Drug Administration
Chief Financial Officer, Food and Drug Administration
Director, Office of Budget
Assistant Secretary for Financial Resources
For more information or to watch the hearing, please visitappropriations.house.gov.
Sens. Sherrod Brown (D-OH), Roger Wicker (R-MS), Ben Cardin (D-MD) and Susan Collins (R-ME) introduced legislation March 3 that would remove barriers tolifesaving colorectal cancer screenings and treatment. The Removing Barriers to Colorectal Cancer Screening Act, S.624, would revise a Medicare policy thatcurrently charges seniors for colonoscopies when a polyp is removed even though cancer screenings are promoted as a free service under Medicare. The billwould eliminate cost-sharing under Medicare for preventive screening colonoscopies, even if a polyp or tissue is removed. As two-thirds of colorectalcancers occur in Medicare-aged individuals, reducing barriers to screenings will improve care and health outcomes while saving money for both seniors andthe Medicare program. “Preventive care like colonoscopies helps save lives and keep Americans healthy — so we must do everything possible to encouragepatients to take advantage of these procedures,” said Sen. Brown in apress release. “Unfortunately, under current policy — Medicare beneficiaries can be surprised by added costs following a colonoscopy. With a quick fix to current law,we can make sure seniors have truly free access to life-saving colorectal cancer screenings.” U.S. Reps. Charlie Dent (R-15-PA), Donald M. Payne, Jr.(D-NJ), Michael Fitzpatrick (R-PA) and Joe Courtney (D-CT) also introduced a companion bill, H.R.1220, in the House of Representatives.
Sens. Chuck Grassley (R-IA) and Sherrod Brown (D-OH) introduced legislation March 5 to require that Medicare prescription drug plans, Part D, carry sixcategories of prescription drugs offered to participating beneficiaries. The legislation comes after the Centers for Medicare and Medicaid Services (CMS)tried to limit the categories by regulation. The Grassley-Brown legislation would require that Medicare Part D sponsors include all covered drugs in sixprotected categories and classes in their plan formularies: Anticonvulsants, antidepressants, antineoplastics, antipsychotics, antiretrovirals andimmunosuppressants for the treatment of transplant rejection. The legislation would allow the Secretary of Health and Human Services (HHS) to use theformal regulatory process, including public notice and a comment period, to establish exceptions that would allow Part D plan sponsors to exclude a drug inone of the six protected classes or to limit access to a protected drug through utilization controls such as requiring prior authorization. The legislationwould take effect beginning in plan year 2015. In January 2014, CMS issued proposed rules that would have narrowed the protected classes toanticonvulsants, antiretrovirals and antineoplastics, beginning in plan year 2015. Antipsychotic drugs would have continued to be treated as a class ofclinical concern in 2015 and until CMS determined that it was appropriate to change the criteria for these products. In May 2014, CMS announced it wouldnot finalize the proposed regulations relating to the six protected classes.
More information on the legislation can be found in the accompanyingpress release.
On March 10, the Senate HELP Committee will hold a hearing entitled “Continuing America’s Leadership in Medical Innovation for Patients.” The hearing willrepresent the committee’s first official action on its version of the House Energy and Commerce Committee’s “21st Century Cures” initiative. HELP CommitteeChairman Alexander (R-TN), along with Sen. Burr (R-NC) released a white paper earlier this year seeking feedback from stakeholders on ways to speed up drugand medical device development, possibly by revamping FDA’s clinical trial and review policies. HELP Committee Ranking Democrat Patty Murray (WA) isworking with Alexander on the effort.
Francis Collins, M.D., Ph.D.
National Institutes of Health (NIH)
Margaret Hamburg, M.D.
Food and Drug Administration (FDA)
For more information, or to view the hearing, please visitwww.help.senate.gov.
In anupdated draft guidance agenda documentreleased Feb. 27, the Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) announced its intention to include a generic drugguidance document for evaluating abuse-deterrent properties of generic opioid drug products on its roster list for 2015. FDA last month testified beforecongressional appropriators that it is working on testing protocols and evaluation standards for generic opioids with abuse-deterrent properties, and plansto publish guidance in the near future. As it currently stands, after an opioid is approved, its manufacturer often works to improve its abuse-deterrentqualities to make it harder to misuse and abuse. However, these new qualities do not necessarily extend the original drug’s patent protection or marketingexclusivity. As a result, FDA may subsequently approve drugs generic to the original, i.e., a non-abuse-deterrent opioid drug. FDA has defended thispractice by saying it is up to manufacturers to prove that their new abuse-resistance measures actually live up to their claims and deter abuse, and thatwithout such data it is under no obligation to prevent generic competition. “Thus far, four opioid product labels have been approved with abuse deterrenceclaims consistent with the 2013 draft guidance. FDA is in the process of revising and finalizing this guidance. However, the fact that this guidance is notyet final has not and will not prevent FDA from evaluating proposals to include abuse-deterrence language in the labeling of specific products,” said theagency in its 2016 budget justification document. A date for the release of the guidance document was not provided.
On March 6, the Food and Drug Administration (FDA) announced the approval of Zarxio (filgrastim-sndz), the first biosimilar product approved in the UnitedStates. A biosimilar product is a biological product that is approved based on a showing that it is highly similar to an already-approved biologicalproduct, known as a reference product. Only minor differences in clinically inactive components are allowable in biosimilar products. A biosimilar productcan be approved by the FDA only if it has the same mechanism(s) of action, route(s) of administration, dosage form(s) and strength(s) as the referenceproduct, and only for the indication(s) and condition(s) of use that have been approved for the reference product. Sandoz, Inc.’s Zarxio is biosimilar toAmgen Inc.’s Neupogen (filgrastim), which was originally licensed in 1991. Zarxio is approved for the same indications as Neupogen, and can be prescribedby a provider for patients with cancer receiving myelosuppressive chemotherapy, patients with acute myeloid leukemia receiving induction or consolidationchemotherapy, patients with cancer undergoing bone marrow transplantation, patients undergoing autologous peripheral blood progenitor cell collection andtherapy, and patients with severe chronic neutropenia. “Biosimilars will provide access to important therapies for patients who need them,” said FDACommissioner Margaret A. Hamburg, M.D. “Patients and the health care community can be confident that biosimilar products approved by the FDA meet theagency’s rigorous safety, efficacy and quality standards.” FDA’s approval comes five years after the Biologics Price Competition and Innovation Act becamelaw as part of the Affordable Care Act (ACA), ushering in a pathway for the FDA to review applications from drugmakers to sell biosimilars.
On March 4, the U.S. Food and Drug Administration (FDA) launched theagency’sfirst mobile application (app) specifically designed to speed publicaccess to valuable information about drug shortages. The app identifiescurrent drug shortages, resolved shortages and discontinuations of drugproducts. Drugs in short supply can delay or deny needed care for patients.Drug shortages may also lead health care professionals to rely onalternative drug products, which may be less effective or associated withhigher risks than the drug in shortage. “The FDA understands that healthcare professionals and pharmacists need real-time information about drugshortages to make treatment decisions,” said Valerie Jensen, associatedirector of the Drug Shortage Staff in the FDA’s Center for Drug Evaluationand Research. “The new mobile app is an innovative tool that will offereasier and faster access to important drug shortage information.” App userscan search or browse by a drug’s generic name or active ingredient, andbrowse by therapeutic category. The app can also be used to report asuspected drug shortage or supply issue to the FDA.
On March 2, CMS released the2015 National Impact Assessment of Quality Measures Report (2015 ImpactReport). The 2015 Impact Report demonstrates that the nation has made clearprogress in improving the health care delivery system to achieve the threeaims of better care, smarter spending and healthier people. This report is acomprehensive assessment of quality measures used by CMS. The reportsummarizes key findings from CMS quality measurement efforts and recommendednext steps to improve on these efforts. Specifically, the report outlinesthe performance on quality measures over time and improvements achieved.Findings from the report include research on 25 CMS quality programs andhundreds of quality measures from 2006 to 2013 and builds on the prior 2012Impact Assessment Report. Many of these measures are also included inincentive programs that link payment to quality performance.
On March 5 and 6, the Medicare Payment Advisory Commission (MedPAC) met to discuss Medicare payment policy issues, including upcoming recommendations thecommission will issue to Congress later in March. While no recommendations were made on the 340B Outpatient Drug Program, the Commission recommended withregard to the hospital short-stay program that Congress revise the SNF eligibility requirement for formally admitted patients, so that time spent inoutpatient observation status also counts as part of their three-day prior hospitalization requirement; require that all acute-care hospitals notifyMedicare beneficiaries in outpatient observation status for more than 24 hours that being under observation may impact their cost-sharing for their currenthospital visit as well as their eligibility for SNF coverage; and include payment for self-administered drugs for patients under observation on abudget-neutral basis within the hospital outpatient prospective payment system. A full list of topics covered by the meeting includes:
- Hospital short-stay policy issues
- Part B drug payment policy issues
- Synchronizing Medicare policy across payment models for determining beneficiary premiums
- Generic prices and the role of non-preferred generic tiers in Part D
- Risk sharing in Medicare Part D
For more information, please visitwww.medpac.gov.
Last week, CDC named Stephen C. Redd, M.D.,the new director of CDC’s Office of Public Health Preparedness and Response (OPHPR). Dr. Redd is a Rear Admiraland Assistant to the Surgeon General in the United States Public Health Service. He comes to OPHPR from CDC’s Influenza Coordination Division and bringswith him the expertise and experience of working at CDC for nearly 30 years and serving 29 years in the U.S. Public Health Service Commissioned Corps. Inhis new role, Dr. Redd will be leading OPHPR, which is CDC’s principle coordinator of all preparedness and response activities. OPHPRprovides strategic direction, support and coordination for activities acrossCDC and with local, state, tribal, national, territorial and internationalpublic health partners.
3. State Activities
On March 5, New Hampshire has become the sixth state to get permission to implement its own style of Medicaid expansion after the Centers for Medicare& Medicaid Services (CMS) approved the state’s Medicaid expansion waiver. The waiver allows the state to buy private insurance for residents withincomes up to 138 percent of the federal poverty level, or about $15,900. Gov. Maggie Hassan (D) last year signed into law a bill that expanded Medicaidand created a temporary bridge program that allowed beneficiaries to go into the state’s managed Medicaid plans. Under state law, had the mandatory premiumassistance program waiver not been approved by March 31 the managed care “bridge program” would have ended. With the new waiver, the state will beginpurchasing private insurance for individuals or subsidizing existing employer coverage once the next open enrollment period begins Nov. 15, 2015, withcoverage kicking in Jan. 1, 2016. “Federal approval of this waiver is another important step forward for the New Hampshire Health Protection Program, whichwill help it increase the positive impact that it is already having on our people and our economy,” said Governor Hassan in apress release. “Our bipartisan health care expansion plan is auniquely New Hampshire solution that continues to make progress, and I look forward to continuing to work with the legislature to maintain our commitmentto our people, businesses, and the future of our economy through this critical program.” As of March 3, 36,404 people had enrolled as part of expansion,according to the state.
A bill to allow terminally ill patients to use experimental drugs and devices that have yet to receive full approval from regulators is on its way to Gov.Gary Herbert for his signature. The House voted 69-3 on Monday to approve Senate amendments to HB 94, providing final approval by lawmakers. The Senateearlier approved it 26-0, and the House also had earlier endorsed it on a 72-1 vote. The bill by Rep. Gage Froerer,R-Huntsville, would allow patients, in partnership with their doctors, toapproach drug companies directly and request treatments that have passed thefirst phase of the Food and Drug Administration’s (FDA) approval process,showing they are safe for human trials. Patients may face paying the fullcost of the drug.
4. Regulations Open for Comment
In anotice released March 5 by the Federal Register, the Food and DrugAdministration (FDA) is seeking feedback from stakeholders on possible newmethodologies for generic drug clinical studies and ways to demonstratebioequivalence as part of its regulatory science priorities for 2016 underGeneric Drug User Fee Amendments (GDUFA). FDA noted it will take feedbackfrom stakeholders into account when creating next year’s regulatory scienceplan for generic drugs. A public hearing has been scheduled for June 5,where FDA plans to hear from stakeholders on six specific areas includingscientific or technical advancements that would help that currently limitgenerics’ availability, innovative approaches to preapproval development ofgeneric drugs, advancements in scientific approaches to evaluate therapeuticequivalence of generic drugs through later stages of their lifecycle. Theagency’s efforts come as House and Senate lawmakers are also looking at waysto revamp clinical trial design, though up to now they haven’t focused ongeneric drug-specific issues, and identification of high-impact publichealth issues involving generic drugs, among others. The notice comes as theHouse of Representatives 21st Century Cures discussion legislative draftalso seeks to revamp clinical trials by allowing trial sponsors to proposeincorporating adaptive trial designs for alternative statistical methodsinto proposed clinical trials and streamlining the institutional reviewboard.
In anannouncement Feb. 17, the Food andDrug Administration (FDA) revealed that it has formally re-opened the comment period for a controversial generic drug labeling proposed rule and will holda public meeting next month to address concerns with the rule and possible alternatives. The rule, which FDA proposed in 2013, would allow generic drugmakers to unilaterally update safety information and would require generic drugmakers to modify their labels independently of their brand-namecounterparts, something that only brand-name drugmakers can currently do before receiving agency permission. The FDA proposed the rule in response to a2011 U.S. Supreme Court decision that federal law does not permit generic drugmakers to make such changes independently and, therefore, they should not beheld accountable for a failure to warn against a risk. Stakeholders will have until April 27 to comment on the proposed rule; the agency’s public hearingto receive more input from stakeholders will be held on March 27 from 8 a.m. to 5 p.m. at FDA’s White Oak campus.
On Feb. 13, U.S. Food and Drug Administration (FDA) issued fivedraft guidance documents related to drug compounding and repackagingthat will help entities comply with important public health provisions; guidance will be applicable to pharmacies, federal facilities, outsourcingfacilities and physicians and comes as an outcrop of the Drug Quality and Security Act (DQSA), enacted by Congress in November 2013, in response to adeadly fungal meningitis outbreak that was linked to contaminated sterile compounded drug products. Specifically, the documents include potential directionon outsourcing facility registration, outsourcing facility adverse event reporting, drug repackaging, mixing, diluting and repackaging biological products,and a draft Memorandum of Understanding (MOU) with the states. The draft guidance documents are available for public comment until May 14, while draftcomment for the draft MOU is open until June 13.
On Feb. 4, the Food and Drug Administration (FDA) releaseddraft guidance, entitled IndividualPatient Expanded Access Applications: Form 3926, for a new, shorter application for patient access to experimental drugs. The draft comes in response toconcerns that the existing process for “compassionate use” for experimental drug applications was too arduous. In the guidance, FDA says the newly proposedform would take doctors 45 minutes to complete whereas the existing form is estimated to take 100 minutes. Under the old system, FDA required that a “coversheet” be included with any IND submission, known as Form 1571. However, that form was originally intended to be used by companies involved in drugdevelopment, not physicians, who submit the vast majority of expanded access requests. FDA said it was “concerned” that some physicians might notunderstand how to complete that cover sheet “and associated documents because it is not tailored to requests for individual patient expanded access.” PeterLaurie, FDA’s associate commissioner for public health strategy and analysis, said the changes would greatly simplify the compassionate use process. Theold form “called for 26 separate types of information and seven attachments,” he noted. “The new form calls for a small fraction of that. The new draftform, when finalized, will require only eight elements of information and a single attachment.” The changes announced by the agency are expected to affecta significant number of patients each year; in 2014, FDA processed 1,758 single patient investigational new drug applications and emergency investigationalnew drug applications—97 percent of all expanded access requests. Comments and suggestions for the draft document should be submitted by April 13, 2015.
On Feb. 6, the Food and Drug Administration (FDA) posted aFederal Register notice reopening the public docket to solicit commentson certain topics related to Generic Drug User Fee Amendments of 2012 (GDUFA) implementation and the GDUFA Commitment Letter that accompanies thelegislation. FDA will reopen the comment period to the public docket associated with the Sept. 17, 2014, GDUFA Public Hearing on Policy Development for anadditional 30 days. Specifically, the agency has requested public input on the five draft guidance documents that were issued to facilitate implementationof GDUFA and on future policy priorities including recommendations for additional guidance topics to facilitate GDUFA implementation. FDA also requestedfeedback on issues that may arise in consideration of 180-day exclusivity provided for by paragraph IV patent certifications. Finally, FDA requestedfeedback on the specific criteria FDA should apply to identify an abbreviated new drug application (ANDA) as a first generic eligible for expedited ANDAreview. FDA will take the information presented at the public hearing and in comments to the docket into account when developing the fiscal year 2015 GDUFApriorities. Interested persons may submit either electronic comments regarding this document towww.regulations.gov or written comments to the Division of Dockets Management by close of businessMarch 9.