FDA Seeking Comments on New Guidance Regarding Rare Diseases

The FDA has released a new draft guidance document titled, “Rare Diseases: Natural History Studies for Drug Development.” The guidance document impacts pharmacy supply chain stakeholders, especially drug manufacturers and specialty interests.

What Does the Guidance Do?

According to the FDA, the Agency is publishing this draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. A natural history study collects information about the natural history of a disease in the absence of an intervention, from the disease’s onset until either its resolution or the individual’s death. Although knowledge of a disease’s natural history can benefit drug development for many disorders and conditions, natural history information is usually not available or is incomplete for most rare diseases, therefore, natural history information is particularly needed for these diseases.

Why You Should Participate:

If you are supply chain stakeholder looking to diversify your business into biological products or to serve rare disease patients, the trend is pointing towards greater utilization of specialty products. Comments are suggested to ensure that the government has your viewpoint in mind when developing further guidance and proposed rules.

There are multiple ways to respond to the “Rare Diseases: Natural History Studies for Drug Development” guidance document. Contact Frier Levitt Government Affairs to have your voice heard.

Biosimilar Naming: Where We’ve Been and Where We Are

This article originally appeared in The Center for Biosimilars.

March has been a rollercoaster month for the healthcare industry. From the sudden resignation of FDA Commissioner Scott Gottlieb, MD, to the announcement of the FDA’s current thinking on nonproprietary names of biological products, I have been hearing many questions from stakeholders on what, exactly, is happening with biosimilars. Let’s take a quick look at the FDA’s latest guidance on naming and at where the FDA is headed with new leadership.

According to Commissioner Gottlieb, the guidance, titled Nonproprietary Naming of Biological Products, accomplishes 3 things:

• The FDA no longer intends to modify the proper names of biological products that have already been licensed or approved without an FDA-designated suffix in their proper names.

• The FDA does not intend to apply the naming convention to the proper names of transition biological products.

• Going forward, for interchangeable biosimilars, the FDA intends to designate a proper name that is a combination of the core name and a distinguishing suffix.

Since the Affordable Care Act created the biosimilar pathway, the FDA has been trying to create the most appropriate policies for naming biological products, as well as trying to ensure drug safety or pharmacovigilance. In 2015, the FDA released 3 guidance documents, including a question-and-answer document, on the FDA’s viewpoint on the Biologics Price Competition and Innovation Act and on quality and scientific considerations for demonstrating biosimilarity. In 2017, the FDA deviated from the European Medicines Agency and the World Health Organization and finalized guidance saying that both originator biologics’ and biosimilars’ names would include 4-letter, FDA-designated meaningless suffixes.

Regarding the FDA’s 2017 suffix policy, Commissioner Gottlieb stated that “By applying this policy to originator and biosimilar products alike, the FDA sought to advance the goal of patient safety—which the suffixes promote—without creating a misimpression that products with such suffixes are somehow inferior to those without. In addition, the FDA announced in that guidance that the agency was considering retrospectively changing the names of biological products already on the market to begin adding distinguishable suffixes. Many believed that the FDA would continue to evolve on this issue, and this month’s release proves them right.

So what happens next? First, the comment period ends on May 7, 2019, so the industry has until then to submit viewpoints on this particular policy. I do not think that Commissioner Gottlieb’s resignation will create setbacks to the FDA’s ambitious views of encouraging greater generic and biosimilar utilization. It is expected that acting FDA Commissioner Norman “Ned” Sharpless, MD, will fill the void starting in April until a permanent replacement is found. Sharpless does have significant industry experience, and, in my opinion, it seems as though HHS Secretary Alex Azar has confirmed in a recent statement the FDA’s path of aggressively introducing competition into the market to lower drug prices will remain for some time. The only question is whether this new naming guidance document by the FDA will achieve clarity and safety for industry stakeholders, including patients.

2019 Specialty Drug Pipeline & Policy Outlook: A Glimpse into Potential Blockbuster Drugs and the Policies that Govern Them Webinar Recording

The market has recently witnessed an acceleration of several new classes of drugs that were approved by the FDA. With specialty drug spending showing no signs of slowing down and the drug pricing debate raging in Congress, what are the current drugs in the specialty pipeline that stakeholders can take advantage of? Most importantly what policies are being developed that will govern accessibility and reimbursement for manufacturer, specialty and home infusion stakeholders?

In this recorded webinar, Ronald W. Lanton III, Esq., Executive Director of Frier Levitt Government Affairs, discusses:
• Overview of current policy
• Discussion of upcoming possible blockbuster medications
• Examination of market trend opportunities
• Guidance on next steps

By the end of this presentation, participants will be able to:
• Understand the current policy in place
• Identify new possible blockbuster medications in the specialty pipeline
• Comprehend new developing policies that will govern accessibility and reimbursement for manufacturer, specialty and home infusion stakeholders

Contact Frier Levitt Government Affairs today.

ONC and CMS Introduce Proposed Rules on Target Data Blocking

Both the Office of the National Coordinator for Health Information Technology (ONC) and the Centers for Medicare and Medicaid Services (CMS) have introduced proposed rules on Target Data Blocking, which, if properly tailored and modified, have the potential to substantially impact Accountable Care Organizations (ACOs), Independent Physician Associations (IPAs), Clinically Integrated Networks (CINs) and other value-based care organizations (VBCOs) by greatly expanding data transparency as between payers, including Medicare Advantage Organizations (MAOs), and VBCOs.

The CMS proposed rule on target data blocking is designed to transition the industry towards interoperability, and further builds upon CMS’ goals, deriving from the 21st Century Cures Act and Executive Order 13813, to improve access to, and the quality of, information that Americans need to make informed health care decisions, including data about health care prices and outcomes, while minimizing reporting burdens on affected plans, health care providers, or payers.

According to the ONC, their proposed rule on target data blocking would implement certain provisions of the 21st Century Cures Act, including more fully and completely describing the conditions and maintenance of certification requirements for health information technology (health IT) developers under the ONC Health IT Certification Program (Program), the voluntary certification of health IT for use by pediatric health care providers, and by insulating certain reasonable and necessary IT activities that do not constitute information blocking. The implementation of these provisions would advance interoperability and support the access, exchange, and use of electronic health information. The proposed rule would also modify the 2015 Edition health IT certification criteria and Program in additional ways to advance interoperability, enhance health IT certification, and reduce burden and costs.

Why You Should Participate:

For VBCOs, expansion and clarification of the 21st Century Cures Act prohibition on “information blocking” presents a rare opportunity to create a more level playing field as between VBCOs and payers by forcing the enhancement of payer data transparency, which is critical for VBCOs to gather and/or review the data and methodologies held/employed by payers to calculate patient risk scores and to confirm the accuracy of payer-calculated accrued shared savings (or shared risk). Additionally, VBCOs, being in the unique position of having comprehensive in-house data analytics departments and proprietary healthcare data analytics methodologies or software, must be certain that the new Health IT requirements imposed by the proposed rulemakings are both clear and reasonably implementable from both a cost and technological standpoint.

Submitting targeted, concise comments to CMS and ONC expressing questions and concerns relevant to the above issues is one means of ensuring your company’s continued success in the fast-changing value-based care landscape. Indeed, even if the agencies disagree with a proposed suggestions for improving the rules, in so doing, they will have to provide reasons therefor, thereby providing you with invaluable advanced guidance for full compliance with the rules prior to their implementation.

There are multiple ways to respond to the ONC’s “21st Century Cures Act: Interoperability, Information Blocking and the ONC Health IT Certification Program” and/or CMS’ “Medicare and Medicaid Programs; Patient Protection and Affordable Care Act; Interoperability and Patient Access for Medicare Advantage Organization and Medicaid Managed Care Plans, State Medicaid Agencies, CHIP Agencies and CHIP Managed Care Entities, Issuers of Qualified Health Plans in the Federally-facilitated Exchanges and Health Care Providers” proposed rules. Contact Frier Levitt Government Affairs to have your voice heard.

Sweeping Proposed Rule on Rebate Safe Harbor has Potential to Impact all Drug Supply Chain Stakeholders

HHS Secretary Azar has proposed a new rule that will eliminate a Safe Harbor to the federal Antikickback Statute that provides legal protection for rebate arrangements allowing drug makers to pay PBMs to secure their drugs position on PBMs’ Medicare formularies. The goal of eliminating this Safe Harbor, and replacing with a new one, is to ultimately lower prescription drug prices for patients and Medicare and to upend the complicated structure for how drugs are priced. If passed, the proposed rule has the potential to dramatically impact pharmacies’ acquisition price and pharmacy reimbursement rates. It could also potentially alter distributor profit margins, as well as impact the commercial space. The move, while laudable, requires substantial input from stakeholders.

According to Secretary Azar, “This proposal has the potential to be the most significant change in how Americans’ drugs are priced at the pharmacy counter, ever, and finally ease the burden of the sticker shock that millions of Americans experience every month for the drugs they need.” The 123-page proposed rule, however, does not address the impact on pharmacy reimbursement, the wholesale distributor model or manufacturer profits. CMS has traditionally left reimbursement rates largely to the “market forces” and has not weighed in on the diminishing pharmacy margins. This rule, if implemented without additional guidance, has the potential to further erode pharmacy margin caused by PBM spread pricing and to erode the margins of other stakeholders.

In addition to eliminating the current Safe Harbor, the proposed rule also creates a new Safe Harbor protecting discounts offered to patients at the pharmacy counter and would also protect fixed fee services arrangements between manufacturers and PBMs. The impact of such fixed fee service arrangements is another aspect stakeholders must carefully consider.

All drug supply chain stakeholders should analyze HHS’s sweeping proposed industry change. The proposed rule currently has little, if anything, to address the impact on such stakeholders, making it imperative for stakeholders to participate in Secretary Azar’s request for comment. The deadline to submit comments to HHS is April 8, 2019.

Frier Levitt Government Affairs, LLC, along with the attorneys at Frier Levitt, LLC, has scoured the rule, and gathered feedback from stakeholders ranging from retail pharmacies, specialty pharmacies, chains, distributors, national associations of providers and manufactures. We have a strong understanding of the impact of the proposed rule and are working with stakeholders to avoid unintended negative consequences.

Submitting your viewpoints will be critical since this proposal reflects a priority of the Administration. Contact Frier Levitt Government Affairs today to have your voice heard.

New FDA Proposed Rule Looking to Define the Term “Biological Product” Seeks Comments

The Food and Drug Administration (FDA) is out with a new proposed rule targeting pharmaceutical manufacturer and specialty pharmacy stakeholder interests. With the new proposed rule, the FDA is looking to amend its regulation that defines “biological product” to incorporate changes made by the Biologics Price Competition and Innovation Act of 2009 (BPCI Act), and provide an interpretation of the statutory terms “protein” and “chemically synthesized polypeptide.” Under this interpretation, the term “protein” would mean any alpha amino acid polymer with a specific, defined sequence that is greater than 40 amino acids in size. A “chemically synthesized polypeptide” would mean any alpha amino acid polymer that is made entirely by chemical synthesis and is greater than 40 amino acids but less than 100 amino acids in size.

The comment period for the new proposed rule on biologics is now open. The deadline to comment is February 25, 2019.

Biologics is a cutting-edge sector within the healthcare industry. Since biologics are not traditional drugs, they will need their own classification as they become more utilized. Reimbursement and regulatory oversight involving safety of biologics will depend on how the term “biological product” is defined in this proposed rule. Pharmaceutical manufacturers and specialty pharmacy stakeholders can participate in this decision by submitting comments. Contact Frier Levitt Government Affairs today to have your voice heard on this very important topic.

Comments Wanted: New ICER Report to Assess if Clinical Evidence Exists for Significant Increase in Prescription Drug Prices

Public comment is being sought on the Institute for Clinical and Economic Review’s (ICER) recently posted draft protocol titled the “Unsupported Price Increase” (UPI) report. This report analyzes significant prescription drug increases and looks to determine whether or not new clinical evidence exists that could be used to support those increases. Once finalized, the protocol will guide the development of the first of these annual reports, currently scheduled for release in October 2019.

The comment period closes on February 13, 2019.

ICER is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. They are quietly becoming a strong influential force on government policy. The recent announcement of ICER’s collaboration with the Department of Veterans Affairs (VA) Pharmacy Benefits Management (PBM) Services to support VA coverage and price negotiations with drug manufacturers makes it clear that their influence on drug pricing will only continue to increase in the future.

If you are a manufacturer, GPO, wholesaler or similarly situated life science or healthcare stakeholder, it is imperative that you participate to ensure having your voice heard. Contact Frier Levitt Government Affairs today for assistance in submitting your comments for this very important policy development before the upcoming deadline.

Take Action: New CMS Proposed Rule Targeting Manufacturers, Patient Assistance Programs, ACOs, Payers and ACA Exchange Stakeholders

The Centers for Medicare and Medicaid Services (CMS) has issued a new proposed rule targeting Accountable Care Organizations (ACOs), payers and Affordable Care Act (ACA) exchange stakeholders. The proposed rule, titled “Patient Protection and Affordable Care Act; HHS Notice of Benefit and Payment Parameters for 2020“, reflects a priority of the Administration around the Patient Protection and Affordable Care Act (PPACA) of reducing fiscal and regulatory burdens across various areas within the law. This proposed rule has several components that affect different stakeholders.

Manufacturers and Patient Assistance Program Stakeholders:

Copay accumulators are being implemented by insurance companies and Pharmacy Benefit Managers (PBMs), harming patient access. With this payor program, the value of copay assistance cards/coupons issued by manufacturers do not count towards out-of-pocket costs that are applied toward deductibles. The result has caused a cost shift onto consumers and away from employers and payers.

In this proposed rule, the Administration defends the use of copay accumulators stating:

“The availability of a coupon may cause physicians and beneficiaries to choose an expensive brand-name drug when a less expensive and equally effective generic or other alternative is available. When consumers are relieved of copayment obligations, manufacturers are relieved of a market constraint on drug prices which can distort the market and the true costs of drugs. Such coupons can add significant long-term costs to the health care system that may outweigh the short-term benefits of allowing the coupons, and counter-balance issuers’ efforts to point enrollees to more cost-effective drugs.”

“We propose, for plan years beginning on or after January 1, 2020, notwithstanding any other provision of the annual limitation on cost sharing regulation, that amounts paid toward cost sharing using any form Start Printed Page 291of direct support offered by drug manufacturers to insured patients to reduce or eliminate immediate out-of-pocket costs for specific prescription brand drugs that have a generic equivalent are not required to be counted toward the annual limitation on cost sharing. Not counting such amounts toward the annual limitation on cost sharing would promote: (1) Prudent prescribing and purchasing choices by physicians and patients based on the true costs of drugs and (2) price competition in the pharmaceutical market.”

Participating in this proposed rule is crucial for manufacturer and patient assistance programs to maintain patient access and stop unnecessary risk shifting from occurring in consumer spending.  We can help advocate your concerns.

ACOs, Payers and ACA Exchange Stakeholders:

Specifically, the proposed rule would set forth parameters and provisions related to the risk adjustment and risk adjustment data validation programs, cost sharing parameters, and user fees for Federally-facilitated Exchanges and State-based exchanges on the Federal Platform.

The proposal outlines changes that would allow greater flexibility related to the duties and training requirements for the Navigator program and proposes changes that would provide greater flexibility for direct enrollment entities, while strengthening program integrity oversight. Lastly, the proposal discusses policies that are intended to reduce the costs of prescription drugs. This includes proposed changes to Exchange standards related to eligibility and enrollment, exemptions, and other related topics.

The Administration has been making several changes to the ACA via regulation, as earlier Executive Orders described how regulatory changes would be used to make drastic reductions to the ACA. If you have an interest in the ACA or a direct one based off of an interest in the Exchanges, it is advised that you participate in this rule to ensure that your interests are represented. Not doing so will subject your organization to whatever policies may or may not arise from this rulemaking, potentially placing your organization and your priorities at a disadvantage.

Frier Levitt Government Affairs (FLGA) helps stakeholders comment on proposed regulations. FLGA understands the nuances around industry and has strategies that can help successfully communicate stakeholders’ positions to CMS.

The deadline to comment is February 19, 2019, so it is important that you contact Frier Levitt Government Affairs today to make sure your voice is heard.

CMS Permits Use of Step Therapy for Part B Drugs with Medicare Advantage Plans Affecting Physician Dispensing and Specialty Pharmacy Patients

The Centers for Medicare and Medicaid Services (CMS) recently announced a new policy affecting Medicare Advantage plans, specifically those Medicare Advantage beneficiaries who rely on prescription drugs from Medicare Parts B and D. This new CMS guidance effective, January 1, 2019, permits MA plans to use “step therapy” for Part B drugs as part of a patient-centered care coordination program. Simply put, step therapy would require physicians to write prescriptions for a less expensive alternative drug and see if it succeeds or fails before writing for some additional expensive medications. In what will likely end up resulting in unnecessary costs and delays many providers including national oncology organizations have publicly criticized the guidance as removing patient and physician choice and harming individualized treatment.

Not surprisingly, the PBM lobbying group, Pharmaceutical Care Management Association (PCMA), has publicly favored the guidance. The big three PBMs have common ownership with MA Plans. The new rule will allow Part D plans the ability to cross-manage drug decisions across Part B and Part D. Instead of a physician choosing what drug to prescribe, the PBM would decide through Step Therapy. Under the previous CMS rule, PBMs had little control over Part B drugs—that was the domain of physicians.  The new rule will embolden the PBMs’ ability to control dispensing with step therapy; will enable PBMs to control formulary design, thus allowing them to secure even greater “rebates” from manufacturers and better spread pricing. PCMA called for this rule change in its July 16, 2018 Statement to Secretary Azar regarding the American Patients First: Blueprint to Lower Drug. Unfortunately, the consequence of this rule will be to increase the list price of drugs and patient copayment, particularly for those suffering from serious disease states.

The issue for the industry now is how to deal with the possibility of higher out of pocket costs, uncertain patient outcomes and unnecessary interference with the doctor patient relationship. The first piece of advice for Part B and Part D providers is to be vigilant in your response to the increasingly utilized Request for Information (RFI). The Request for Information is a method that agencies extend to interested stakeholders to identify issues of concern to the industry that could be technical in nature. It is strongly encouraged that you reach out to agencies offering RFIs such as this would could help avoid policy changes that hurt patient care and provider autonomy.

Over the last 12 months, CMS has increasing relied on the issuing of Requests for Information (RFIs). An RFI is part of the rulemaking process wherein an agency, such as CMS, proposes a rule or policy that it is looking to either add or change. Proposed rule announcements are usually followed by a “comment period”, public workshops/hearings, a final comment period and then a final rule with effective date. The RFI process provides opportunities for industry stakeholders, including national associations, to comment on the technical aspects of a proposal. However, the Administration is using agencies like CMS to distribute RFIs as a way for the industry to guide the conversation on what should be included. This could provide your organization with another way to influence policy.

It is clear that the pace of federal and state government policy making in the healthcare and life sciences spaces has quickened and continues to raise the stakes for market participants. Frier Levitt Government Affairs, LLC, along with legal insight from Frier Levitt, LLC, helps physician dispensers and specialty pharmacies navigate market and policy changes and helps provide the necessary tools to stay ahead. Contact us today.

Modifying HIPAA Rules: OCR Looks for Input from Physicians, Hospitals and Healthcare Stakeholders

The Office for Civil Rights (OCR) has issued a Request for Information (RFI) seeking input from the public on ways to modify the Health Insurance Portability and Accountability Act (HIPAA). OCR intends to use the public’s comments to remove regulatory obstacles and decrease regulatory burdens in order to facilitate efficient care coordination and case management to promote value-based health care while preserving the privacy and security of protected health information.

Covered entities and business associates often defer thorough HIPAA compliance due to the onerous requirements and/or limited administrative resources required to develop and enforce a comprehensive policy and procedure manual. However, this places these companies at significant risk. OCR’s HIPAA enforcement has, and will likely continue, to rise.

This proposal offers providers an opportunity for physicians, hospitals and healthcare stakeholders to submit feedback to OCR highlighting certain obstacles they may face due to HIPAA regulations that do not meaningfully contribute to the privacy and security of protected health information.

The deadline for the comment period ends February 12, 2019. If you are a healthcare stakeholder and would like to participate, contact Frier Levitt Government Affairs today. We have several strategies to help you or your organization engage in this process and have your voice heard.