Hemophilia Federation: Washington Wire – July 2020

WASHINGTON, Aug. 1 -- The Hemophilia Federation of America issued the following news:

On July 21, CSL Behring and Ferring Pharmaceuticals announced a recall of multiple lots of Stimate (desmopressin), in the United States and in other countries around the world. The recall was prompted by the manufacturer's discovery of out-of-specification levels of the active ingredient in the marketed product. This announcement has raised many troubling questions among national and international patient organizations and community members. HFA and NHF submitted a letter to CSL Behring and Ferring asking, among other things:

* For a full account of the circumstances surrounding this serious event;

* What are the implications for patients who use or have used the product;

* How the companies plan to ensure patient safety going forward; and

* How the companies explain their delay in notifying U.S. customers of the recall.

As we've previously reported, HFA and NHF convened a Safety Summit six months ago to address community concerns relating to a series of drug safety reports that emerged over the course of 2019. The Safety Summit's mission was to "articulate expectations for monitoring, informing, educating, and communicating issues related to product safety," with attention to the role of each stakeholder in the process. Eighty-five stakeholders, including CSL Behring, participated in the summit. It is disappointing that the bleeding disorders community finds itself once again facing some of the very gaps in safety notification standards that were comprehensively considered at the Safety Summit.

HFA and NHF will keep the bleeding disorders community informed as further information is gathered about the Stimate recall.

Congress tackles fifth COVID relief package

On July 27, Senate Republicans released a proposed fourth COVID relief package, the Health, Economic Assistance, Liability Protection and Schools (HEALS) Act. The Senate bill provides for some $1 trillion in additional federal spending - a marked decrease from the $3 trillion proposed in the HEROES Act that passed the House of Representatives in May. Another difference between the Senate and House bills: the Senate HEALS measure provides no additional federal funds for Medicaid and none of the health coverage extensions that patient advocates requested (opening an emergency special enrollment period to obtain health insurance in the federal ACA Exchanges; enhancing premium subsidies for ACA plans; subsidizing COBRA premiums for laid-off or furloughed workers seeking to maintain job-based coverage; etc.).

The HEALS Act does include a second round of Economic Impact Payments (akin to the March 2020 CARES Act, which provided up to $1200 in direct payments to individuals), and replenishes funding for the Paycheck Protection Program. The Senate bill also includes a federal unemployment supplement, but at greatly reduced levels from the CARES Act provision that lapsed on July 31 ($200/week instead of $600/week for 60 days, then transitioning to a formula that sets benefits at 70% or less of a worker's former earnings). These supplemental federal unemployment benefits would only last through the end of 2020. The Senate bill also contains a plethora of a provisions relating to education, hospital funding, telehealth waiver extensions, employer liability, etc.

House and Senate Democrats have criticized the HEALS bill as inadequate - while even within the ranks of Senate Republicans, there are grumblings that the bill goes too far. Given these divisions, lawmakers are far from reaching agreement, even as they say they want to pass a final relief package in August.

Quick Hits:

* On July 23, U.S. Secretary of Health and Human Services renewed the HHS determination that COVID-19 constitutes a national public health emergency. This declaration allows HHS to grant waivers, adjust Medicare reimbursements, and take other steps to handle the ongoing public health crisis. As HFA has previously reported, one HHS requirement linked to the declaration is that states that accept enhanced federal Medicaid funding are restricted from disenrolling Medicaid beneficiaries for the duration of the emergency. (To date, no state has refused enhanced federal Medicaid funding, so all are subject to the Medicaid non-disenrollment requirements).

* On July 24, the White House announced that the President had signed four executive orders addressing drug pricing issues. Three of the executive orders address: rebate reform for drugs covered by the Medicare Part D program; a pass-through to patients of certain discounts on EpiPens and insulin; and importation of prescription drugs from Canada. A fourth executive order (the text of which has not been published) reportedly lays down an ultimatum to drug manufacturers: come up with a plan to reduce drug prices, within 30 days, or else the Administration will move to link Part B drug reimbursements to the prices paid overseas. Observers note that there will be significant challenges in implementing these executive orders, which do not in and of themselves carry the weight of law.

* On July 24, the House of Representatives passed H.R. 7608, an appropriations bill for fiscal year 2021, that includes an amendment giving the U.S. Food and Drug Administration the authority to recall drugs. "The bill includes language providing FDA, for the first time, with mandatory recall authority for prescription and over-the-counter drugs," according to a summary prepared by the House Appropriations Committee.

* On July 17, a federal appeals court in Washington, D.C., upheld a Trump Administration 2018 rule expanding the availability of short-term health insurance plans that don't have to meet ACA standards. Short term plans can deny coverage and/or can charge higher premiums based on health status; don't have to cover essential health benefits such as prescription drugs; can put dollar limits on covered benefits; and don't have to cap patient out-of-pocket spending. As previously reported, HFA had joined with 12 other groups to file an amicus curiae brief in the litigation over the short-term plan rule. In other litigation news, the Administration and the state of Arkansas on July 14 asked the Supreme Court to reverse an appeals court decision that blocks Arkansas and other states from implementing Medicaid work reporting requirements. And on July 29, a federal district court in New York barred the Administration from implementing or enforcing its public charge rule for the duration of the COVID-19 public health emergency. The court found that the public charge rule discourages immigrant families from seeking health care, thereby exacerbating the risks of the pandemic.

* HFA staff participated in the Hemophilia Alliance virtual Hill Day in late July, advocating for enactment of the "Hemophilia SNF Access Act" ( 3233and H.R. 5952). The SNF bills address the problem that, under current law, Medicare reimburses skilled nursing facilities on a bundled, per diem rate that doesn't come close to covering the costs of bleeding disorders treatments. As a result, patients with bleeding disorders typically cannot access SNF care. The bipartisan House and Senate bills will allow SNFs to bill Medicare a separate, additional amount for bleeding disorders treatments.

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To: Brian Puglisi, PharmD, Quality Assurance Manager, Global Quality, CSL Behring, LLC

Paul Stapel, Director, Quality Assurance, Ferring Pharmaceuticals, Inc

Re: Pharmacy-Level Recall of desmopressin nasal spray (Stimate)

Dear Mr. Puglisi and Mr. Stapel,

The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) are national nonprofit organizations that represent individuals with bleeding disorders across the United States. Our missions are to ensure that individuals affected by hemophilia and other inherited bleeding disorders have timely access to quality medical care, therapies, and services, regardless of financial circumstances or place of residence. Both organizations accomplish this through advocacy, education, and research.

We are writing concerning the announced recall of desmopressin nasal spray (Stimate) manufactured by Ferring Pharmaceuticals and licensed and distributed in the U.S. by CSL Behring. The recall, which is due to out-ofspecification levels of the active ingredient, is deeply disturbing - particularly since Ferring has issued a Health Hazard Safety Evaluation, warning that certain defective units of the product could "pose a significant hazard and may cause adverse health consequences due to increased concentrations of desmopressin." Beyond this alarming defect, and the fact that the affected lots appear to have been distributed as early as December 2017 - more than 2.5 years ago - we are also troubled by the fact that U.S. customers are learning of the recall 10 days later than their counterparts in countries such as Brazil and Singapore.

With this letter, NHF and HFA are initiating an ongoing dialogue so that our organizations can inform the patients we represent about the reasons for the recall, your proposed strategy for handling the recall, and implications for patients who use the product now and in the future. We have some immediate questions for CSL Behring and Ferring and ask that you respond to these questions in writing as quickly as possible.

1. Please describe the specific nature of the product deviation. If only certain lots were affected, please specify which lots, quantify how many vials were affected, and describe how many affected vials were distributed in the U.S.

2. What is the basis for Ferring's preparation of a Health Hazard Safety Evaluation and its warning that "there is a reasonable probability that the use of, or exposure to, certain defective units of this product could pose a significant hazard and may cause adverse health consequences due to increased concentrations of desmopressin"?

3. Have Ferring and/or CSL Behring received notice of any adverse events affecting patients who used the product, either in the U.S. or overseas? What would be the potential medical implications for patients who have used or continue to use defective units?

4. Please describe, specifically and in chronological order, how and when the product deviation was discovered, and what steps are being taken to correct the deviation and prevent it from recurring.

a. During what time period were the affected products manufactured?

b. Please provide a detailed timeline with dates of

i. Ferring's discovery of the product deviation

ii. Ferring's notification of CSL Behring that there was a problem with the product

iii. Ferring's notification of the U.S. Food and Drug Administration

iv. Ferring's and/or CSL Behring's notification of U.S. recipients

5. Has Ferring halted production of the product?

a. If yes, do you expect that production will resume? When?

i. When would U.S. distribution resume?

b. Has Ferring and/or CSL Behring notified the FDA of possible shortages of the product?

6. What actions are Ferring and/or CSL Behring taking to accomplish the recall as well as ensure that patients in the U.S. receive timely notification of the recall?

a. Please explain why CSL Behring and/or Ferring determined that a pharmacy-level recall would be the appropriate level of recall in this instance.

b. Does a pharmacy level recall mean that Ferring and/or CSL Behring will take no steps to identify and/or notify affected patients?

c. How and to whom should patients return the product? Have pharmacies been provided with instructions on how and where to return affected product?

d. Has Ferring and/or CSL Behring submitted notice of the recall to the Patient Notification System (PNS)? If so, when will the PNS notification be distributed? If Ferring and/or CSL Behring do not plan to activate the PNS, why not?

e. Please describe how Ferring and/or CSL Behring are announcing the recall and explain your reasons for using different modalities (e.g. email vs. FedEx) to contact different groups of stakeholders.

7. What, if any, medical guidance is Ferring and/or CSL Behring providing to U.S. health care providers and pharmacies? We request that you provide us with copies of all communications between either of your companies and health care providers and/or pharmacies regarding the product recall.

a. Are you recommending any specific laboratory testing?

b. Are you recommending that patients transition to another treatment?

8. Based on available website information, recalls were announced in other countries as early as July 10,2020. Why did it take so long to notify patients in the U.S.?

We look forward to your prompt response with answers to these questions. We hope to keep open lines of communication as this situation develops. Please contact Nathan Schaefer, Vice President of Public Policy for NHF ([email protected]) and Sonji Wilkes, Senior Director of Policy, Advocacy and Government Education for HFA ([email protected]) to schedule a call to respond to our questions.

Sincerely,

Dr. Leonard Valentino, President and CEO, National Hemophilia Foundation

Sharon Meyers, EdD, CFRE, President and CEO, Hemophilia Federation of America