2012-06-21

Congress Finalizes FDA Bill on User Fee Programs & Drug Shortages
On June 18, the United State Senate and House of Representatives filed the final Prescription Drug User Fee Act (PDUFA) legislation (The Food and Drug Administration (FDA) Safety and Innovation Act, S. 3187) including important provisions to mitigate drug shortages. In addition to addressing drug shortages, the bill would create user fee programs for generic drugs and generic biologic drugs, or biosimilars, and include provisions that focus on the safety of the drug supply chain, among other issues. The House passed the bill on Wednesday, June 20, and the Senate is expected to take up the bill the week of June 25.

In recent weeks ASH had been leading advocacy efforts to ensure that biological products were included in the bill's requirement that manufacturers provide advanced notification of stoppages or interruptions in production that could result in a drug shortage. In addition, ASH sought to remove the exemption of products derived from human plasma proteins and recombinant products replacing human tissue – products used for the treatment of hemophilia and other bleeding disorders – from the early reporting mandate in the House and Senate bills.

The advanced notification requirements in the final legislation apply to drugs which are "life-supporting, life-sustaining, or intended for use in the prevention of or treatment of a debilitating disease or condition including drugs used in emergency medical care or during surgery that are not radio pharmaceutical drugs." While the legislation does not include biological products in the advanced notification requirements, it gives the Secretary of the Department of Health and Human Services (HHS) the authority to apply these requirements to biological products including plasma protein therapies and their recombinant analogs by regulation if it would benefit public health.

The treatment of biological products in the final legislation reflects the significant difference between the House bill, which included biologics in this section, and the Senate bill, which limited the advanced notification requirements to sterile injectible products. The House and Senate resolved their differences by leaving the decision to the HHS Secretary and the regulatory process. Based on discussions with FDA on the legislation, agency officials have supported the inclusion of biological products in the advanced notification requirements. ASH's advocacy was influential in removing the exemption of plasma protein therapies and their recombinant analogs from the advanced notification requirements in the final bill.

Over the past 18 months, ASH has worked closely with Congressional leaders as they have developed drug shortage legislation. ASH has made several recommendations on ways to prevent and mitigate shortages, including to: increase FDA authority, require that the Secretary disseminate information about drug shortages to patient and provider organizations, enhance inter- and intra-agency coordination on drug shortages, require that the Agency Strategic Plan consider the impact of drug shortages on research and clinical trials, examine the impact of current FDA requirements on shortages, and provide economic incentives to manufacturers of critical drugs.

The Society's advocacy has been successful and the final bill includes several of ASH's key recommendations, including:

• Requiring that the Secretary disseminate information about drug shortages to patient and provider organizations;
• Requiring enhanced inter-and intra-agency coordination and requiring increased and effective communication with outside stakeholders; and
• Requiring that the Strategic Plan consider the impact of drug shortages on research and clinical trials.

More information about drug shortages and ASH advocacy is available on the ASH website.

Senate Committee Passes FY 2013 NIH Funding Bill, But Future Cuts Loom
On June 14, the Senate Appropriations Committee approved along party lines, 16-14, its FY 2013 Labor-HHS-Education spending bill, which covers most agencies within the Department of Health and Human Services (HHS). The Committee provided $30.723 billion for the National Institutes of Health (NIH), a $100 million increase over the FY 2012-enacted level and the president's FY 2013 request. The bill also includes $4 billion in funding over Republican objections to implement the health care overhaul law and the bill retains the salary cap at Executive Level II ($179,700) implemented this year.

The Society was pleased that its recommendations concerning research related to sickle cell trait and participation in athletic activities, research in the development of blood stem cells from induced pluripotent stem (iPS) cells, and efforts to prevent and mitigate drug shortages were included in the Committee report. These recommendations were shared with the Senate during the Committee on Government Affairs Hill Day and reflect the influence of ASH advocacy.

Following the Senate Committee action, normal process would be for the full Senate to approve the bill and for the House of Representatives Committee to follow suit. However, in this election year, it is expected that further legislative action will stop until September when the Congress will not have time to complete action on appropriations but instead will pass a Continuing Resolution that will keep the government funded past the beginning of the fiscal year on October 1. After the elections a "lame duck" Congress is expected to return to Washington to make final funding decisions for FY 2013.

On January 2, 2013, the across-the-board automatic spending cuts known as a sequester and mandated by last year's Budget Control Act are required to take effect. The effects of the oncoming automatic spending cuts are already starting to be felt by government agencies and will build in the second half of this year. The sequester will reduce spending by $109 billion over the course of the entire fiscal year and $1.2 trillion over nine years. Federal agencies, defense contractors and other organizations that rely on government contracts or grants are faced with making decisions on hiring, budgeting and projects for FY 2013 without any certainty of how much they will be able to spend. There is very little in the way of detail of how the $109 billion in automatic cuts would affect agencies. For example, we do not know the exact amount of cut NIH is facing although it is expected it will be between eight and nine percent. ASH will continue to monitor this issue and share additional information as it becomes available.

In the meantime, ASH will be enhancing and strengthening its advocacy efforts to include the following:

• On-line advocacy campaigns
• Town Hall Meetings – Beginning in July, members of the Grassroots Network will receive weekly alerts of when Town Hall meetings are scheduled in their congressional district/state. We find that attending these events (one hour) and asking about the candidate's position on NIH funding is an effective way to raise the NIH on the candidate's radar.
• Letter to the Editor Campaign – In July we will implement a campaign to submit a letter-to-the –editor focused on the importance of NIH funding. The goal is to place as many Letters to the Editor in local papers as possible.
• Build the ASH Grassroots Network – Over the summer we will also launch a campaign to invite your colleagues to participate in the ASH Grassroots Network.
• Patient Advocacy – We have already notified hematology-related patient advocacy groups about ASH's online campaigns and shared a sign-on letter with them. We will continue to reach out to patient groups.
• Fly-in Advocacy Day – We are planning to schedule a special one-day "fly-in" for interested members of the Grassroots Network during the lame duck session in November –December. The purpose will be to increase our contact with congressional offices and urge they protect the NIH.

Nominations Now Being Accepted for ASH Advocacy Leadership Institute
The ASH Advocacy Leadership Institute was created in 2011 as part of the ASH Recruitment and Retention Initiative to provide additional opportunities for ASH members to learn more about advocacy, health policy, the legislative process and to become engaged in the Society's activities. The second annual ASH Advocacy Leadership Institute will take place September 12-13, 2012, in Washington, DC. This two-day leadership workshop is a unique opportunity for ASH members gain better understanding of the Society and its activities, as well as legislation and health policy affecting hematology research and practice. Please nominate your colleagues or forward this e-mail to those who would be interested in this opportunity. To learn more about the 2012 ASH Advocacy Leadership Institute, to submit a nomination, and to see the program from 2011, please visit the Institute's website: www.hematology.org/ALI.