Access to Care | U.S. Federal

Access to Therapies

The majority of plasma protein therapies are used by patients with rare, chronic and life-threatening diseases and conditions. Plasma protein therapies represent a unique class of human- and cell-derived branded biologicals. From the source material originating from donated plasma, through purification, fractionation and final distribution, the production of these lifesaving therapies is distinct from the larger biopharmaceutical industry, and similarly produces distinct, non-interchangeable treatments. PPTA is committed to preserving timely and appropriate access to all brands of life-saving plasma protein therapies in all sites of care. PPTA meets frequently with Members of Congress and the Administration to ensure that policies impacting the plasma protein community reflect the distinct position of the plasma protein industry, as well as the unique nature of the rare disease patient who rely on these therapies for their lifesaving treatment.

Articles:

• An analysis and decision tool to measure cost benefit of newborn screening for severe combined immunodeficiency (SCID) and related T-cell lymphopenia
  
  

Comments:

• 21st Century Cures Initiative Comments
• 21st Century Cures Initiative Comments re: Clinical Superiority Requirements

Letters:

• Follow Up Letter to Commander Pedley

Issue Brief:

• Plasma Protein Therapies: Unique Biologicals
• Sequestration

Medicare Coverage

Plasma protein therapies used by Medicare beneficiaries predominately are reimbursed through Medicare Part B, which covers physician-administered therapies. It is essential that reimbursement for plasma protein therapies is sufficient to maintain access. PPTA regularly meets with staff on the relevant Congressional Committees handling Medicare policy to discuss the unique needs of the industry and patients and the importance of considering rare disease patients and treatments in health policies.

Comments:

• Hospital Outpatient Prospective Payment System Calendar Year 2016 Proposed Rule [CMS–1633–P] (August 2015)
• End-Stage Renal Disease Prospective Payment System, Quality Incentive Program, and Durable Medical Equipment, Prosthetics, Orthotics, and Supplies [CMS–1614–P] (August 2014)
• Hospital Outpatient Prospective Payment System Calendar Year 2015 Proposed Rule [CMS–1613–P] (August 2014)
  
• Hospital Outpatient Prospective Payment System Calendar Year 2014 Proposed Rule [CMS–1601–P] (September 2013)
• Hospital Outpatient Prospective Payment System Calendar Year 2013 Proposed Rule [CMS–1589–P] (September 2012)
• Hospital Outpatient Prospective Payment System Calendar Year 2012 Proposed Rule [CMS-1525-P] (August 2011)
• Parallel Review of Medical Products [Docket No. FDA-2010-N-0308] (December 2010)
  
• Medicare Program: Proposed Changes to the Hospital Outpatient Prospective Payment System and CY 2011 Payment Rates [CMS-1504-P] (August 2010)
• Medicare Program; Payment Policies Under the Physician Fee Schedule and Other Revisions to Part B for CY 2010 [CMS-1413-P] (August 2009)
• Medicare Program: Proposed Changes to the Hospital Outpatient Prospective Payment System and CY 2010 Payment Rates; Proposed Changes to the Ambulatory Surgical Payment System and CY 2010 Payment Rates [CMS -1414-P] (August 2009)
• Medicare Program: Proposed Changes to the Hospital Outpatient Prospective Payment System and CY 2009 Payment Rates [CMS -1404-P] (September 2008)
  
• Medicare Program; Revisions to Payment Policies Under the Physician Fee Schedule and other Revisions to Part B CY 2009 [CMS-1403-P] (August 2008)
  
• Final Rule: Medicaid Program; Prescription Drugs [CMS-2238-FC] (January 2008)
  
• Medicare Program: Proposed Changes to the Hospital Outpatient Prospective Payment System and CY 2008 Payment Rates [CMS-1392-FC] (January 2008)
  
• Medicare Program: Proposed Changes to the Hospital Outpatient Prospective Payment System and CY 2008 Payment Rates [CSM-1392-P] (September 2007)
  
• Proposal for the 2009 Healthcare Common Procedure Coding Systems Codes Application (September 2007)
• CY 2008 Physician Fee Schedule Proposed Rule (August 2007)
  
  

Letters:

• PPTA Comment Letter on the Discussion Draft on the Wholesale Distribution of Prescription Drugs (October 2007)

 
Issue Briefs:  

• 2013 Reimbursement Primer
• Changes in Medicare Part B Payment for Physician Office Drugs and Biologicals
• Average Sales Price (ASP) Reimbursement
  
  

Other:

• Medicare IVIG Access Stakeholders' Principles (November 2013) - document with Stakeholders
• The Moran Company Study, Trends in Medicare IVIG/SCIG Utilization for Primary Immune Deficiency Patients by Site of Service (December 2012)
• Hospital Outpatient Prospective System Payment Rates (August 2009) - to the Advisory Panel on Ambulatory Payment Classification Groups
• Maintain Access to Life Sustaining Plasma Protein Therapies Oppose Cuts to Medicare Drug Reimbursement -- key points (February 2013)
• Principles for the Establishment of Prescription Drug Pedigree Requirements for Plasma Protein Therapies (January 2008) 
  
  

Health Care Reform

The Affordable Care Act (ACA) contained many important provisions for people with chronic and rare diseases. However, as the ACA is implemented, it is vital to ensure that patients do not encounter access issues. PPTA is monitoring implementation of ACA to make sure it does not adversely affect patients who rely on life-saving plasma protein therapies.

Comments:

• Exchange and Insurance Market Standards for 2015 and Beyond (Re: CMS-99499P) (April 2014)
• Comments on Basic Health Program Proposed Rule (November 2013)
• Internal Revenue Service (IRS) notice of proposed rulemaking on the medical device tax (77 Fed. Reg. 6028, Feb. 7, 2012) (Proposed Rule) (May 2012)
• Pre-regulatory guidance on Essential Health Benefits Bulletin (January 2012) - to HHS
  
  

Letters:

• Pathway for Biosimilars Act (HR 1548) (May 12, 2009) - to Representatives Eshoo, Barton and Inslee
• Financing Comprehensive Health Care Reform: Proposed Health System Savings and Policy Options (May 26, 2009)- to the Senate Finance Committee
• Follow-On Biologics (H.R 1956), the Patient Protection and Innovative Biologic Medicines Act of 2007 (HR 1956) (June 2007)

Issue Briefs:  

• Medigap Reform (March 2012)
• Essential Health Benefits (January 2012)
  
  

Other:

• PPTA Statement on Signing of Health Care Reform Bill (March 2010) – Statement
• "Makers of Plasma Protein Therapies Support Eshoo's Biosimilars" (July 2009) -FDA Week article
• Side-by-side comparison of follow-on biologics bills before the Senate HELP Committee (July 2009)
• Patients Who Depend on Plasma Protein Therapies Need Legislative Protection from Unpredictable Risks to Which an Abbreviated Product Approval Pathway Could Expose Them (June 2009) Prepared by Hogan & Hartson LLP
• Emerging Health Care Issues: Follow-on Biologic Drug Competition (June 2009) - link to Federal Trade Commission report
• PPTA Health Care Reform Principles (May 2009)
  
  

Orphan Drugs and Therapies

The Orphan Drug Act, enacted 30 years ago, continues to stimulate a healthy environment for the development of rare disease therapies. Given that the majority of plasma protein therapies are used by patients with extremely rare diseases and conditions, PPTA continually monitors and promotes policies that support the investment in innovation and marketing of these therapies.

Comments:

• Branded Prescription Drug Fee (26 CFR Part 51) Public Hearing on Proposed Regulations (November 2012) – to IRS
• Clinical Superiority (January 2012) - to FDA
• Guidance Implementing the Annual Pharmaceutical Fee on Branded Prescription Drug Sales (June 2011) to IRS
  
  

Letters:

• The Preserving Access to Orphan Drugs Act (S. 1423) (December 2012) - to Senate Committee of Finance
• Preserving Access to Orphan Drugs Act of 2011 (HR 2672) (September 2011) - to House Committee on Ways and Means
• Preserving Access to Orphan Drugs Act of 2011 (HR 2672) (September 2011) to House Committee on Energy and Commerce
• Preserving Access to Orphan Drugs Act of 2011 (HR 2672) (August 24, 2011) to Reps. Gerlach and Altmire
• Preserving Access to Orphan Drugs Act of 2011 (S . 1423) (August 24, 2011) to Senators Toomey, Casey and Wyden
  
  

Issue Brief:

• Annual Pharmaceutical Fee (May 2013)

Comparative Effectiveness Research

PPTA recognizes the importance of advancing informed healthcare decisions through independent comparative research and studies. The intended use of comparative research results is to assist patients, clinicians, purchasers and policy-makers in making informed health decisions on how to effectively and appropriately prevent, diagnose, treat, monitor and manage diseases. It is essential to consider the effect of such research on rare disease patient populations and the ability to access the plasma protein therapy that will promote the best health outcome for the patient.

Comments:

• PCORI's National Priorities for Research and Research Agenda (March 2012) - to the Patient-Centered Outcomes Research Institute
  

Letters:

• Board of Governors of the Patient-Centered Outcomes Research Institute (June 30, 2010) -to Gene Dodaro, Acting Comptroller General
• Support for the Patient-Centered Outcomes Research Act of 2009 (S 1213) (July 10, 2009) - to Senators Baucus and Conrad
• Comparative Clinical Effectiveness Research - Comments on S. 3408 from the 110th Congress (March 2, 2009) - to Senators Baucus and Conrad 
  
  

340B Discount Drug Pricing Program

All PPTA members participate in the 340B program which provides steep discounts on drugs and therapies to safety-net hospitals and other statutorily covered entities. PPTA closely monitors policy developments related to oversight and integrity of the 340B program covered entities in order to protect access to life-saving plasma protein therapies.

Comments:

• Information Collection Request: Enrollment and Re-Certification of Entities in the 340B Drug Pricing Program and Collection of Manufacturer Data to Verify 340B Drug Pricing Program Ceiling Price Calculations [OMB No. 0915-0327—Revision] (December 2015)
• Notice, 340B Drug Pricing Program Omnibus Guidance [RIN 0906-AB08] (October 2015)
• Exclusion of Orphan Drugs for Certain Covered Entities Under the 340B Program [RIN 0906-AA94] (July 2011) - to the Health Resources and Services Administration, U.S. Department of Health and Human Services
• Civil Monetary Penalties (November 2010) - to the Health Resources and Services Administration, U.S. Department of Health and Human Services
• Administrative Dispute Resolution Process (November 2010) - to the Health Resources and Services Administration, U.S. Department of Health and Human Services
  
  

Letters:

• Policy documents pertaining to the 340B Drug Pricing Program (March 2012) - to HRSA and OPA
• The Disproportionate Share Hospital Prohibition for Purchasing from Group Purchasing Organizations (September 23, 2009) - to Jimmy R. Mitchell, Director, Office of Pharmacy Affairs, Health Resources and Services Administration, U.S. Department of Health and Human Services
• Affordable Health Choices Act (Sections 611 and 612) (June 30, 2009) - to Senate HELP Committee