AARDA Nationwide Advocacy

AARDA Advocates for Policies Important to People Living with Autoimmune Disease and Their Families

The American Autoimmune Related Diseases Association is actively promoting proposals to protect and improve key policies in the federal and state arenas that are critical to the health and well-being of people living with autoimmune disease and their families. AARDA supports an array of stakeholders that collectively deliver care and innovate for new and more effective treatments.

Recent News

Published in the Morning Consult August 26, 2020
By: Randall Rutta
Individuals living with rare and chronic diseases, or any other serious underlying health conditions, have an acute understanding of the critical importance of patient access to the latest treatments and therapies. For the autoimmune community, pharmaceutical discovery and innovation is a lifeline to health and well-being that, for many, is nothing short of life-changing.

The United States is an indisputable global hub of medical innovation that creates breakthrough, life-saving therapies for those with autoimmune and other diseases, along with so many other devastating conditions. In fact, U.S.-based firms fund 44 percent of the world’s medical research and development and invest 75 percent of global medical capital.

Now, with the onset of COVID-19, our nation’s leadership role in medical innovation is more important than ever. It offers hope for combating this devastating novel coronavirus as it has delivered treatments for so many people with serious illnesses and chronic conditions in the past.

Take, for example, remdesivir – a compound originally invented by Gilead Sciences for the treatment of Ebola.

While it ultimately proved ineffective for that disease, remdesivir has yielded a different but critically important clinical outcome: Patients suffering from COVID-19 that are treated with remdesivir are discharged from the hospital four days faster on average. This both ensures and expands needed hospital capacity for other at-risk patients, particularly those suffering from chronic underlying health conditions. And remdesivir is just one striking example of innovation that the United States is known for, and is a comfort to the millions around the globe who seek a viable therapeutic response to the pandemic – and who know it will be the U.S. innovation ecosystem that will ultimately deliver.

Yet, a recent experience concerning remdesivir is also an example of American innovation at-risk, as this and other breakthrough medicines are potentially undermined by an organization that few are familiar with, but that wields outsized influence: the Institute for Clinical and Economic Review.

ICER has appointed itself the sole arbiter of new-drug value, and its reports and value-assessment announcements inform policymakers, industry, insurers, patient groups and the public about what ICER believes pharmaceutical manufacturers should charge for their drugs.

ICER issues these decrees asserting “patient engagement” and “increased access” – yet, a brief glance behind the curtain reveals that neither is a meaningful part of ICER’s value assessments. The organization states that collaboration with industry partners and public comment from relevant stakeholders are necessary inputs to its analyses, but the reality is much different.

So, while remdesivir provides a strong example of U.S. medical innovation, it also serves as strong example of ICER’s flawed and disingenuous approach to valuing new treatments and therapies during this time of unprecedent global pandemic and generally.

In fact, in reviewing remdesivir, ICER departed from its own established value framework, which was updated this year with input from a broad range of stakeholders and requires rigorously analyzing clinical data and convening key stakeholders to inform these value calculations. ICER did not consult with critical stakeholders – neither Gilead, nor patient groups, nor others – prior to rendering a judgment on remdesivir. Nor did they invite public comments (a customary step before finalizing any recommendations).

ICER failed to rigorously analyze available data and failed to inform key stakeholders, including the drug’s manufacturer. Based on ICER’s own standards and value framework, the remdesivir valuation was both premature and incomplete. Its wholly underwhelming follow-up report did nothing to substantively address or meaningfully respond to the important points third-party stakeholders were making.

From 2014 to 2018, U.S. companies developed about twice as many new drugs as all of Europe combined. Consider that not a single ICER review of a breakthrough rare disease therapy that emerged during this time period resulted in a “high value” rating, and one begins to perceive an agenda-driven — rather than science-driven — institution that nevertheless claims to consider patients.

If this trend continues, and we find that ICER’s treatment of remdesivir is some kind of “new normal,” patients facing rare and chronic diseases and underlying health concerns should be worried about safe and effective access to needed medications, both now and in the future.

To be clear, the need for and utility of pharmaceutical value assessment is not in question. In fact, there are nearly two dozen different frameworks available that are data-driven, comprehensive, scrubbed of the use of quality-adjusted life years and meaningfully inclusive of patients and attentive to their priorities for health and life.

But if ICER’s remdesivir analysis – one that not only misses the mark objectively, but disregards ICER’s own established value framework – becomes the standard, it poses a grave threat to the U.S. innovation ecosystem that millions of patients have come to rely on.

The best way to move beyond this agenda-driven, myopic view of value is simple: calculate and consider value the right way. This means engaging with all constituencies in good faith, considering all relevant data, and presenting a holistic view of value that goes beyond simply the point of view of an insurer.

The consequences of devaluing new treatments and therapies are simply too dire to ignore the outsize value that life-saving innovation has during a time of global pandemic – and the contributions to health equity realized by ensuring disproportionately impacted communities have access to breakthrough therapies.

Randall Rutta is president and CEO of the American Autoimmune Related Diseases Association.

AARDA in Action

August 2020

July 2020

  • Campaign to encourage CMS to drop a provision in a Medicaid Pricing Rule that would apply unworkable requirements on drug manufacturers that could undermine copay coupon assistance programs that help patients access and afford their medicines and care, increasing the financial burden to patients during in the challenging environment created by the COVID-19 pandemic
  • Signed onto patient group letter encouraging Florida state legislators to guard against high patient cost-sharing for prescription medicines by opposing insurance plan use of accumulators
  • Joined patient advocates on a letter to Ohio state legislators requesting action to protect patients from a practice being implemented by various health insurance plans, pharmacy benefit managers, and employers
  • Lead sign-on letter with IDF and LFA urging Congressional leadership, as part of the next Coronavirus (COVID-19) legislation, to task the CDC with working with stakeholders to develop a recommendation to ensure the needs of immunocompromised Americans are adequately addressed

June 2020

  • Signed letter in support of the Part B Access to Seniors and Physicians (ASP) Coalition’s mission to preserve patient access to Medicare Part B covered services, including restoration of full access restore unfettered access to Medicare Part B covered drugs for beneficiaries enrolled in Medicare Advantage (MA) plans
  • Letter to CMS to encourage you to make the development and availability of treatments for pain – and particularly non-addictive options – a high priority within your agencies
  • Joined PIPC on a letter urging ICER to pause any future development of assessments related to COVID-19 and focus on partnering with stakeholders in the development of rigorous and patient-centered methodologies
  • Signed onto letter organized by the Aimed Alliance to Congress in support of legislation that would prohibit the use of prior authorization and step therapy during the public health crisis. Comparable to prior letter sent to governors, state legislators, and CMS

May 2020

  • Letter to CMS on the recent interim final rule related to the COVID-19 public health emergency comment period titled Medicare and Medicaid Programs; Policy and Regulatory Revisions in Response to the COVID–19 Public Health Emergency (IFC), issued by the Centers for Medicare and Medicaid Services
  • Joined patient groups advocating to Expand Access to Part B Therapies in a letter calling on CMS calling to allow more flexibility for patients to receive in-home infusions and injections of therapies and treatments
  • Signed onto patient group letter encouraging New York state legislators to guard against high patient cost-sharing for prescription medicines by opposing insurance plan use of accumulators
  • Joined letter spearheaded by NORM Tennessee opposing TN Blue Cross Blue Shield decision to deny coverage of a new rare disease breakthrough treatment of Thyroid Eye Disease in favor of steroids, which are not approved by the FDA for this purpose

April 2020

  • Letter to HHS urging that the Department’s Office for Civil Rights issue additional guidance to ensure that schemes to ration scarce health care resources during the COVID-19 pandemic comply with federal nondiscrimination laws, with some 400 organizations in conjunction with the Partnership to Improve Patient Care (PIPC)
  • Letter to Congress urging expansion of eligibility for the paid family and medical leave program to include individuals determined by the Centers for Disease Control and Prevention (CDC) to be at high risk for adverse complications from COVID-19 and working members of their households, with the Cystic Fibrosis Foundation
  • AARDA/NCAPG letter to Senate urging to weigh-in with CMS to assure that patients covered by Medicare Part B are able to access their infusion and injection treatments at home
  • Letter to House Appropriations leaders to request continued support for the critical and highly successful defense health research programs funded through the Congressionally Directed Medical Research Programs (CDMRP)

March 2020

  • AARDA comments to CMS on NBPP proposal to undermine copay assistance coupon programs offered by drug manufacturers on behalf of the National Coalition of Autoimmune Patient Groups, and also with the All Copays Count and I Am Essential coalitions
  • Comments to CMS regarding proposed rule on Contract Year 2021 and 2022 affecting Medicare Part D beneficiaries, urging reconsideration of a preferred specialty tier, enhancement of real-time benefit tools, and maximum out-of-pocket caps, smoothing mechanisms, and a fix to the out-of-pocket “cliff,” with the MAPRx Coalition
  • Letter to Congress promoting passage of the Safe Step Act (HR 2279/S 2546), as part of the Safe Step Act Coalition
  • Letters to Governors, State Medicaid Directors, Insurance Commissioners, and Executive Directors of Boards of pharmacy urging that they act to protect rare disease patients and high-risk populations during the COVID-19 crisis, with the Every Life Foundation
  • Letter coordinated by the Arthritis Foundation to House leadership supporting an appropriation for osteoarthritis research at the US Department of Defense Congressionally-Directed Medical Research Program, one of many communications to congress supporting funding of general and disease-specific autoimmune research projects at DoD

February 2020

  • AARDA/NCAPG letter to all Senate and House Members to request that any consideration or efforts to use reference international drug prices in setting prices for medicines in American health care be rejected, and also with PIPC and ASP Coalition
  • Outreach to Congress in support of “Strengthening Innovation in Medicare and Medicaid Act” and other steps to improve and protect the CMS CMMI with the Healthcare Leadership Council
  • Letter to the Food and Drug Administration to oppose any proposed linkage between the Institute for Clinical and Economic Review (ICER) and FDA with the Partnership to Fight Chronic Disease
  • Letter to Oklahoma legislators opposing legislation to allow drug importation, coordinated by the National Grange; one of a series of communications with state legislatures on this topic, including Oregon, Maine, and New York

January 2020

  • Letter to House Committee Leadership in support of the “Seniors Prescription Drug Relief Act of 2019,” (S.2911) to provide a much-needed “smoothing” mechanism for Medicare beneficiaries to spread payment for Part D out-of-pocket costs across the calendar year, with MAPRx Coalition
  • Letter to Congress urging action to reverse an increase in Medicare Part D out-of-pocket (OOP) costs that will occur in 2020, with MAPRx Coalition

December 2019

  • AARDA at Open Public Hearing to encourage the FDA Dermatologic and Ophthalmic Drugs Advisory Committee to support expedited and favorable consideration of a breakthrough drug for the treatment of Thyroid Eye Disease; TEPEZZA approved for march market launch.
  • Comments to CMS opposing advanced rulemaking that would apply international reference pricing and other restrictions affecting Medicare Part B beneficiaries with the Part B Access for Seniors and Physicians (ASP) Coalition and Partnership to Improve Patient Care (PIPC)