The Advocate

Volume 1, Issue 5

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July 2021

In This Issue:

ASGCT Recommends Mechanisms for Adequate Medicare Reimbursement
ASGCT Voices Support of Value-Based Purchasing
Society Shares Insights on Clinical Challenges
Policy News


ASGCT Advocacy

ASGCT Recommends Mechanisms for Adequate Medicare Reimbursement

ASGCT submitted comments last month on the FY22 Inpatient Prospective Payment System (IPPS) proposed rule. The Society’s comments highlight the proposed change to broaden the procedure codes that would be assigned to MS-DRG 018 and rename that MS-DRG to include both CAR T and other immunotherapies. Our comments request CMS to ensure those changes do not impact the ability of providers to offer to Medicare beneficiaries any currently approved therapies included in the DRG. More specifically, ASGCT indicated the following positions:  

  • Recognition that CMS is proposing assignment of TIL therapies to the most similar DRG (018).   

  • Recommendation to further consider the appropriateness and patient access implications before grouping these two types of therapies together on a long-term basis.  

  • Recommendation to change the name of the DRG to immune effector cell therapies if CMS includes TIL therapies in this DRG upon initial approval. 

  • Support of the continued exclusion of clinical trial cases from the payment calculations used to set the relative weight of the MS-DRG 018.  

  • Support of the use of NTAPs in the current system for all products that meet the statutory and regulatory criteria for such payment. 

In addition, ASGCT indicated the following recommendations on how the current Medicare reimbursement system could be improved if it is to be retained:  

  • NTAP reform. ASGCT recommends:  

    • Quarterly review of NTAP-qualifying products approved by the FDA (vs. annual).  

    • Ability for manufacturers to apply for an NTAP earlier (before the product is marketed).  

    • An increase in the cap for NTAP amounts from 65 percent to 100 percent, or a uniform NTAP equal to the product acquisition cost for gene and cell therapies.  

    • NTAP eligibility for three full years prior to rate-setting and/or: 

  • Establishment of new MS-DRGs prior to NTAP expiration. 

ASGCT Voices Support of Value-Based Purchasing

In late 2020 CMS finalized a rule that will allow a new reporting mechanism (multiple best prices) to enable manufacturers to enter into value-based purchasing (VBP) arrangements with payers (state Medicaid programs and commercial insurers) for drugs and biologics. Following the change in Administration, CMS recently issued a new proposed rule that would delay for six months the January 1, 2022 effective date for this new provision until July 1, 2022, which will allow CMS to operationalize the complex system changes required. ASGCT submitted comments last month that acknowledge CMS’ need for additional time for implementation and restated some of our key positions for the benefit of the new Administration. ASGCT commented on the original draft rule with its standard position, which is to support the enabling of novel payment models, such as VBP arrangements. The Society reiterated a request for CMS to clarify whether it intends to enable VBP arrangements that contain payment-over-time provisions, which ASGCT would support.

Society Shares Insights on Clinical Challenges

In June ASGCT responded to a request for information from the National Center for Advancing Translational Sciences (NCATS) on the challenges that the rapid pace of development of gene therapies places on the existing diagnostic and delivery infrastructure. To facilitate the equitable and efficient delivery of gene therapies to more patients, the Society noted its support of key policy priorities, including the following: Medicaid coverage of genetic testing; allocation of additional funds to the Center for Biologics Evaluation and Research (CBER); full coverage and enabling of novel payment models; ensuring the federal Recommended Uniform Screening Panel (RUSP) keeps pace with treatment approvals; and encouraging the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) to make their process transparent, predictable and timely and to collaborate with and rely upon FDA. ASGCT also highlighted its methods for creating resources for its Patient Education Program to facilitate timely awareness of gene therapy options for patients and their families.

Policy News

  • The NEWDIGS (New Drug Development Paradigms Initiative) FoCUS (Financing and Reimbursement of Cures in the US) program at MIT released a new white paper on Emerging Market Solutions for Financing and Reimbursement of Durable Cell and Gene Therapies. FoCUS’ Mark Trusheim will present on the Medicaid pipeline for gene therapies at the ASGCT Policy Summit in September. 

  • Novartis published long-term data in June that supports Zolgensma’s use in pre-symptomatic SMA patients. These findings reinforce the need for robust state newborn screening programs to diagnose children as early as possible to enable early access to approved treatments. 

  • This week bluebird bio's adrenoleukodystrophy gene therapy, Skysona, was approved by the European Commission for patients with certain genetic mutations who don't have a sibling who is a match for a stem cell transplant.  

  • MACPAC (Medicaid and CHIP Payment and Access Commission) published its June 2021 report to Congress, which included recommendations on high-cost specialty drugs. Their recommendations include increasing the base rebate amount for therapies with accelerated approval until post-market surveys are completed. MACPAC’s Executive Director will also be presenting at the Policy Summit on the Commission’s additional considerations specifically for gene and cell therapies. 

  • On July 1, EMA released two draft guidelines for public comment. These guidelines assist sponsors of investigational medicinal products (IMPs) for new drugs and biologics in deciding which manufacturing changes are considered a substantial modification that will need prior approval. 

  • The Newborn Screening Saves Lives Reauthorization Act (H.R.482), passed the House on June 23. The companion bill (S.350) has been introduced in the Senate. 

  • In June, Yescarta became the first CAR-T therapy to be approved in China under a joint venture with Kite and Fosun.


  • Register to attend the third annual ASGCT Policy Summit, taking place in a hybrid format September 22-24, 2021! The event will be held in-person in Washington, DC and streamed live online. Andi Lipstein Fristedt, FDA Deputy Commissioner for Policy, Legislation, and International Affairs will give a keynote presentation on Key Issues in Regulatory Policy. You can get more information about the full Policy Summit agenda, health and safety protocols for in-person attendees, and travel information on ASGCT’s website.  

  • Register to attend ASGCT’s virtual AAV Integration Roundtable on August 18. While the risk of rAAV-mediated oncogenesis in humans is only theoretical, this virtual event will address varying reports from preclinical research on the integration profile of AAV vectors and identify further research needs on this topic.  


ASGCT Policy Summit

September 22-24, 2021

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