ASH Clinical News Focus on Rare Diseases | Page 17

POLICY UPDATES
A Phase III Study for Patients With Newly Diagnosed Acute
Promyelocytic Leukemia (APL) Using Arsenic Trioxide and Alltrans Retinoic Acid (NCT02339740)
study design: Open-label, phase III efficacy study
study start date: June 2015
estimated study completion date: April 2023
study status: Currently recruiting participants
estimated enrollment: 158
sponsor: Children’s Oncology Group
This study assesses treatment with tretinoin and arsenic trioxide
in patients with newly diagnosed acute promyelocytic leukemia
(APL). Standard treatment for APL involves high doses of
anthracyclines, which are known to cause long-term side effects.
Tretinoin may stop the growth of cancer cells by blocking some
of the enzymes needed for cell growth, completely removing or
reducing the need for anthracycline chemotherapy, which may
reduce some of the long-term side effects.

LYMPHOMA & MYELOMA
Phase 3 Multi-Center Randomized Study to Compare Efficacy
and Safety of Romidepsin CHOP (Ro-CHOP) Versus CHOP
in Patients With Previously Untreated Peripheral T-Cell
Lymphoma (NCT01796002)
study design: Randomized, parallel-group, open-label, phase III
safety/efficacy study
study start date: January 2013
estimated study completion date: July 2024
study status: Currently recruiting participants
estimated enrollment: 420
sponsor: The Lymphoma Academic Research Organisation
Although CHOP (cyclophosphamide, hydroxydaunorubicin,
vincristine, prednisone) has limited efficacy in the setting of
peripheral T-cell lymphoma (PTCL), it is widely used for the
treatment of this disease. Trial investigators are evaluating
whether adding the histone deacetylase inhibitor romidepsin
to CHOP (Ro-CHOP) could improve its efficacy in patients with
previously untreated PTCL.

Phase II Study of ABT-199 (GDC-199) In Patients With
Relapsed Or Refractory Waldenström Macroglobulinemia
(NCT02677324)
study design: Open-label, phase II safety/efficacy study
study start date: April 2016
estimated study completion date: February 2023
study status: Currently recruiting participants
estimated enrollment: 30
sponsor: Dana-Farber Cancer Institute
This study is assessing the safety and efficacy of ABT-199 – an
oral agent that blocks BCL-2, a protein that is important for the
survival of Waldenström macroglobulinemia cells – for patients
with Waldenström macroglobulinemia who have relapsed after
receiving at least one prior therapy or whose disease is refractory
to treatment.

ASH Calls for Payment
Innovation in Rare Diseases
Such as Sickle Cell Disease

The Center for Medicare and Medicaid Innovation (CMMI),
a branch of the Centers for Medicare and Medicaid Services
(CMS), was created by the Affordable Care Act in 2010. Since
then, CMMI has received $10 billion in funding over 10 years
to create more than 50 different payment and delivery
models, such as bundled episodes for hospitals and the
Oncology Care Model.
As part of their multifaceted initiative to address the burden
of sickle cell disease (SCD), the American Society of Hematology (ASH) sent CMMI a letter in April 2016 requesting that
it explore new payment models to improve the care of these
patients. In the letter, ASH congratulates CMMI on its many
endeavors to address payment reform but expresses concern
that individuals with rare diseases such as SCD may be left
out of this reform.
Many experts believe that the care of people with SCD is in
part hampered by a payment system that does not reward ongoing care of individuals with difficult chronic diseases, particularly when they are more likely than not to be covered by
Medicaid. The letter also acknowledges that patients who live
in areas without specialized sickle cell centers tend to receive
“scattered” care from a variety of physicians and hospitals.
Most primary-care physicians are not able to adequately care
for SCD patients, given the limited time and payment associated with office-based evaluation and management codes.
To address this problem, ASH proposed a risk-based
model in which a single organization would be responsible
for costs associated with patients with SCD in a given region.
This model will be designed so that participants have a strong
incentive to improve care for patients who are outside of their
existing care network and who have been poorly served by the
existing system.
“We hope that the economic realities of practice do not serve
as a barrier to ensuring that patients receive state-of-the-art care,”
ASH President Charles Abrams, MD, wrote in the letter. “ASH
urges CMMI to invest the necessary resources to test payment
models to help individuals suffering from this disease.”

Do Major Changes to
MACRA Implementation
Overlook Rare Diseases?
In late April 2016, CMS released their proposed rule for implementing the Medicare Access and CHIP Reauthorization
October 2016

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