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MITIGATING HIGH-COST CLAIMS - National Alliance of Healthcare Purchaser ...
ACTION BRIEF
Employer Strategies that Drive Health, Equity and Value

MITIGATING HIGH-COST CLAIMS
A CLOSER LOOK AT HEMOPHILIA

                              Although hemophilia is a rare disorder
ACTION STEPS FOR              affecting about 25,000 people in the
EMPLOYERS:                    US, it ranks among the highest-cost
                              healthcare conditions, averaging
1. Use value-based
                              $270,000 per claimant annually.1
   insurance design to
   ensure that health         As with many rare diseases, hemophilia
   plans support patients     commonly requires lifelong treatment.
   with hemophilia and        Because of hemophilia’s low incidence,
   their families.            employers often have little to no
                              understanding of the disease and
2. Set expectations           what makes treatment so costly. Most
   that health plans and
                              employers hand off the development of
   other providers cover
                              hemophilia cost containment strategies
   new, evidence-based
                              to intermediaries, such as pharmacy
   treatments.
                              benefit managers (PBMs), carriers,
3. Ensure Hemophilia          TPAs or pharmacy consultants. While
   Treatment Centers          these vendors may develop appropriate
   are in all networks.       strategies that result in the lowest total
                              cost of care, conflicts can exist that
4. Accommodate                increase employer costs unnecessarily.          implementation to vendor partners who
   employees with
   bleeding disorders         When it comes to assuring optimal               may be conflicted. This will help them
   by providing on- or        patient outcomes at the lowest total cost       gain insight into the key cost levers that
   near-site support.         of care, employers should first consider        drive spend, as well as which traditional
                              having dialogue with hemophilia                 plan design strategies can create access
5. Educate and cover          experts, before simply handing                  barriers that can have a signficiant
   health plan members        off the strategic development and               impact on spend.
   with hemophilia,
   connecting them to the     “When it comes to rare diseases, like hemophilia, copay accumulator
   right health benefits
   and care management        adjustment programs, and step therapy/fail-first language can backfire
   resources.                 when implemented as a one-size-fits-all strategy. For patients and
                              payers alike, the unintended consequences that can result from these
                              obstacles can be catastrophic.”
                                                                           —Kollet Koulianos, Senior Director of Payer Relations,
                                                                                               National Hemophilia Foundation.
MITIGATING HIGH-COST CLAIMS - National Alliance of Healthcare Purchaser ...
UNDERSTANDING
HEMOPHILIA
Hemophilia is a bleeding disorder,
usually inherited, in which the blood
does not clot properly. With effective
treatment, most hemophilia patients
can live full lives and enjoy most of the
activities other people do.
Hemophilia results from a mutation in
one gene that normally directs clotting
factor proteins. As a result of impaired
clotting, unchecked bleeding can occur
within joints, causing chronic joint
disease and pain; in the head, causing
seizures and paralysis; or in other            dollars in government funding have          rare diseases from seeking treatment.
vital organs.                                  been used to improve medical care,          Instead, a “compassionate managed
                                               services, education, and the safety and     care” approach includes a more flexible
There are two types of hemophilia:
                                               surveillance of factor replacement          plan design that makes treatment of rare
Hemophilia A, resulting from a lack
                                               products.                                   diseases affordable and effective.
or deficiency of clotting factor VIII,
and hemophilia B, resulting from a                                                         For example, consider what is happening
                                               WHAT EMPLOYERS CAN
lack or deficiency of clotting factor IX.                                                  with copay accumulator adjustment
                                               DO TO EFFECTIVELY
Hemophilia A (classic hemophilia)                                                          programs. These programs are designed
                                               ADDRESS RARE
is four times more prevalent than              DISEASES SUCH AS
hemophilia B and is often severe.              HEMOPHILIA                                    The Triple Aim Approach:
                                               Use value-based insurance design              Optimal Outcomes at the
RECENT TREATMENT                                                                             Lowest Total Cost of Care
                                               to ensure that health plans support
ADVANCEMENTS
                                               patients with hemophilia and their            A high-cost rare disorder like
Treatment for hemophilia is advancing.                                                       hemophilia requires a high-
                                               families.
Recent developments include novel non-                                                       touch care approach. Hemophilia
                                               Care for rare diseases such as                Treatment Centers (HTCs) are
factor biologic agents and investigational
                                               hemophilia can be very expensive.             federally recognized centers of
gene therapies. In gene therapy, the                                                         excellence that subspecialize in
                                               Traditional plan designs have limited
patient’s defective gene is replaced with                                                    rare hemotologic disorders using
                                               flexibility in terms of deductibles,          an integrated, multi-disciplinary
a normal gene to enable the production
                                               copayments and co-insurance. This             approach to care—the “gold
of the active enzyme and prevent the                                                         standard.” There are 141 HTCs in the
                                               type of design can have negative
development and progression of the                                                           U.S. A directory is available here.
                                               consequences on employee health by
disease. Advocacy groups are pushing                                                         Having a chronic disease such
                                               allocating resources to low-value care
for further care advances that can be                                                        as a bleeding disorder often
                                               and discouraging employees from
provided at a lower cost. These include                                                      entails spending much time and
                                               seeking high-value care for essential but     effort navigating the healthcare
biosimilars, some of which are just
                                               expensive treatments.                         system. At an HTC, hematologists,
finishing clinical trials and high-cost gene                                                 pharmacists, nurses, social workers,
therapies that are coming for hemophilia.      The consequence can be that employees         psychosocial professionals,
                                               (or their dependents) do not seek             and physical therapists provide
The National Hemophilia Foundation
                                               adequate care for rare diseases because       coordinated treatment at one
(NHF) has advocated for the needs                                                            location with services available
                                               of unaffordable out-of-pocket costs,
and interests of people affected by                                                          24/7/365. Contracting with HTCs
                                               resulting in both a lower quality of life
hemophilia and related bleeding                                                              as in-network providers enables
                                               and ultimately more-costly care as the        employers to improve patient
disorders for almost 60 years. NHF                                                           outcomes and manage treatment
                                               disease, unchecked, results in more
advocates educate elected officials and                                                      costs. When HTCs are not in
                                               serious consequences.
others in healthcare and government                                                          the plan network and specialty
about the unique needs of people with          In other words, traditional plan designs      pharmacy is carved out to an
                                                                                             exclusive provider, total cost of care
bleeding disorders. As a result of             can be less effective, with built-in cost
                                                                                             can increase significantly.
efforts at the federal level, millions of      barriers that discourage employees with
MITIGATING HIGH-COST CLAIMS - National Alliance of Healthcare Purchaser ...
to help employees with rare diseases         safer and more effective than traditional
reduce their out-of-pocket expenditures      treatments.
when they need expensive drug                Payers are encouraged to make sure
treatments. However, the application         their benefit plans cover these new
of these copay accumulator programs          treatments. For treating hemophilia,
is being challenged in recent CMS            doctors now have access to recombinant
rulings. As a result, employees end up       factor VIII, artificially created in a lab.
paying more for the drugs they vitally       Recombinant factor VIII can be used
need. Employers can design plans to          instead of factor VIII obtained from
lower copays for effective, high-cost rare   human blood (frozen plasma), avoiding
disease care.                                the risk of viral infections, which are
The University of Michigan Center            sometimes transmitted in human blood.
for Value-Based Insurance Design is          The NHF is supporting the advancement
promoting Value-based insurance design       of gene therapy, as scientists better             What is Value-Based Plan
(V-BID) principles for marketplace and       qualify who might be best suited for the          Design?
employer health plans. These designs         therapy. Issues of cost and long-term             “It’s the idea that insurance design
seek to eliminate the underutilization       durability, efficacy, and safety are being        should be built on the concept
                                                                                               that high-quality treatments and
of high-value care, such as that for         determined.                                       providers should cost less. This
hemophilia, that occurs when plans
                                             Ensure your health plan offerings                 kind of smart pricing is intended
allocate more resources to low-value                                                           to improve health and minimize
                                             include providers with hemophilia
care with no clinical benefit. With value-                                                     waste.”
                                             experience and expertise.
based plans, employers incur savings by                                                                     —Tara Bishop, MD, MPH
                                             HTCs represent the gold-standard
making high-value care more accessible
                                             model of care for the management of
and affordable to employees.
                                             bleeding disorders. These specialized         Accommodate employees with
Set expectations that health plans
                                             centers provide comprehensive care for        bleeding disorders by providing on-
and other providers cover new,
                                             individuals with hemophilia, including        or near-site support.
evidence-based treatments.
                                             the development of specific treatment         Under the Americans with Disabilities
New treatments for hemophilia are            plans, monitoring and follow up of            Act, employers are obligated to
becoming available. Often, they are          affected individuals, and state-of-the-art    accommodate employees with bleeding
                                             medical care.                                 disorders. In particular, if asked by the
                                             This care results in documented               employee, employers must:
                                             improvements in outcomes, including              Provide the employee with a private
                                             a lower likelihood of death, fewer                place to self-administer infusions.
                                             hospitalizations, and lower use of
                                                                                              Offer a safe place for the employee to
                                             emergency services.
                                                                                               store needles and infusion supplies.
                                             Individuals who go to HTCs will not
                                                                                           Educate and cover health plan
                                             only receive outstanding medical care,
                                                                                           members with hemophilia,
                                             but also benefit from an experienced,
                                                                                           connecting them to the right health
                                             caring staff that takes time to develop
                                                                                           benefits and care management
                                             comprehensive treatment care plans for
                                                                                           resources.
                                             patients and families. Since hemophilia
                                             is heritable, genetic counseling is           Tutorials on rare diseases and best
                                             included to help families make informed       treatment practices are available from
                                             reproductive and other decisions.             national organizations and can be shared
                                                                                           with employees with these conditions.
                                             The treatment centers not only provide
                                                                                           For example, the NHF promotes a five-
                                             specialty care but also act as a resource
                                                                                           step regimen for people with hemophilia:
                                             for primary care doctors and dentists.
                                             This approach can serve as a benchmark        1. Get an annual comprehensive checkup
                                             for other network provider options.              at a hemophilia treatment center.
MITIGATING HIGH-COST CLAIMS - National Alliance of Healthcare Purchaser ...
2. Get vaccinated—hepatitis A and B are                       important for those with severe                               REFERENCE
   preventable.                                               disease because infusion of factor VIII                         1    Zhou ZY, Koerper MA, Johnson KA, et al.
3. Treat bleeds early and adequately.                         concentrate is most effective within one                             J Med Econ. 2015:18(6) 457-65..
                                                              hour of the onset of a bleeding episode.
4. Exercise and maintain a healthy
                                                              In general, rapid treatment is important
   weight to protect your joints.
                                                              because it reduces pain and damage
5. Get tested regularly for blood-borne                                                                                           RESOURCES FOR
                                                              to the joints, muscle, or other affected
   infections.                                                                                                                    EMPLOYERS:
                                                              tissues or organs; improves quality of
Additionally, care management                                 life; and boosts productivity.                                      • National Hemophilia
resources can be used to train                                                                                                      Foundation
                                                              Employers can take action to ensure
individuals with hemophilia who may                                                                                               • Midwest Business Group on
                                                              that employees with rare diseases get
need to self-administer treatment, such                                                                                             Health Hemophilia and Bleeding
                                                              their jobs done while also managing
as prophylactic (i.e., preventive) factor                                                                                           Disorders Toolkit
                                                              their health at optimal levels. By
infusions or on-demand factor infusions,                                                                                          • Comprehensive Care
                                                              understanding and honoring the
depending on disease severity.                                                                                                      Sustainability Collaborative
                                                              rights of rare-disease patients and
Parents and affected individuals can be                       making resources available to help                                  • Centers for Disease Control and
trained to administer infusions at home.                      them perform their duties, employers                                  Prevention, Hemophilia
Self-infusion training is a service that                      create a supportive environment where                               • Mayo Clinic: Hemophilia Best
is provided as part of HTC-delivered                          employees with hemophilia and other                                   Practices
comprehensive care. This is especially                        rare conditions can thrive.

    ACKNOWLEDGEMENTS
    With gratitude, the National Alliance acknowledges support from the National
    Hemophilia Foundation by way of clinical expertise and funding to produce this
    Action Brief. Final content was determined by the National Alliance.

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The National Alliance of Healthcare Purchaser Coalitions (National Alliance) is the only nonprofit, purchaser-led organization with a national and
regional structure dedicated to driving health and healthcare value across the country. Its members represent private and public sector, nonprofit, and
Taft-Hartley organizations, and more than 45 million Americans spending over $300 billion annually on healthcare. Visit nationalalliancehealth.org,               APRIL 2021
and connect with us on Twitter.
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