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In-Depth Resource for Gene Therapies

GENE THERAPY HUB

Gene therapies are transforming the pharmacy industry in more ways than one.

In order to navigate these new-to-market therapies confidently, you need trusted resources and data. Read on to better understand the impact of new gene therapies on your member population-and what you can do about it. 

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OVERVIEW

What are gene therapies?

Gene therapies are innovative, highly targeted medications that can edit genetic material in a patient’s affected cells and tissues. They have a long history of research and development, and the availability of genetic therapies for both rare and common genetic conditions is about to take off.

Gene therapy has the potential to treat—and cure—some of these disorders, often the most debilitating diseases patients face.

Payment models and patient care systems need to be thoughtfully reimagined and strategically implemented, where it makes sense, to make gene therapy accessible for individuals who would otherwise lack access to these life-changing and life-saving innovations.

Evernorth helps our clients navigate through this complex emerging landscape. We'll be your guide from pipeline tracking and planning for your impact through distribution and management solutions.

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DNA strand

~4,000 diseases are linked to gene disorders—many of which lack any effective treatment

A life-changing—but expensive— treatment option

While gene therapies promise extraordinary outcomes, few can afford their high price tags, including ones costing $850K and $3.5M per treatment.

30x
the median U.S. household income is spent per person
$5B
to research and develop a gene therapy

Preparing for the impact of gene therapies

Gene therapies are quickly coming to market, and with them the potential for life-changing outcomes for some of the most debilitating conditions patients face. With these new innovations, however, come complexities for patients, their caregivers and payers. Talking Specialty: Gene Therapies discusses what you can do to prepare now and how Evernorth’s suite of solutions and services can set you up for success.

Covering the basics on cell and gene therapy
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Want to know which pipeline drugs to watch?
Access our latest gene therapies pipeline update.
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FEATURED ARTICLE

Innovation in hemophilia drug development and cost-efficiency tools

As treatment for hemophilia evolves, data proves a good specialty pharmacy partner can help minimize waste and drive efficient practices.

DETAILED BREAKDOWN

Explore the tabs to understand the gene therapy landscape to make impactful, informed decisions.

Overcoming the costs | Preparing for impact | Simplifying member experience

Overcoming the costs

The need for coverage

Plans have a spectrum of coverage options to consider with the influx of novel gene and cell therapies. By offering proactive and comprehensive coverage, plan sponsors can reduce their own risk and control costs while ensuring their members have access to these groundbreaking treatments.

Have this conversation with your Evernorth account team soon. Equip your decision makers with the right resources, pipeline insights and sophisticated modeling tools, so you can develop a clear vision for how your organization will handle gene therapy coverage. Your plan will need the right protection from million-dollar price tags, as well as a path to expand coverage for members.

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Cost management

Each year comes with a new wave of approvals to plan for. Planning now for your approach to gene therapies will help you manage costs more predictably in the future.

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Expanding access

Making a coverage plan for gene therapies supports health care technology that can engineer bodies to fight cancer and reverse rare diseases—all while giving hope to so many who didn’t have it before.

Competitive benefits

Including gene therapy coverage in your benefits can help set your organization apart by illustrating your strong commitment to the health of your employees and their families.

Jennifer Iverson

“When families face a rare and devastating diagnosis, they should be able to begin treatment promptly, without worries about how to afford vitally needed medications.”

–Jennifer Iverson, PhD, Sr. Director, Specialty Drug Management, Express Scripts

Related articles on this topic:

Preparing for impact

Making the gene therapy pipeline actionable

Developing a well-informed, proactive approach is critical for plan sponsors to assess their potential exposure to gene therapy. Every population is different, and the pipeline will continue to grow.

Plan-specific forecasting tools keep you informed of future impact to your members, because gene therapies are no longer only for rare cases. Nearly a quarter of the current pipeline will affect large populations and therapy areas—including cardiology, metabolic disorders, neurology and rheumatology.2

2. https://newdigs.mit.edu/sites/default/files/FoCUS_Research_Brief_2017F211v011.pdf

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50-75
gene cell therapies expected to be approved in the U.S. by 2030
1,000+
gene and cell therapies currently in development
Gene Therapy Discussion Guide

Not every approved gene therapy will be appropriate for everyone, as other effective treatment options may be available at a more affordable price

Each drug’s expected utilization should be evaluated based on patient demographics, disease severity, client-specific mix and clinical trial data and literature. Sophisticated forecasting models that assess these areas provide a better estimate of potential gene therapy utilization than traditional tools.

Related articles on this topic:

Simplifying member experience

Removing the burden for patients and plans

Gene therapies and the conditions they treat are distressing enough without the added challenge of coordinating care and benefits. Faced with a rare condition diagnosis, some patients try to navigate the health care system themselves. It’s a complex and confusing journey where many struggle to understand their coverage and options.

The average time to receive a diagnosis for a rare condition is 7.6 years, which can feel like a lifetime when looking for answers.3

3. globalgenes.org/2013/04/10/rare-disease-impact-report-quantifies-patient-and-caregiver-challenges

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Supply chain pharmacy technology

Not just any specialty pharmacy is up to the task of managing and handling the fulfillment of gene therapies. Companies rely on robust supply chain capabilities to properly preserve and regulate products as they travel to the patient.

Read our article about the Proper Care for Life-Saving Innovation 

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More access, plus extra care

Evernorth is home to the only specialty pharmacy with access to the majority of approved gene replacement therapies to date—and one of a few pharmacies with experience in handling, packaging and shipping these sensitive medications.

Ask for a virtual tour of Accredo to get a behind-the-scenes look

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Leading distributor

As the first and only distributor of the first approved gene replacement therapy, CuraScript SD offers a unique model to satisfy the high-cost, complex fulfillment needs that go along with these therapies.

Learn about our comprehensive distribution solutions

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At Evernorth, our solutions ease the journey to connecting those with rare conditions to life-changing drugs—from access, to distribution, to utilization.

It’s in our DNA to help members live their best lives. We understand the complexities that make it difficult to coordinate gene therapy care and coverage for both plans and members. Let us guide you.

Frequently asked questions

Gene therapies introduce genetic material into a person’s DNA to “edit” and replace faulty or missing genetic material that leads to disease. Unlike nearly all other medications that are repeatedly taken over time, these therapies typically are administered once with the intent to provide long-term benefit, and in some cases, a cure.

For example, one of the first gene modification therapies on the market was Luxturna®, which treats Leber congenital amaurosis (LCA). LCA is a rare, inherited eye disease that can lead to severe vision loss or blindness. LCA is caused by a mutation of a retinal gene that shares responsibility for providing instructions to form various proteins involved in healthy vision. Luxturna, which is administered through a medical procedure one eye at a time, modifies the defective gene so the body can begin producing the necessary proteins. This prevents or drastically slows the rate of vision loss.

A research scientist drops specimen into pipettes.

Gene therapy can be used to treat conditions within a wide range of therapy classes. A growing number of FDA-approved gene therapies on the market today, such as:

A wide open eye

Luxturna® can cure or dramatically slow the progression of inherited retinal dystrophy—a rare condition causing blindness

 

The human brain

Zolegensma® improves life expectancy for spinal muscular atrophy (SMA) in infants, the leading genetic cause of infant mortality

 

A drop of liquid

Zynteglo® treats beta thalassemia, a blood disorder that affects the body’s ability to make hemoglobin

 

The human brain

Skysona® for patients living with cerebral adrenoleukodystrophy (CALD), a rare and devastating neurologic disease

All four treatments are covered under our Embarc Benefit Protection solution.

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The average cost of developing a successful drug is $2.6 billion. For gene therapies, the average cost of research and development soars to $5 billion, which largely gets passed on to patients and their insurance providers.

Gene therapy differs from other targeted medicine as its treatment is intended to provide long-term benefit by altering the specific genetic defect. To justify clinical cost and risk, conditions appropriate for gene therapy usually have high rates of mortality or progressive co-morbidities, such as permanent organ damage and severe disability.

They may also require complex and expensive medical treatment and other interventions. While gene therapies cannot reverse existing organ damage or disabilities, they can prevent the need for long-term adherence to complex maintenance medications, improving the patient’s quality of life.

With such high risks and rewards associated with these conditions, gene therapies can carry prices from $850,000 to $3.5 million or more per treatment.

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Orphan status gene therapy drugs are used to treat extremely rare diseases. For example, AADC deficiency affects how a person’s cells send signals to each other. People born with this condition do not make enough aromatic l-amino acid decarboxylase (AADC), an enzyme that is critical to brain and nervous system function. AADC affects just 1 in 11 million.

Another example is X-linked adrenoleukodystrophy, which affects the nervous system and adrenal glands, leading to progressive loss of the protective fatty tissue that surrounds nerves in the brain and spinal cord. The incidence is 1 in 318,000 male births.

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Gene therapy articles
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Take the next step in supporting life-saving innovations

We can help you prepare and plan your gene therapy strategy to cover your members.