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.
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.
~4,000 diseases are linked to gene disorders—many of which lack any effective treatment
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.
Gene therapies, today and tomorrow
By supporting and encouraging evidence-based access to gene therapy, payers and other segments of U.S. society will be positioned to benefit from future advancements and biopharmaceutical breakthroughs that drive systemic change.
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.
“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
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
The $5.2B gene and cell therapy market is estimated to grow tenfold by 2031
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.
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
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.
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:
Zolegensma® improves life expectancy for spinal muscular atrophy (SMA) in infants, the leading genetic cause of infant mortality.
Roctavian® was the first gene therapy for the treatment of hemophilia A, a rare genetic bleeding disorder that primarily affects males.
Lenmeldy®, one of the most expensive gene therapies on the market, treats children with the rare genetic disease metachromatic leukodystrophy (MLD).
Casegevy® and Lyfgenia® were developed to treat sickle cell disease, a group of inherited blood disorders that affects approximately 100,000 people in the U.S.
Ensure access to breakthrough gene therapy drugs while protecting your plan and members from high costs with our Embarc Benefit Protection solution.
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.
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.
