Aligning Value and Cost Considerations to Improve Access to, Outcomes of Gene Therapies

A panel at Asembia 2021 Specialty Pharmacy Summit in Las Vegas addressed challenges in the treatment journey of patients with rare diseases, efforts to improve access to gene therapies, and cost and value considerations for stakeholders.

With rare diseases affecting 30 million people nationwide, or 1 in 10 Americans, the impact of these conditions is not limited to a small subset of individuals, but a growing population of patients and families who continue to be burdened by health care access and cost issues.

Moderator Julia Wermerskirchen, PharmD, senior director, Strategic Partnerships, Optum Frontier Therapies, opened the session “Gene Therapy: How Various Stakeholders Collaborate to Impact Patient Access and Outcomes,” presented at Asembia 2021 Specialty Pharmacy Summit in Las Vegas, by spotlighting the challenges faced by patients with rare diseases and gene therapy manufacturers entering the market.

“These therapies require a heightened level of coordination, and are just logistically more complex. They come with questions about the implications of one-time treatments and concerns about durability, as well as questions about opportunities for re-dosing,” she said. “There's the dynamics of high upfront costs combined with the potential for improving not only the condition, but also potentially offsetting other high costs of care.”

With a myriad of questions pertaining to value, access, and cost, Wermerskirchen acknowledged that the rapidly growing and evolving market of cell and gene therapies requires a collaborative effort among experts across the health care ecosystem to support patients in their treatment journey and drive positive outcomes.


Wermerskirchen asked each person on the panel to discuss important considerations for stakeholders in creating patient access to these therapies. The panelists were Lisa Carman, vice president, HEOR and Value-Based Contracting, Optum Life Sciences; Brad Martin, vice president, Market Access, Specialty Trade, and Distribution, Taysha Gene Therapies; and Mindy Cameron, life sciences consultant, AdvocacyWorks.

Pinpointing key factors of keeping the patient at the forefront, standing behind your product, and distinguishing value across the spectrum of progression for rare diseases, the panelists then forayed into the patient journey. Cameron, whose son has Duchenne muscular dystrophy, addressed the steps that her advocacy group took to improve access to patients with the rare condition and challenges that persist.

“We've done natural history studies. We've provided a lot of funding for the beginnings of these companies that are now at the forefront of developing these gene therapies," Cameron said. "So, we have made a tremendous investment in these therapies, and we're seeing now that the way that clinical trials are designed, we have patients being left out of access.”

This issue of value and how payers weigh risk in clinical trial enrollment for emerging gene therapies was further discussed as Carman said that increased data collection, such as patient-reported outcomes (PROs), have helped to distinguish benefit in patients with rare diseases whose functionality cannot be measured in a simple claim.

Notably, much of these efforts in determining value for novel gene therapies has to begin at a pre-commercialization stage, noted Martin, as communicating with payers on meaningful, clinical endpoints requires a market access strategy composed of clinical development, regulatory, and ethical factors.

“You have to optimize 3 journeys from an access perspective–you have the patient journey, you have your product journey, and you have your reimbursement journey,” he said. “Without understanding the clinical package that you have with the access, you won't get rapid uptake….As manufacturers, you need an endpoint that gets you an approval, but also endpoints that support what's that value of the product to those patients.”

From a patient perspective, Cameron highlighted that knowing the entities involved in the care of patients is vital, especially as children, who represent 50% of all cases in rare disease, are typically covered by Medicaid state insurance.

Furthermore, in addressing concerns regarding cost for these expensive gene therapies, Carman noted 3 key issues to consider in bending the cost curve:

  • How the drug is purchased and who covered the cost
  • Patient outcomes and financial recovery if responsiveness to therapy is insignificant
  • Utilization management criteria

“It helps assure to the payer that if [the therapy] doesn't work, there is some risk-sharing there….The other piece, too, is over time, durability is the main question to payers,” said Carman.

Carman added that by tracking patients along their treatment journey through an outcomes-based approach of extracting data such as PROs and claims, deriving this real-world evidence, whether positive or negative, would be crucial information for coverage.

“What I can tell you is the way that my team looks at it is how do we build an infrastructure to be able to support what you guys want to launch with in the market and what payers need to be able to make decisions to get the right drugs to the right patients.”


Ultimately, identifying the different value streams to personalize care for each patient would ameliorate coverage issues in these populations, said Cameron, who mentioned further challenges of changes in insurance, payer collaboration, and tracking patients as they age as additional considerations for stakeholders.