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Managing cell and gene therapies (CGT)

A diverse pipeline of CGT is coming to the market.

Posted: September, 2022

Effectively managing cell and gene therapies

Health plans and sponsors are dedicated to providing members with access to CGT across a variety of diseases. But often, CGT comes with an ultra-high price tag. CGT are expected to grow from $4 billion a year in sales in 2021 to over $45 billion in 2026.*

For health plans to effectively manage access to CGT, it will be essential to understand real-world patient outcomes while managing significant financial volatility.

Health plans of all sizes agree that outcomes-based contracting strategies are needed to manage the emerging pipeline of CGT, but what can a health plan do to offer members access to CGT, collect real-world evidence (RWE) and monitor outcomes?

It's complicated

Every stakeholder has an interest in ensuring members get access to the best care, including life-saving CGT. Health plans and sponsors want to provide the appropriate members access to CGT. But getting there is the challenge.

Providing access to these expensive therapies creates two major concerns for health plans:

  1. Confirming that members are achieving the outcomes for which the health plan is paying 
  2. Managing the details, risk and expense that comes with each new CGT and its corresponding patient program 

Traditional rebate programs alone don’t cut it

Traditional pharmacy rebate programs can provide some cost relief for high-cost therapies to health plans and sponsors. But traditional rebate programs alone don't work well for CGT because of their one-time dose and long-term potential cost offsets.

Such programs don't work because they: 

  • Apply a uniform discount to each prescription, whether or not the therapy works for the patient
  • Don’t track patient outcomes over time
  • Don’t inform appropriate management strategies for CGT

Pay for outcomes, not for treatments

Outcomes-based contracts require life science manufacturers to share in the risk of potential CGT treatment failures. They also provide ongoing information about how treatment is working within a health plan’s own populations.

However, all health plans, small or large, meet challenges in implementing CGT programs. 

Smaller health plans may have limited experience with outcomes-based contracts. Or they don’t have the resources or bandwidth for this type of initiative, so they may need help getting started. Larger health plans may be more likely to serve members who are prescribed a CGT.

But a large health plan's challenges are in two key areas:

  • Aligning with manufacturers to identify meaningful and measurable real-world outcomes around which to share risk
  • Having infrastructure, analytic resources and expertise to follow members consistently and impartially over time?

Monitor outcomes with real-world evidence

Real-world evidence (RWE) is critical when monitoring outcomes and measuring CGT performance. In providing members access to CGT, a health plan's value proposition is in offsetting expensive disease-related care over the years of improved health they provide.

Monitoring for CGT needs to occur over a longer period of time, following the patient journey. Depending on the CGT, it can be three years to as many as seven years. A lot can change during that time.

Members may move from health plan to health plan or provider to provider over time. Once members get access to CGT, ongoing member health monitoring to collect RWE becomes a powerful tool. It can help inform ongoing utilization management and help us understand the value of each CGT program. 

CGT are different than other treatments we’ve seen so far. A dedicated infrastructure is needed to monitor and measure patient outcomes.

Example: Long-term monitoring of members with hemophilia

Long-term monitoring is needed after a member with hemophilia receives CGT. Health plans need to be able to collect outcomes data long-term for these members who received CGT, including:

  • Blood factor levels
  • Factor replacement therapy
  • Bleeding events
  • Surgeries

Members may also change providers or engage several providers in their care. For example, their gene therapy provider may not be their primary hematologist. If members change health plans, it may be difficult to measure long-term costs. 

One solution manages all your CGT programs

Optum has a solution to help health plans collaborate with drug manufacturers and manage risk associated with the high cost of medical specialty drugs, including CGT.

Our single platform approach helps health plans avoid having to establishing a new effective data process for each CGT. It enables outcomes-based contracts to be organized by individual therapeutic programs (for example, hemophilia, sickle cell disease or spinal muscular atrophy) versus a single CGT.

The solution can help health plans and life science manufacturers by supporting:

  • Longitudinal patient-level tracking
  • Clinical and claims-based outcomes measurement
  • An auditable process to support financial reconciliation, where all parties can trust the data

Expertise from a trusted partner

Optum is committed to providing solutions that support health plans and life science manufacturers to get the right treatments to the right patients at the right time.

Existing relationships with health plan clients facilitate our outcomes-based solution by providing data to support our patient journey analytics. Optum may also collect medical records from providers for additional information. This is where the scale of the Optum risk and quality organization comes into play. 

When claims data alone are not enough to measure outcomes, we tap into this infrastructure to collect the necessary charts and abstract relevant information. 

As health plans continue to offer members access to new CGT, they can do so confidently knowing they have the trusted expertise of Optum.

Find out more about how you can take control of your emerging medical specialty drug spend:



About the author

Erin Hulbert, MS
Director, Value-Based Services, Optum

Erin Hulbert leads the value-based services (VBC) team, which measures outcomes to support rebates for value-based agreements as well as partners with life sciences companies to successfully navigate the contracting landscape and communicate the value of their products to payers. Hulbert joined Optum in 2010 and was previously a member of the Health Economics and Outcomes Research (HEOR) team. She has extensive experience in using RWE outcomes analysis and research using observational and prospective methodologies including administrative claims, clinical data, medical charts, surveys and clinical trials across a variety of therapeutic areas.

*Kemler S, Lohr A. Cell and gene therapy investment outlook in 2022 and beyond. Cell & Gene. Feb. 21, 2022.