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Promoting patient access to gene therapies for rare disorders

Optum Life Sciences experts discuss value-based contracts at the 5th Annual Gene Therapy for Rare Disorders Conference.

4-minute read

Promoting patient access to gene therapies for rare disorders

At the recently held 5th Annual Gene Therapy for Rare Disorders Conference, Erin Hulbert, Director of Value-Based Contracts for Optum Life Sciences, represented our team during a fireside chat about access to gene therapy for people living with hemophilia. After the conference, I asked Erin about some of the key themes explored during this session as well as at the conference overall.

Erin, what role does the Optum Life Sciences team play when it comes to gene therapies for rare disorders, and how did your team contribute to this fireside chat?

Within Optum Life Sciences, the Value-Based Contracting (VBC) and Health Economics and Outcomes Research (HEOR) teams leverage real-world evidence to analyze the cost and quality performance of gene therapies. There are two approved gene therapies in the U.S. today, and based on the pipeline, they’ll be joined by many more over the next several years. Our team provides ongoing information about how the treatments are working in specific populations, and we work with manufacturers to structure and support reimbursement strategies that help alleviate payer concerns over the rising cost of gene therapy.

This is where value-based contracting can be a tool that helps expand coverage: Since these therapies are so new, payers have limited evidence to lean on when making reimbursement decisions. Because of the high upfront costs of these treatments, it’s completely understandable that health plans and PBMs might be apprehensive about their use. In the worst case, the payers may end up limiting access to a specific drug. The immediate effect is that the patient is denied a potentially life-altering treatment. The longer-term impact is a vicious cycle of insufficient evidence; without patient access to these treatments, we lose the opportunity to track outcomes and generate the evidence that everyone needs to really evaluate the long-term cost-effectiveness of these therapies.

A value-based contract (VBC) helps manufacturers and payers share the risk, which can open doors to treatments for patients that may otherwise be closed.

Can you describe the specific VBC example you discussed in this session and explain what made it work?

We spoke about our work with treatments for hemophilia, a disease that prevents one’s blood from clotting normally, which can cause severe bleeding. Certain factors specific to hemophilia, like hospitalizations and bleeding events, are observable in claims data and are thus easier to track. While standard-of-care treatments can be lifesaving, they can also be very expensive. All these factors — the availability of data, the impact on outcomes and the high financial cost — create conditions suitable to the use of a VBC when these next-generation cell and gene therapies come to market.

What are some of the challenges you’ve experienced with VBCs for gene therapy?

Most of the steps required to conduct a traditional HEOR study can be applied to gene therapies, but there are 2 important differences: Gene therapies will generally involve a smaller patient sample and will require an extended period of time to trace outcomes.

Working with groups this small makes the analytics very different from what you might see in a standard VBC; for more traditional VBCs, we often calculate a population statistic and then apply that to all the claims for that therapy. With gene therapies, the small sample size introduces variability for all the stakeholders — including both the payer and the manufacturer — and that makes it especially important to capture every outcome, both the successes and the failures. 

The longer post-treatment observational window introduces complications, too. Several million plan members and their dependents change health plans each month, so it can be difficult for any one insurer to track outcomes over time.

Following patients long enough to observe the durability is important, since there is a possibility that gene therapies aren’t always one-and-done. Some patients may require additional treatments.

What have you learned so far to help you address some of these challenges?

We’ve learned that it takes a long time to fully assess gene therapy outcomes and get to a full picture. These contracts and studies require a thoughtful, big-picture approach, so it’s important to work through the analysis with manufacturers prior to approaching a health plan; this way, manufacturers can see what the data look like and let that inform their approach to the VBC.


Optum can identify and track patients who have received cell or gene therapies and consistently monitor the success or failure of these therapies. Our technology will allow reporting on long-term patient outcomes to support VBCs, even in the event that a patient changes health plans.

What are some other ways your team is leveraging Optum data and analytics to support manufacturers with cell or gene therapies?

We use claims and clinical data to identify analytic populations of patients who might be eligible for gene therapies in order to describe the current cost of care and burden of illness. We can see how clinicians diagnose and treat patients with rare diseases today; this helps us determine potential metrics, like a specific lab or test, that can support VBCs. These metrics can also help manufacturers figure out if they need to educate clinicians or change provider behavior in order to ensure those critical metrics get captured at the right times. Our team creates data models for this type of work, too, so that we can look at potential scenarios and help design win-win contracts between payers and manufacturers.

How did your session fit into the larger Gene Therapy for Rare Disorders Conference?

A handful of other sessions had real-world data themes like ours. It was a great conference to attend because of the breadth of topics — there are so many innovative therapies in the development pipeline. One of my favorite parts about attending these events is simply getting to be with others who are passionate about this topic. There’s so much being done — which is why it’s important for Optum to be a part of the rare disease community and have a voice in all these important conversations about bringing innovative cell and gene therapies to market.

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To support patient access, we need to track outcomes the right way. This means choosing the right metrics that are routinely captured by providers and following patients for long periods of time. The work that Erin and the rest of our VBC and HEOR teams are doing will help manufacturers — and all health care stakeholders — create agreements that help providers deliver appropriate, cost-effective treatments to patients who need them most.

Work with us on your value-based contracting needs

About Erin Hulbert

Director, Value-Based Services, Optum Life Sciences


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