Six steps healthcare leaders should consider before launching a cell and gene therapy program | Viewpoint

Emerging novel cell and gene therapies allow for the possibility of untethering patients from a lifetime of burdensome, costly chronic care with a single treatment.

Alex Pham

The authorization of over thirty-five cell and gene therapies and the imminent approval of several additional advanced-stage gene therapies have left many hospitals and healthcare systems evaluating the cost-effectiveness and viability of these therapies prior to venturing into this burgeoning market segment.

While new gene therapies are shown to be very promising, the full clinical impact of these treatments is not yet known, driving questions on coverage, cost efficiency and delivery. Many health systems are also not set up to coordinate the procurement of a novel gene therapy, or they may not fully understand the complexity of implementing a gene therapy program.

While the pipeline changes continuously, 17 cell and gene therapies are predicted to come to market in 2024, and by 2030, 54 approved cell and gene therapies are expected in the FDA pipeline. It is essential for healthcare institutions to adequately assess their readiness to deliver a cell and gene therapy program by conducting a thorough evaluation of their capabilities and strategic inclinations towards market entry.

Here are six steps to building a successful gene therapy program that health care leaders should consider.

1. Determine readiness to enter the market

Understand that the procurement of a cell or gene therapy drug is not the only end goal and that determining readiness requires broad cross collaboration across the health system.

Assessing the operational infrastructure and readiness to start a successful cell and gene therapy program is vitally important. Building a governance committee consisting of executive-level and department-level experts can help leaders better determine a health system’s readiness. Hospital departments that are needed at the table to engage in the collaborative process include medical, pharmacy, legal, procurement, finance, business development and strategy.

Implementing gene and cell therapies and more importantly ensuring that they perform to clinical and financial expectations will truly test a health system’s integration and collaboration framework.

2. Identify specific domains where the health system possesses inherent strengths for a successful program launch

One of the most important factors for a health system’s success in implementing a cell and gene therapy program lies in the strengths of the health system’s competencies and specialties.

A deep understanding of the strengths and specialties of the provider group is necessary as many sub-specialties are leveraged for programs of this scale.

Additionally, the organization will want to understand other specific domains of strength which could include an established research or academic program within the health system; strong collaboration between medical, pharmacy and the executive leadership team; financial and patient navigation services; as well as, a highly complex patient population and an established specialty pharmacy program.

3. Develop a detailed roadmap of the patient journey and interventions tailored to the health system's needs

This roadmap should be meticulously tailored to the unique characteristics and aspirations of a health system, including infrastructure requirements, staffing considerations, regulatory compliance, patient care protocols, quality assurance measures and highlight the potential gaps in capabilities for selected therapies.

Cell and gene therapies often require highly complex care coordination not only within the health system, but also with other outside partners such as the manufacturer and others. Even still, each therapy has individualized requirements. Having a foundational roadmap will help for growth and scale for continued expansion and innovation.

4. Decipher financial billing strategies tailored to each selected cell and gene therapy

Navigating a potentially multi-million-dollar drug therapy involves a multi-disciplinary approach that can be extremely time intensive and costly for health systems.

Health care systems should understand payor dynamics and have the capability to negotiate directly with payors. Often, these therapies carry risk that should be considered when coverage is negotiated with the payors. This often requires working closely with a managed care group that understands all potential costs that is informed by a multi-departmental revenue integrity team so that it may communicate with the payor directly.

Other considerations include prior authorizations, geographic complexities, patient financial access, assessment of proper staff training, legal and risk management, orchestration of stakeholders and devising a procurement pathway.

Determining the optimal billing structure for selected cell or gene therapies is another important step. Factors such as treatment costs, reimbursement rates, payer mix, revenue projections and funds flow, provide invaluable insights to support strategic decision-making, reduce risk and support financial sustainability. Relevant financial modeling that considers both private and public payors should be evaluated to inform the most sustainable billing structure recommendations.

5. Market health system as a distinguished treatment center with manufacturers

Fostering strategic partnerships with manufacturers to bolster a health system’s reputation and service offerings within the cell and gene therapy landscape can go a long way to ensuring a health system gains access to these new therapies.

The cell and gene manufacturer landscape is a broadening, but narrowly focused market. Some manufacturers only exist to develop a single therapy and the pipeline changes weekly. There is market share to gain through stratifying a health system’s patient population and serving as a distinguished treatment center. Conducting a comprehensive market analysis and ensuring the team has the expertise in place to negotiate and forge strategic partnerships will be critical to program success.

6. Prepare to mitigate risks that drive unnecessary cost, diminishing the clinical and financial values of a program

As gene therapy clinical trials are increasingly on the rise, little is known about the long term risks associated with these treatments, and more research is needed to further understand the potential delayed effects of these new therapies.

For example, the phase ½ and phase 3 clinical trials of BlueBird Bio’s LentiGlobin gene therapy for sickle-cell disease had to be discontinued in 2021 after two patients were diagnosed with cancer five years after receiving treatment. Although many cell therapies are well understood, some carry significant financial burden even post-treatment and may not provide curative treatment.

Still, considerable advances have been made in the approach for several disease states such as neuromuscular disease and blood disorders. Health systems should take care to understand the safety risks and long term effects of these treatments to help balance the risks and avoid unnecessary costs later down the road.

Conclusion

A successful cell and gene therapy program will involve regular communication and collaboration within the health system to ensure alignment, address any emerging challenges and drive progress towards the defined objectives of the program.

A thorough financial and clinical readiness evaluation prior to launching a program, and equipping the health system with the knowledge, resources, and strategic direction necessary to establish itself as a reputable cell and gene therapy provider, will foster enhanced patient care and organizational success within this innovative domain.

Alex Pham is vice president, client strategy of Clearway Health.