Key Takeaways from the AMCP Annual Conference

The Academy of Managed Care Pharmacy (AMCP) hosted its 25th annual conference in Houston, Texas, March 31-April 3.

The annual conference of the Academy of Managed Care Pharmacy (AMCP) in Houston, Texas, delivered a wide range of educational sessions alongside opportunities to better understand emerging trends and challenges affecting the pharmaceutical and managed health care sector. Three subjects caught our attention: chimeric antigen receptor T-cell (CAR-T) therapies; real world evidence (RWE); and the healthcare provisions of the Inflation Reduction Act (IRA).

Prepare for more CAR-T therapies

With the rapid growth of cell and gene therapy approvals, particularly chimeric antigen receptor T-cell (CAR-T) therapies, the US healthcare system is at a pivotal point. CAR-T therapy, a type of immunotherapy that uses the patient’s genetically modified T-cells, offers potentially curative outcomes for patients with severe or refractory conditions, but comes with high financial costs. The conference heard that, as the CAR-T therapy pipeline expands and treatment protocols evolve, managed care organizations will need to develop strategies to balance such innovations with the ability to cover their high costs and offer treatment to patients in an equitable and sustainable way.

At a conference session titled: “Chimeric Antigen Receptor T-Cell (CAR-T) Therapies: Perspectives and Challenges in Managed Care”, speakers examined the total cost of care and treatment episodes for Food and Drug Administration (FDA)-approved CAR-T therapies. They detailed an evidence-based view of CAR-T therapies through the lens of Milliman’s Consolidated Health Cost Guidelines database, a medical and pharmacy claims analysis. AMCP faculty and key opinion leaders discussed how managed care health plans are already adapting their coverage and management approaches to accommodate existing CAR-T therapies, as well as how they are preparing for new approvals and ongoing advancements in cell therapies.

Key themes discussed at AMCP included:

1. Utilization Management Strategies
Speakers provided an in-depth look at how healthcare payers design and evolve prior authorization criteria to ensure clinically appropriate use of therapies while controlling costs. The session highlighted the balance between access and affordability, emphasizing evidence-based approaches and real-world impact.

2. Centers of Excellence (COE)
The discussion examined the growing role of COEs in driving standardization of care, improving clinical outcomes, and minimizing variability in treatment delivery. COEs were also presented as effective models for containing costs and enhancing overall healthcare quality.

3. Case Management Strategies
Speakers emphasized the value of proactive case management, including coordinated care models, patient navigation, and adherence programs. These strategies were shown to reduce hospital readmissions, prevent adverse events, and support better patient engagement across complex therapeutic areas.

4. Benefit Design Considerations
Insights were shared on how payers are navigating the challenges of aligning pharmacy and medical benefits. Key topics included member cost-sharing structures, value-based insurance design, and reinsurance strategies to improve access and manage financial risk for high-cost therapies.

5. Long-Term Safety

Given the unknown mechanisms of cell and gene therapies on influencing our biological systems, regulators and payers have expressed uncertainty about the long-term adverse effects of these medicines. Cancer malignancies in particular may develop over an extended period of time, requiring longitudinal follow-up data to observe such low latency adverse events (e.g. over a 15 year time horizon). European data (e.g. in the Netherlands, Nordics) often has better longitudinal depth than many US claims sources, offering the promise of long-term safety observations and a deeper understanding of the impacts of CAR-T therapies.

The session was designed to update attendees and to enable them to:

Analyze the drivers of variability in episodic costs for CAR-T treatments across different populations and settings
Evaluate the ongoing and emerging challenges managed care leaders face when integrating high-cost, high-impact therapies into formularies and care pathways
Apply best practices in prior authorization, network design, and member engagement to support appropriate utilization and access

Getting ready for future launches of novel CAR-T therapies requires cross-functional collaboration, robust data analytics, and forward-looking policy development. Payers must remain flexible and ready to combine evidence-based strategies with operational innovation to ensure patients have access to the latest CAR-T therapies and up-to-date insights from longitudinal follow-up of exposed patients in the real world.

Real world evidence adds value

Since 2017, the FDA has published a series of guidance documents on RWE and RWD. The FDA defines RWD as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. Examples of RWD include data derived from electronic health records, medical claims data, data from product or disease registries, and data gathered from other sources (such as digital health technologies) that can inform on health status.” ^[i]^,[ii] . According to the FDA, the guidance is intended to help realize “the full potential of fit-for-purpose RWD to generate RWE that will advance the development of therapeutic products and strengthen regulatory oversight of medical products across their lifecycle.”

Within the industry, there is general agreement that RWE can support value demonstration of pharmaceuticals and medical devices that may be used to impact decision-making by health plans and pharmacy benefit managers. Currently, US payers, while seeing the value of RWE, do not fully understand how to interpret and incorporate RWE routinely in their appraisals and decisions – more guidance is needed. To help close this gap, AMCP has developed standards for using RWE in payer healthcare decision-making, soon to be published in the Journal of Managed Care & Specialty Pharmacy. The AMCP guidance has been developed by a task force of US payers and pharmaceutical companies and will outline a framework and criteria for RWE to support RWE planning, generation and payer education. Notably, Lumanity has previously synthesized key RWE frameworks into a unified assessment criteria for selecting fit-for-purpose regulatory and payer-accepted datasets.

While the gold standard of evidence for supporting decision-making is efficacy, safety and tolerability from randomized controlled trials (RCTs), limitations in developing these trials (such as budget, time, and resources) restrict comprehensive outcome collection, especially when considering the performance of treatments over time in a real-world setting. Thus, relying on data from RCTs only can lead to uncertainty in payer decision-making based on gaps in evidence.

Delegates at the AMCP conference heard how RWE can help to close such gaps, leading to more informed decisions:

Selection of fit-for-purpose datasets paired with high-quality analytic methods can result in meaningful RWE that fills evidence gaps, illustrating how treatments perform in the real world, especially during post-launch and over long-term time horizons. This may also reflect or confirm current clinical practice
RWE can act as “homework studies” to understand the patient population, identify an optimal product positioning, and inform the design of more robust clinical trials (e.g. subgroup definition, incorporation of payer-relevant outcomes). Evidence generated earlier in the product lifecycle can shape pharmaceutical and investor understanding of unmet need, burden of disease, natural history, health care resource utilization (HCRU) and costs of disease – and provide important contextual evidence in regulatory submissions and payer evidence packages
RCTs often have limited follow-up data and limited sample sizes typically representing a homogenous patient population. RWE can track a higher volume of more representative patients with the possibility of long-term data that matures over time. This can address critical questions of drug utilization, effectiveness in different populations who may have been under-represented in RCTs, adherence, HCRU, and long-latency adverse events that may develop years after exposure

Notably, RWE has become more commonly submitted to payers in recent years, as seen in the updated AMCP dossier format v5.0.

From a regulatory perspective, as previously mentioned, the FDA has issued guidance and has an RWE program in regulatory considerations. As part of the guidance developed from the IRA, the Centers for Medicare & Medicaid Services (CMS) evaluates RWE as part of a manufacturer submission during Medicare drug price negotiations (MDPN). As observed in the explanations of the maximum fair price (MFP) from the first cycle, many references of RWE are considered, as this evidence reflects recent patient use and real-world practice. CMS is likely to continue considering RWE during future MDPN alongside supporting evidence from RCTs.

Combining RWE with RCTs has benefits

A consideration that may be useful is to combine RCT and RWE methods – RCTs and RWE should be complementary rather than mutually exclusive. For example, pragmatic trials could incorporate elements of both RCTs and RWE. These trials would use randomization but follow up with RWD collection methods to assess outcomes. It is worth noting that this type of long follow-up design is already performed routinely as part of post-authorization efficacy studies (PAES) and post-authorization safety studies (PASS). Tools like target trial emulation support the conditions to replicate an RCT in a real-world setting. If an RCT is not feasible, advanced statistical methods applied in RWD can result in high-quality, meaningful RWE. For example:

In rare diseases, synthetic control arms can be a powerful tool for creating a real-world benchmark for comparison of the trial population
In regulatory-facing PASS or PAES, target trial emulation is increasingly being used as a tool to mimic the conditions of an RCT, but in an observational context. It may be possible to follow up on patients from the trial itself, leveraging solutions like tokenization to identify trial participants in a real world setting

RWE can provide insights to the real-world safety, efficacy, and adherence of drugs across a broader patient population, complementing the more controlled scope of RCTs and contributing to a fuller snapshot of the impact of a drug on patients.

The Inflation Reduction Act affects decision-making

The conference discussed the healthcare provisions of the IRA and its impacts on US drug pricing. The reforms are heavily impacting commercial decisions in terms of pipeline reprioritization for Medicare-heavy therapies, reassessment of launch pricing strategies, and increased payer scrutiny of value and outcomes.

The IRA has empowered CMS to negotiate down prices for high-spend Medicare drugs after a set number of years, effectively eroding pharmaceutical company revenues for brands that are heavily prescribed for Medicare beneficiaries. Manufacturers are also subject to Medicare rebates if their drug prices rise faster than inflation. In practice, this constrains pricing flexibility and impacts the approaches pharmaceutical companies have traditionally used to manage margin and lifecycle revenue.

Under the IRA’s Part D redesign, more financial risk is shifted to health plans and manufacturers. This shift may lead to greater formulary management, higher premiums, and cutbacks to manufacturer-funded patient assistant programs.

Price negotiations

The IRA may discourage innovation. With an unknown financial environment 9 years after the first approved indication, manufacturers have little incentive to invest in small molecules in the future
The IRA may have inadvertently made formulary access more restrictive for drugs selected for Medicare price negotiations versus therapeutic alternatives. One study conducted by the National Pharmaceutical Council^[i] assessed patient access to 10 negotiated drugs compared to their potential therapeutic alternatives and found that, prior to MFP, the chosen drugs generally had broader coverage and less restrictive utilization management in 2024. However, post-IRA negotiations, it is expected that having an MFP may reduce rebate flexibility and put these drugs on less favorable tiers (possibly resulting in more restrictive coverage)
Manufacturers must offer drugs to covered entities at the lower of the MFP or the 340B ceiling price. Prior to the IRA, there was considerable margin for 340B covered entities due to discounts. However, post-IRA, there is less margin for these drugs. Consequently, there may be an incentive for payers to push patients off MFP drugs using utilization management for higher rebate or higher list drugs

Conclusion

The AMCP annual conference in Houston, Texas, delivered on its promises to offer continuing education opportunities on emerging trends and challenges and insights to navigating the fast-paced developments of managed care, while also showcasing the latest products and services designed to improve patient outcomes. For the 4,000 or so payers, pharmacists, manufacturers, academics and others drawn to the event, the discussions were thought-provoking. We left feeling energized by the promise of CAR-T therapies and the expanded impact of RWE on payer decision-making.

References

[i] US Food & Drug Administration, Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices | FDA

[ii] US Food & Drug Administration, Real-World Evidence | FDA,

Selected Guidance

Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices

Framework for FDA’s Real-World Evidence Program

Use of Electronic Health Records in Clinical Investigations

Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products

Data Standards for Drug and Biological Production Submissions Containing Real-World Data

Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products

Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products

Submitting Documents Utilizing Real-World Data and Real-World Evidence to FDA for Drugs and Biologics