AMCP takes aim at developing new frameworks for Medicaid funded sickle cell gene therapies, real-world evidence, and AI in formulary decision making

AMCP Nexus 24, the recent Academy of Managed Care Pharmacy (AMCP) conference in Las Vegas, USA, discussed some transformative topics – among them cell and gene therapies for sickle cell disease, making intelligent use of real-world evidence in formulary decisions, and the user-friendly application of Artificial Intelligence (AI) to health plan formulary decision making. These topics were tied together by a focus on patients and the steps US States, health plans and the pharmaceutical industry need to take to improve patient-centered care.

Clear messages from the conference were that the AMCP is in the process of creating a Market Access framework for incorporating use of data, new gene therapy treatment plans, payer strategies, and coverage initiatives within sickle cell disease. For other health conditions, conference sessions focused on real-world evidence (RWE) showcased how the AMCP is creating a Real-World Evidence Framework for incorporating use of real-world data (RWD) into its unapproved and approved dossiers. This “RWE Initiative” aims to develop an education curriculum “to elevate payer skills and confidence in utilizing RWE in Access Decisions”. The education program aims to equip payers with the knowledge and skills needed to use RWE standards in evaluating formulary access. The work is a collaboration between the AMCP, health plans, integrated delivery networks, pharmacy benefit managers and RWE experts. Here are our key takeaway messages from AMCP Nexus 24.

Sickle cell disease

Sickle cell disease has long presented significant challenges for patients and healthcare providers alike. This genetic condition primarily affects people of African, African American and Caribbean decent and is characterized by the production of sickle shaped red blood cells. These misshapen red blood cells carry less oxygen, and cluster and block the patient’s veins leading to debilitating pain, frequent infections, and organ damage, often requiring hospitalization when the patient is in a sickle cell crisis.

Some 20 years ago (2004) the US Congress passed legislation which was signed into law creating an optional health benefit on the Medicaid program for patients with sickle cell disease. The legislation was intended to enable US States to cover the costs of sickle cell patients’ life-saving blood transfusion treatments, genetic counseling, testing and other treatments and services for people with sickle cell disease.

Increase access to gene therapies

Against this backdrop, speakers at AMCP Nexus 24 discussed the new treatment landscape for sickle cell disease which is seeing the emergence of targeted cell and gene therapies. The Centers for Medicare & Medicaid Services’ (CMS’) Innovation Center is to pilot outcomes-based agreements for cell and gene therapies. Data for 2024 shows some 60% of the Americans who have sickle cell disease are enrolled in Medicaid.

The pilot is intended to reduce hospitalizations and the co-morbid conditions stemming from sickle cell disease which currently cost the US health system some $3 billion per year. If successful, these outcomes-based agreements could enhance affordable access to cell and gene therapy treatments that are potentially life-saving and transformative for patients and their caregivers. Gene therapies for treating sickle cell disease and other complex conditions offer significant potential to enhance patient outcomes and reduce long-term healthcare expenditures. However, the high costs associated with these therapies can create financial challenges for US State budgets.

In the upcoming year, the CMS will collaborate with participating US States and manufacturers to establish a framework aimed at increasing access to gene therapies for sickle cell disease. As part of this model, the CMS will negotiate an outcomes-based agreement with participating manufacturers, linking the pricing of treatments for sickle cell disease to their effectiveness in improving health outcomes for patients treated under Medicaid. The overall aim of this market access initiative is to bridge the gap between social health care programs, patient access to new treatment plans, and improving the standard of care for patients with sickle cell disease.

The CMS plans to tackle additional care delivery gaps and challenges faced by sickle cell patients receiving cell and gene therapies during the outcomes-based agreement negotiation process. This includes requiring manufacturers to provide a clearly defined range of fertility preservation services for patients undergoing gene therapy for sickle cell disease. Moreover, CMS will offer optional funding to states that undertake initiatives aimed at enhancing equitable access to cell and gene therapies and promoting comprehensive, multi-disciplinary care for patients treated under Medicaid who have sickle cell disease and who are receiving gene therapy. These initiatives may involve expanding or increasing reimbursement rates for optional Medicaid benefits and services, such as behavioral health and care management services.

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Leveraging AI and RWE in Health Plan Formulary Decision Making

In other developments, audiences at AMCP Nexus 24 heard how AI is transforming the pharmaceutical industry – its impact is perceptible from drug discovery to drug dispensing, patient care and counseling. AI can enhance how pharmacy and health plans make informed, evidence-based formulary decisions. Essentially, the conference concluded that AI can help decision makers with its efficient aggregation and analysis of huge datasets, by conducting comparative effectiveness and cost-effectiveness research, and by providing ongoing decision support. Going forward an overall focus will be on how AI, and separately RWE, can each support formulary decision making.

Speaking at a conference session, Douglas Burgoyne, Chief Strategy Officer at Cooperative Benefits Group and Adjunct Associate Professor at the University of Utah College of Pharmacy, said health plans and pharmacy benefit managers are already using AI to aggregate data, summarize information and enhance their formulary decision making processes. Addressing two conference sessions on AI (“AI in Healthcare: From Cutting Edge Innovations to Practical Applications in Formulary Decision Making”; and “Executive to Executive: Leading Managed Care Organizations Today”), Burgoyne outlined potential use cases for incorporating AI in formulary decision making and utilization management activities at US health plans.

A key application to support formulary decision making is AI’s ability to aggregate vast datasets and information to assess comparative evidence of safety, efficacy, and cost-effectiveness. This has interesting implications for core value tools, including AMCP dossiers, which have served as key summary evidence tools for many years. In terms of data sources, AI can draw data from multiple sources including clinical trials, RWE, electronic health records, insurance claims, patient registries, scientific literature and regulatory databases. AI can also leverage big data processing wherein advanced machine learning algorithms can quickly process large amounts of structured and unstructured data efficiently, to identify relevant information from multiple sources.

Other areas where AI may be a game changer now and in the near future include

  • Cost management – The conference heard how AI can help to identify opportunities for generic substitution of brand-name drugs. Decision support systems driven by AI can provide formulary committees with evidence-based recommendations by searching for data on safety, efficacy, and cost-effectiveness. Indeed, such systems can already provide real-time recommendations to healthcare providers at the point of care by listing suitable generic alternatives when practitioners are prescribing medications. With its talent for data analysis, AI can analyze large datasets from electronic healthcare records, prescription databases, and pharmaceutical sales to identify patterns and opportunities to substitute generics for brands. Moreover, based on patent expiration dates, FDA approvals, and market trends, machine learning algorithms can look to the future and assess which brand-name drugs are most likely to have generic equivalents
  • In terms of prior authorization automation – AI can quickly process or pre-approve these authorizations, and this can reduce administrative burdens
  • Population health insights – AI can identify trends in medication use and adherence, in doing so it can support tailored interventions for important subpopulations. In terms of personalized medicine, AI can help to tailor formulary decisions by identifying which treatments are most effective for particular subgroups of patients
  • As noted earlier, AI can be used to predict trends. It can evaluate trends in claims data to make more informed decisions regarding formulary management, clinical program development, fraud protection, etc. Indeed, such AI initiatives have become a priority for health care payers. Many appear to be implementing AI applications to improve efficiencies, reduce costs, and address human error or bias, although this is not unique to this topic
  • Health care payers have also acknowledged they need appropriate processes and standards for AI utilization within healthcare decision making. This should ensure transparency, accuracy and validity of the data they use

Reaching for standards in RWE

It is clear that AI has a major role to play now and in the future of health and formulary decision making processes. According to Burgoyne, AI “is not going to stop, it’s only going to get bigger and more prevalent”. As it does so “We have to put guard rails, policies and procedures in place today to make sure that what we do tomorrow is the best we can do.” At Nexus, the AMCP demonstrated how it has already taken this onboard as it showcased its RWE Initiative, a two year program of work. The AMCP is creating a RWE framework for incorporating use of data into its unapproved and approved dossiers. Another aim is to develop an education curriculum “to elevate payer skills and confidence in utilizing RWE in Access Decisions”. The educational program will aim to equip payers with the knowledge and skills needed to use RWE standards in evaluating formulary access. The work is a collaboration with health plans, integrated delivery networks, pharmacy benefit managers and RWE experts.

Leveraging diversity through RWE

As part of health care research, RWE is becoming an important source of evidence reflecting how patients are treated in clinical practice. RWE is defined by the Food and Drug Administration as “the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD,” while RWD is defined as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources,” such as electronic health records, medical claims, disease registries, or other sources such as mobile or other digital technologies.[1]

The AMCP Research Institute[2] created a RWE Initiative to develop guidance for aligning RWE produced by pharmaceutical manufacturers with the needs of key stakeholders, such as payers and formulary decision makers, in health care research. This initiative aimed to bridge the gap between the interest of payers in using RWE for decision making and the generation of appropriate evidence by pharmaceutical manufacturers.

In the first phase of this process, the Institute implemented a real-world leaders forum to understand the type of RWE used in decision making. This phase was conducted in two parts to (1) Confirm payer perspectives of RWE in a survey; and (2) To engage a focus group of payers, formulary experts, and representatives from pharmaceutical manufacturers. The second phase consisted of another focus group and a more detailed survey to facilitate discussions around barriers and drivers to support the adoption of RWE in decision making. From these engagements an initial framework emerged as a first set of standards that will be followed by future goals to support the use of RWE in managed health care.

Why payers still hesitate to use RWE

Interest in RWE has been increasing but payers still hesitate about using it.

Reasons why:

  • The main barrier is the absence of common guidelines
  • Challenges around defining outcomes consistently and the methods for data collection
  • Decision makers lack of technical expertise in data analysis
  • Decision makers lack understanding of how and when to use RWE
  • Decision makers lack trust in pharma-sponsored RWE studies

How to fix the issue:

Standardized guidelines and transparency in methods and reporting of RWE is needed to overcome these barriers. This could be achieved by:

  • Improving trust and confidence in RWE with education that will improve decision makers’ understanding of study design and reasoning
  • Developing guidelines for RWE evaluation and interpretation (i.e. create a clear and consistent framework)
  • Providing standards for interpreting RWE within AMCP dossiers (i.e. what is “good” or quality data?)

Relevancy and consistency of data is important. For example, data from randomized controlled trials (RCTs) are comparable and clear given the defined endpoints and study population. RWE is not consistent across studies, so payers are unable to understand value alignment across products.

The timeliness of data is also important.

  • For example, RWE at product launch is not feasible, but years down the line when it is available, there could be additional competitors
  • How do we use the AMCP dossier to manage payer expectations of what should be available and when? Perhaps through potential changes or additions in the dossier and through education of what can be available and when

What Nexus panelists said:

  • Panelists at AMCP Nexus 24 said they are seeing a greater use of natural history of disease studies in RWE
    • For example, actual standard of care for a disease may not be aligned with treatment guidelines
    • RWE can show actual standard of care and outcomes being used, which will support the context of RCTs

Conclusion

Some of the most interesting discussions at AMCP Nexus 24 considered how AI could help to transform formulary decision making and similarly the benefits of truly understanding RWE. The challenges accompanying AI’s use continue to be data privacy, the need for high-quality data inputs, and the need to ensure healthcare professionals can interpret and act on AI’s outputs. The AMCP’s RWE Initiative could provide the solutions and standards that payers and formulary decision makers are seeking. Moreover, the conference session on Medicaid’s approach to treating sickle cell disease with emerging cell and gene therapies could prove to be transformative for patients and cost-saving for US State budgets.

References

  1. https://www.fda.gov/media/120060/download
  2. Represented by Carolyn Ha from PhRMA, Cate Lockhart from AMCP, Matt Mitchell from Select Health/Scripius, and Elizabeth Powers from IQVIA at AMCP Nexus 2024 in Las Vegas, Nevada.