Emerging trends in therapeutics and diagnostics: Perspectives on the 2024 AMCP Foundation Survey

The 2024 AMCP Foundation Emerging Trends Survey identified trends expected to impact managed care pharmacy over the next 5 years across 5 domains. This article provides expert commentary on the findings in the therapeutics and diagnostics domain. Specifically, the survey results on the likelihood of potential scenarios occurring in the next 5 years within the therapeutics and diagnostics domain are shown in Table 1. The methods and full results of the Emerging Trends Survey are reported elsewhere.¹

TABLE 1.

Responses to the Question “How Unlikely or Likely Is It That the Following Will Occur Within the Next 5 Years?”

Scenario: therapeutics and diagnostics domain	Highly unlikely	Somewhat unlikely	Somewhat likely	Highly likely	Somewhat or highly likely
The use of GLP-1 receptor agonists will increase by at least 25% (in part because of additional approved indications).	0.0	3.4	39.4	57.1	96.6
More than 10 new orphan drugs will be approved for rare conditions.	0.6	4.6	33.7	61.1	94.9
The number of original biologic products for which biosimilars are approved for marketing will increase by at least 25%.	1.7	10.9	52.0	35.4	87.4
More than 5 new vaccines will be developed for emerging infectious diseases and/or chronic conditions.	1.7	16.6	54.9	26.9	81.7
The FDA will approve more than 10 new digital therapies.	2.9	17.7	57.7	21.7	79.4
The number of over-the-counter diagnostic tests will double.	1.7	25.1	56.0	17.1	73.1
Expanded pharmacogenomic research data will result in a 25% increase in the use of this type of information in clinical decision-making.	6.9	28.6	50.3	14.3	64.6
Screenings for early detection of cancers will increase by at least 50%.	2.9	34.9	47.4	14.9	62.3
Social determinants of health will be formally incorporated into more than 20% of clinical trials.	8.0	33.7	49.1	9.1	58.3
Gender and racial diversity will be formally assessed and evaluated in greater than 50% of clinical trials.	5.7	36.6	45.7	12.0	57.7
The FDA Accelerated Approval process will expand by 50%.	5.1	48.0	40.6	6.3	46.9
At least 25% of new drug approvals will be based in part on patient-experience data.	6.3	49.1	37.7	6.9	44.6
At least 10% of patients treated for a chronic condition will use an implanted drug delivery system.	17.1	49.1	31.4	2.3

Data are presented as percentages. N = 201 responses.

FDA = US Food and Drug Administration; GLP-1 = glucagon-like peptide-1.

Lockhart’s Perspectives

Therapeutics and diagnostics represent the foundation of managed care pharmacy in the fundamental goal of supporting population health through appropriate use of medications and other treatments.² One important aspect is maintaining and improving the delivery of health care under limited resources, particularly in light of rapidly advancing technology and increased sophistication of new and emerging treatment options. Targeted therapies and other engineered biologic products are topping the pipeline of new products demonstrating a notable advancement in patient care and precision medicine, but often carrying a high price tag.³ Therefore, it is essential that managed care pharmacy professionals representing health plans, pharmacy benefit managers (PBMs), and employers are able to anticipate and prepare for arrival of these important new products to ensure continuity and quality of patient care.

It is not surprising that the topic of glucagon-like peptide-1 (GLP-1) receptor agonists is at the top of the Emerging Trends list. These products are very effective in diabetes and weight management, but they are expensive, and with current and anticipated approved indications, the population eligible for treatment with these products is potentially very large. This is reminiscent of the early 2010s when hepatitis C treatment was transformed by direct-acting antiviral products that offered a cure for what was previously a chronic disease with a high health care burden.⁴ There was no question these products were cost-effective, but there were grave concerns about the affordability given the large population size of patients with hepatitis C and the high cost of approximately $82,000 for a treatment course.⁵ Similarly, GLP-1 products are clearly effective, but overall affordability is a concern in managed care pharmacy. This may be an opportunity for creative solutions such as value-based contracts or other ways to optimize treatment such that the patients who receive the product are most likely to benefit. There is a need for more assessment of long-term implications such as patient adherence and the impact on total cost of care for patients who may have avoided costly complications by reducing body mass.

Rare diseases are also poised to benefit from advancing technology and targeted therapeutics, and survey respondents agreed that it is likely or highly likely that more than 10 new orphan drugs will be approved. The contrast with GLP-1 agonists is the small population size of patients affected by rare conditions. However, orphan drugs are also expected to be very expensive, inviting creativity in managed care pharmacy to afford these life-changing treatments for patients who otherwise have few or no other options.³

The majority of survey respondents indicated it is likely or highly likely that the number of available biosimilars will continue to increase. Given a biosimilar pipeline of more than 80 products in development, this is very likely.⁶ In spite of some success and demonstrated cost savings introduced by biosimilars, the lingering challenge with biosimilars is sluggish market penetration. When biosimilars first emerged, there was some trepidation about whether they truly had no clinically meaningful difference from the reference product.⁷ At the Biologics and Biosimilars Collective Intelligence Consortium, we regularly hear that with growing experience, patients and providers are becoming more comfortable with the approval process and that they are indeed similar and do not disrupt patient care. Now the concern is more about reimbursement and whether the savings incentives are appropriately aligned, and the sustainability of the biosimilar market. Managed care pharmacy is uniquely positioned to support increased biosimilar availability and utilization, as a robust biosimilar marketplace is a benefit to our health care system as a whole.

For biosimilars, the statutory concept of interchangeability in the United States also adds confusion and complexity that is not faced elsewhere in the world. In early 2023, the European Medicines Agency issued a statement that all approved biosimilars are assumed to be interchangeable with the reference product, and with all other biosimilars to the same reference product.⁸ There is legislation before Congress to modify the statutory language to align more with the European Medicines Agency, but it remains to be seen if or when such language may be adopted.

One scenario that 64.6% of survey respondents indicated was likely or highly likely to have an impact on managed care was the advancement of pharmacogenomic testing (and biomarker testing could potentially be included in this category as well) to inform clinical care. Although this was not one of the top-ranked topics, it seems that there is a groundswell around precision medicine and “rational therapeutics” to help determine the best treatment options for each patient. Genetic and biomarker testing is available and recommended to guide treatment for several diseases such as depression and other mood or psychiatric disorders and to inform treatment and dosing in patients with epilepsy.^9-11 Testing is also recommended in several cancer types including advanced non–small cell lung cancer (aNSCLC) to inform targeted chemo- or immunotherapy treatment decisions.^12,13 However, not all patients are receiving those tests and not all are treated accordingly. One study in the United States found that only 59% of patients with aNSCLC received any kind of genetic testing, and among those who received next-generation sequencing tests, 10% did not receive an appropriate targeted therapy, and 40% of patients who received other non–next-generation sequencing tests did not receive an available targeted therapy.¹⁴ Another study found that 73% of patients with aNSCLC who had at least 1 actionable biomarker or genetic mutation received a targeted therapy.¹⁵ Of patients with aNSCLC with at least 1 actionable oncogene mutation and/or programmed death-ligand 1 positivity, only 29% received cancer immunotherapy (CIT) with chemotherapy, 27% received CIT only, and 21% received targeted therapy with chemotherapy (but no CIT).¹⁵ Managed care pharmacy is perfectly positioned to encourage testing and related clinical decisions according to existing national clinical guidelines.

Managed care pharmacy faces remarkable advances in therapeutics and diagnostics, and there are opportunities to play a major role in influencing appropriate medication use. Although the list described in the survey is not exhaustive of all potential developments in therapy, it shows a thoughtful assessment of what we should pay attention to for near-term strategic planning. Building on this insight to anticipate the changing health care landscape will set us up for continued success in ensuring patients get the medications they need at a cost they can afford.

Manolakis’s Perspectives

The therapeutics and diagnostics domain scenarios include key trends plan sponsors face daily. They not only reflect the ongoing, high-priority concerns but also tie directly to the top 3 pharmacy benefit concerns identified through the 2024 Business Group on Health 2025 Employer Health Care Strategy Survey.¹⁶ These include the appropriate use and the long-term cost of GLP-1 agonists, patient and plan affordability of higher-cost drugs, and overall pharmacy costs. Drawing on a 2019 Kaiser Family Foundation analysis of 2017 data from the National Health Expenditures Accounts, private health insurance spent more on total prescriptions ($140B) than Medicare Part D ($101B), out-of-pocket spending ($47B), or Medicaid ($33B).¹⁷ Arguably, and based on spend alone, private health insurance, that portion of spend administered and managed by PBMs and health plans, should receive significant attention from managed care pharmacy. Payers of private health insurance are telling us what they are concerned about, and we should listen as they need our support to help manage tremendous plan cost and drive quality into the pharmacy benefit. Let’s unpack the top 3 scenarios in therapeutics and diagnostics.

Survey respondents were bullish on the anticipated success of current and anticipated research and development efforts related to traditional and specialty drugs. Of the top 3 items from the Emerging Trends Survey, 2 tie to a productive and expanding development pipeline and 1 to increased use of the most impactful drug class we’ve experienced in many years, the GLP-1 agonists. More than 87% of respondents across these 3 scenarios predict occurrence of these scenarios, suggesting they are strong candidates to become trends.

Scenarios 2 and 3 focus our attention on more than 10 new orphan drugs approved for rare conditions and new biosimilars increasing by at least 25%. The approval of 10 new orphan drugs for rare conditions was viewed as likely or highly likely by 94.9% of respondents. Interestingly, this scenario received the highest percentage of votes for highly likely at 61.1%. This scenario is grounded in a long and rich history dating back to 1983 and the passage of the Orphan Drug Act. The science has developed substantially over the years with hundreds of orphan drugs receiving US Food and Drug Administration (FDA) marketing approval, so it comes with little surprise that it received such strong support. What is surprising is that it didn’t hit the top of the list, which was earned by the GLP-1 agonist class of drugs.

Respondents anticipate biosimilar increases as likely or highly likely at 87.4% and new orphan drugs at 94.9%. We know the biosimilar pipeline is full, with market projections of $92B by 2031, reflecting a compound annual growth rate of 16.4% from 2023 to 2031.¹⁸ Much like the patent cliff of 2010 to 2015 that brought payers and patients savings through generic drugs, we will likely look back at this decade as the start of the biosimilar boom. These products have been generating savings within the medical benefit for several years and are now reaching the outpatient pharmacy benefit through Humira’s biosimilars and soon to be Stelara’s biosimilar. Managed care strategies must focus on rapid exclusion of the reference products following launch, even if this means sacrificing rebates in the short term. Chasing rebates is not a long-term play, and biosimilar market launches are not only a scenario with high likelihood but also a trend and our future. Managed care pharmacy must embrace this mentality and drive change to support our employer payers with formulary exclusions and rapid adoption strategies.

I am certain we will look back at the rise of the GLP-1 agonists as a generational change in treating diabetes, obesity/overweight, and other indications that are just becoming understood. The first expanded indication associated with weight reduction and GLP-1 agonists is cardiovascular risk reduction. The cost challenges from the new drugs in this class are well documented, which we can’t ignore, but the therapeutic gains are clinically impressive. Consider the fact that there has never been a drug product like this for treating obesity, and consider the changes to the type 2 diabetes guidelines. If we let our minds wander to the other disease states being studied for treatment with these drugs (ie, chronic kidney disease, Alzheimer disease, addiction, and dementia), the possibilities become exciting and scary at the same time. This is what employers are thinking: specifically, how should our plans cover them and how can the plan afford them? And on equal footing, how can the plan be sure that the allocation of these products within a population is fair and equitable?

The strategic implications for managed care pharmacy are significant for the GLP-1 agonist weight loss drugs. Payers need guidance and support from their PBMs, health plans, and pharmacy consultants on plan design and strategies that promote positive and sustainable health outcomes. Traditional PBM rebate contracting approaches have created financial disincentives for plans to implement appropriate utilization management strategies or the use of third-party, adjunct programming that promote the achievement of these outcomes. Managed care pharmacy has an imperative to create change so that payers are not constrained by disincentives when their weight loss drug utilization approach is in alignment with the FDA label. GLP-1 agonists have and will continue to reshape disease treatment, and they have the potential to reshape how diseases with significant prevalence are treated by managed care pharmacy. Where I started the GLP-1 agonist commentary…it’s generational change. We need to embrace it with payers in mind.

There is much more packed into the findings from the therapeutics and diagnostics domain scenarios, but I will leave that for you to debate and discuss with your colleagues. My ask is that you consider the employer payer in your discussion as you figure out how managed care pharmacy can strategically act to solve the challenges faced because of a changing therapeutics and diagnostics landscape.

Funding Statement

No funding was received for the development of this manuscript. The publication of the supplement was funded by Pfizer.