2025 Drugs to Watch: A Helpful Guide for Payers

Stay Ahead of 2025 Drug Trends

Managing drug trend and spend continues to be a significant challenge for payers, with specialty drug trends rising in double digits according to our latest State of Specialty Spend & Trend Report. As we continue into 2025, understanding the drug pipeline and its potential impact on the market remains crucial for effective drug spend management. Before we dive into our predictions for 2025, let’s examine how our 2024 predictions performed and review our process for identifying these important emerging therapies.

Pipeline Developments Shape Benefit Strategies

The specialty drug pipeline remains robust, with ongoing scientific advancements and investments leading to groundbreaking treatments for previously untreated diseases. We’ve observed the consistent growth in novel drug approvals over the past decade, with specialty drugs consistently representing more than half of all approvals. In 2024, 37 (74%) of novel drugs approved by the FDA’s Center for Drug Evaluation and Research (CDER) were specialty drugs.  These innovations include new therapies in competitive markets offering clinical advantages such as improved efficacy and reduced side effects. In addition, the FDA continues to review existing drugs being developed in new dosage forms and expanded indications.

PSG’s Drugs to Watch List

Our proprietary Drugs to Watch List, developed at the beginning of each year, serves as a summary of new drugs we expect have the potential for significant impact to pharmacy budgets if approved in the upcoming year. We focus on non-oncology scientific advancements, which may possess a variety of the following attributes:  new drug for a previously untreated disease, new high-cost drug, new drug in a competitive therapeutic market that offers some new clinical advantage and notable cell and gene therapies.

2024 Drugs to Watch Performance

Our 2024 Drugs to Watch contained 12 drugs, all of which received FDA approval before the end of the year and have already demonstrated an impact on payer decisions. Notable approvals included:

The impact of these approvals has been significant and can present challenges for PBMs and payers in terms of coverage decisions. For example, Rezdiffra, the first and only FDA-approved drug for NASH, entered the market at an annual estimated cost of over $47,000. It is the first drug option for patients with NASH and is estimated to affect 9-15M people in the United States. For one plan sponsor, we identified 200 members with a diagnosis of NASH.  If half of those members seek treatment with Rezdiffra, annual budget impact would be approximately $4.7M, a drug spend they previously did not incur for these members. Similarly, Kisunla, the second FDA-approved drug with full approval for treatment of patients with early-onset Alzheimer’s, has an estimated annual cost of $30,000. There is a common misconception that this drug is only for Medicare-aged patients. However, utilization could very well occur in a commercial population, affecting the budget significantly. PBM coverage strategies vary widely for Rezdiffra and Kisunla.

2025 Drugs to Watch

We have selected 12 drugs for this list in 2025. Among these drugs is a combination of new dosage forms for treatment of rare conditions, a potential second drug for NASH, a unique drug therapy for hemophilia patients, and a potential new oral therapy in an area that hasn’t seen a new one in more than 20 years for treatment of acute pain. Two of these have already received FDA approval.

For our full list please reach out to PSG.

Alyftrek (Vertex)

Alyftrek, Vertex’s fifth and newest drug for treating the underlying causes of cystic fibrosis (CF), was approved by the FDA early, on 12/20/2024. Alyftrek is expected to expand treatment to an estimated 150 CF patients in the U.S. with one of the additional 31 mutations making them eligible for a CFTR modulator for the first time. Additionally, it offers once-daily dosing, which is an advantage over Trikafta, which has to be taken twice daily with a fat-containing meal.

Suzetrigine (Vertex)

On January 30, 2025, the FDA approved Journavx, a first-in-class non-opioid analgesic for treating moderate to severe acute pain in adults, specifically pain related to surgery or injury. This approval marks the first significant innovative pain medication in over 20 years. Journavx works by targeting a different pathway to reduce pain signals before they reach the brain. As a result, it offers the advantage of being non-addictive, making it a promising alternative to opioid pain relief. Vertex estimates that more than 80 million people in the U.S. are annually prescribed drugs for moderate to severe acute pain.

Sebetralstat (Kalvista)

This is a first-in-class oral drug therapy for on-demand treatment of acute hereditary angioedema (HAE) attacks. If approved, it will become the first oral treatment for HAE acute attacks. As a plasma kallikrein inhibitor, it works similarly to Kalbitor (an injectable acute HAE attack treatment) and oral Orladeyo and injectable Takhzyro (used for HAE attack prevention). HAE is a rare hereditary disorder characterized by painful, unpredictable swelling in different body parts that can potentially be life-threatening. An estimated 6,000 people in the U.S. live with HAE.

Atrasentan (Novartis)

This oral pipeline drug is being studied to reduce proteinuria (elevated protein levels in urine) in patients with IgA nephropathy (IgAN), a progressive autoimmune kidney disease. IgAN primarily affects young adults, causing an accumulation of IgA antibody protein in the kidneys, leading to inflammation and progressive damage that can result in kidney function loss. In some IgAN patients, persistent proteinuria may progress to kidney failure within 10 years. Drugs like atrasentan may help slow this progression. An estimated 110,000 people in the U.S. are living with IgAN.

Azemiglitazone (Cirius Therapeutics)

This pipeline drug is a first-in-class, oral once-daily insulin sensitizer being studied for type 2 diabetes and MASH/NASH. Approximately 6-8 million people in the U.S. are living with MASH, with 1 in 5 patients progressing to cirrhosis. Aimed at improving liver fibrosis and preventing or reversing cirrhosis, Azemiglitazone is a drug to watch as a potential additional treatment option, possibly joining Rezdiffra in treating MASH patients.

Fitusiran (Sanofi/Alynlam)

A novel RNA interference therapy, Fitusiran is a non-factor treatment with potential to treat patients with hemophilia A or B, with or without inhibitors. In clinical studies, the drug demonstrated the ability to reduce annualized bleeding rates by up to 90%. Fitusiran offers the convenience of a once-monthly subcutaneous injection, which could be advantageous compared to existing hemophilia treatment dosing frequencies. In a therapeutic class increasingly exploring gene therapies with similar goals, this drug presents an interesting development to monitor.

Looking Ahead for 2025 Pharmacy Benefits Strategy

As we continue supporting our payer clients in making informed coverage decisions, our Drugs to Watch List remains a valuable tool for anticipating market changes and preparing for new therapy introductions.

The Drugs to Watch List is presented to clients in January and updated monthly with the progress of FDA review and approvals. As drugs are approved, we present relevant information and use our proprietary software tool, Artemetrx, to share the number of members who have diagnoses that correspond to the indications for the new drugs to better assess potential impact specific to each client. Often, this new drug approval information leads to clinical strategy and/or drug class reviews which ensure optimal drug coverage and utilization management.

Understanding potential financial impacts helps inform strategic decisions and optimal investment in pharmacy benefits. We invite you to connect with our experts for a detailed analysis of these drugs and their potential impact on your pharmacy benefits strategy.