For pharma manufacturers, securing expedited approval for a drug in development is typically seen as a win, which will result in faster patient access as well as faster revenue recognition. However, the realities of market access mean that drugs approved by expedited pathways—most notably, the accelerated approval pathway—may face a much more difficult path to coverage and provider uptake.
The use of FDA expedited approval pathways has grown significantly in the past two decades, and expedited approval of one sort or another is now the rule rather than the exception. Last year, 66% of all novel drugs approved by the FDA followed at least one expedited pathway.
Despite the new normal, however, many payers are still reluctant to cover many fast-tracked therapies due to insufficient safety and efficacy data. Payers are also rightfully concerned about the potential for conditionally approved drugs to have their approval revoked. Over the last decade, 23% of accelerated approvals in oncology were withdrawn due to lack of sufficient clinical benefit.
To better understand payers’ point of view on expedited approvals, the MMIT Index team surveyed a representative sample of payers via a monthly event primer. Before we dive into the results, let’s review the various approaches to expedited approval:
- Fast track: Manufacturers must apply for this designation, which is intended to speed the development and approval of drugs for unmet treatment areas.
- Breakthrough therapy: If preliminary clinical evidence indicates that a drug for a serious condition may demonstrate substantial improvement over available therapies (based on a clinically significant endpoint), its manufacturer can apply for this designation by the end of Phase II.
- Accelerated approval: This process allows drugs for serious conditions to be approved conditionally, based on a surrogate endpoint rather than multiple endpoints. Manufacturers must then conduct phase IV confirmatory trials to show that the drug provides a clinical benefit before earning final approval.
- Priority review: Priority review shortens the review timeline by about 5 months, and is meant for drugs that will improve the treatment, diagnosis, or prevention of severe conditions. This designation doesn’t alter the medical standard for approval.
Payers Want Additional Safety and Efficacy Data
Of the four expedited approval pathways, our research indicates that payers are most concerned with the accelerated approval pathway, and the least concerned about priority review.
In the Index survey, most payers (60%) said they are very or extremely concerned about drugs approved via accelerated approval, due to the insufficient review time and inadequate or missing data. As one pharmacy director noted, there is a “lack of data to review” as these products are often “approved with minimum data requirements or surrogate endpoints.”
Most payers are at least moderately concerned over the reduction in required clinical trials for many expedited drugs. Many survey respondents expressed concerns about the scarcity and validity of available data. Without long-term study data, payers cannot be sure that the drug doesn’t also cause unexpected comorbidities or serious side effects.
As one pharmacy director noted, “Expedited approval pathways can sometimes prioritize speed over thorough evaluation, potentially leading to safety concerns for patients if long-term effects aren’t fully understood. These pathways may also increase the risk of bypassing critical clinical trials, which could result in less effective treatments reaching the market. While they are designed to address urgent needs, there is a delicate balance between urgency and ensuring that the treatment’s benefits outweigh any potential risks.”
Typical Utilization Restrictions for Expedited Drugs
In the absence of long-term data, many payers will choose to cover an expedited drug sparingly. Almost half (45%) of surveyed payers report placing specific restrictions on expedited drugs. For most of these payers, the restrictions apply to all lines of business, although two payers exempt their Medicare and Medicaid plans from restrictions.
The most common restrictions imposed on fast-tracked therapies are stringent prior authorization requirements, followed by step therapy requirements. Two-thirds of these payers say they only provide coverage of expedited drugs when there is a confirmed clinical benefit, proven by positive real-world evidence or full FDA approval.
More than half of payers impose a period of exclusion from formulary for a defined period (typically 6-18 months) and/or impose indication-based restrictions, prescriber restrictions, or higher tier placement. Payers that impose new-to-market blocks on expedited drugs are predominantly concerned over the lack of sufficient clinical evidence and real-world evidence (RWE).
When asked what data would mitigate the placement of a new-to-market block, one pharmacy director of a Blues affiliate said, “Robust clinical trial data demonstrating the safety and efficacy of the product would be essential, alongside real-world evidence of its positive impact on patient outcomes. Additionally, long-term follow-up data to assess sustained benefits and any potential risks over time would strengthen the case for approval or acceptance.”
Payers Reject Long New-to-Market Blocks
In February 2025, the Philadelphia-based Independence Blue Cross (IBX) made headlines when it announced a new policy to exclude drugs approved under the accelerated approval pathway from coverage for a period of 18 months. According to MMIT’s survey data, only 10% of payers were very or extremely likely to follow in IBX’s footsteps by creating a similar blanket policy for expedited drugs. As one chief medical officer noted, such a policy “prevents patients from accessing potentially life-saving drugs. Drugs still should be reviewed on the strength of the clinical literature.”
Payers agreed that policies like IBX’s will lead to restricted access, which could in turn create additional problems. As one pharmacy director noted, “There could be significant member and provider abrasion when a FDA approved therapy is not available.” Others noted that such policies would increase the rate of appeals, since “providers and patients may still appeal and request access to the treatments, since the drugs may meet unmet need.”
Of the payers considering implementing a longer new-to-market block policy, more than half (55%) said that clinical and real-world data on safety and efficacy might persuade them from excluding expedited drugs from coverage. One pharmacy director requested “efficacy data, more data to review than what is in prescribing guidelines.” A chief medical officer of a Blues affiliate said his organization was looking for “real world economic/efficacy data. Ideally, quality clinical trials that are not manufacturer sponsored.”
Developing an Effective Launch Strategy
In light of this information, manufacturers must be aware of the ramifications of expedited approval on their launch trajectory. While some physicians are accustomed to requesting medical exceptions for their patients, many others will view this process as too much of an administrative burden.
To set realistic expectations for launch, manufacturers of drugs approved via an expedited process should conduct an analog analysis of other fast-tracked therapies. By choosing relevant, analogous market access scenarios, manufacturers can accurately anticipate how payers are likely to respond to their drug at launch. This valuable information can help manufacturers target the right payers and providers, develop a strong physician education program, and adjust their market access strategy to accommodate likely restrictions.
Manufacturers should also keep in mind the breadth and depth of the data payers want. As one clinical pharmacist said, payers are interested in “complete transparency on inclusion/exclusion criteria,” as well as “transparency on how subjective endpoints were measured, e.g., as endpoints related to patient-reported outcomes.” Others noted their organization’s desire for full disclosure about failed trials, cost offsets, off-label use, and other indications being tested.
Manufacturers might be able to persuade payers not to require medical exceptions if they provide not only the pivotal trial results that led to the drug’s approval, but also proof that those results can be considered clinically meaningful. Providing a transparent recap of clinical trial data will be met with gratitude from payers who are looking to reduce risk. Manufacturers of drugs approved under the accelerated approval pathway must also educate payers on the validity of surrogate endpoints.
The benefits gained from fast tracking novel drugs are numerous, as long as everyone is aware of the potential risks and is committed to working together to mitigate them. Manufacturers can begin to see a profit on their new drug, which will allow them to invest in additional research. Doctors with qualifying patients can save more lives. And most importantly, patients waiting for drugs to be approved so they can begin treatment will be able to access life-saving treatments faster.
To see how MMIT can help you predict uptake of your fast-tracked therapy, learn more about our Strategic Launch Report & Evaluate Forecast.
