As 2025 draws to a close, it is becoming clear that the US and EU’s approval rates for innovative medicines will be markedly lower than they were in 2024. CHMP approval recommendations in the European Union are running at 44 as of late November 2025, contrasting sharply with the European Medicines Agency’s revitalised performance in 2024, which saw the CHMP (Committee for Medicinal Products for Human Use) recommend 64 new drugs for approval, compared with 50 in 2023.
The US Food and Drug Administration (FDA), meanwhile, continues its decline from the exceptional 80 approvals recorded in 2023, with a total of 47 marketing authorisations in the year to late November. This inevitably raises questions over whether staff layoffs and other politically driven upheavals at the agency are having an impact on approval rates – not to mention a potential impact on other agencies such as the EMA, whose practices are aligned to some extent with those of the FDA.
Let’s take a more comprehensive look, then, at what the 2025 new drug approval scores and the more exciting innovative medicines debuting in these two key markets tell us about the pharmaceutical industry’s present state of health, and what we might expect from the industry in years to come. Particularly in the US, the declining approvals count for 2025 is overshadowed by the Trump administration’s restructuring, streamlining and consolidation of federal agencies and workforces.
These transformation and optimisation initiatives have included significant layoffs across the FDA (but excluding drug reviewers specifically) and the Department of Health and Human Services (HHS), as well as the voluntary departures of a number of key FDA personnel involved in new drug approvals. It remains unclear precisely how much these developments may have affected new drug approvals during 2025.
Looking further ahead, the FDA’s reform agenda under the umbrella of HHS Secretary Robert F. Kennedy Jr. includes efforts to reduce drug approval times significantly. The reforms are also geared to maximising the potential of cell and gene therapies, accelerating biosimilar development, simplifying regulatory requirements for clinical trials, and tackling perceived conflicts of interest, particularly related to industry involvement in FDA advisory committee meetings.
This determined overhaul of the FDA status quo has important implications for the agency’s approval performance. It will undoubtedly cause some concerns about the ability of a leaner FDA, with a potentially lighter regulatory touch, to maintain the crucial balance between rigorous oversight and flexible, creative solutions, and to reassure both the public and politicians that drug innovation is being embraced and rewarded, without compromising standards of efficacy, safety, quality and scientific independence.
The US federal government shut down throughout October and the first half of November 2025, after Congress failed to reach a budget agreement for the 2026 fiscal year. The ensuing lapse in FDA appropriations meant the agency could not accept any new drug submissions during this fallow period, while the rejection rate for ongoing submissions had already risen from historical rates in the third quarter of 2025. FDA Commissioner Marty Makar informed colleagues on 1 October that certain agency operations would have to shut down until the funding crisis was resolved, commenting: “While many employees will be furloughed during the lapse period, the FDA will continue to fully execute our public health mission to the extent permitted by law.”
Meanwhile, the EMA has not suffered any comparable disruption to its workforce or operations. All the same, the 44 positive CHMP opinions this year so far look low when set against the committee’s 64 approval recommendations for the whole of 2024. At the time of writing, there was still one more CHMP meeting scheduled before the end of 2025, which could boost the approval count. On the other hand, as last year’s approvals review noted, by late November 2024, approval recommendations by the EMA’s Committee for Medicinal Products for Human Use (CHMP) had already outstripped positive opinions for 2023.
The European agency has, nonetheless, committed to enhancing the efficiency of its assessment and approval processes for new medicines. Among the proposed measures are:
The EMA highlighted the unreliability of long-term planning for initial marketing authorisation applications (MAAs) as a recurrent problem for the regulatory network, which had slowed down drug approval times. In its mid-year report, the agency noted a positive trend in company clock stop extensions for MAAs during the first and second quarters of 2025. These extensions averaged 150 days, down by 18% from an average of 182 days in 2024. This improvement reflected the EMA’s efforts to reinforce best practices in company requests for clock stop extensions, thereby cutting overall delays in the approval process for drugs and vaccines. A 2024 EMA report found that:
In terms of the broader environment for new drug assessment and approval, a comprehensive overhaul of EU pharmaceutical legislation remains in play. The proposals put forward by the European Commission, and subsequently amended by the European Parliament, have prompted concerns about market access and the sustainability of the industry’s business model across the region.
The European Council adopted a consensus position on the proposed pharmaceutical regulation and directive in June 2025. Negotiations with the European Parliament to reach agreement on the legislative package will follow, and then adoption of the new rules once legal and linguistic revisions have been completed. Final adoption is expected between late 2026 and early 2028, with full implementation including an 18-month transition period over the next few years.
As of late November 2025, the FDA’s Center for Drug Evaluation and Research (CDER) has approved 38 new molecular entities and new therapeutic biologicals, compared with 50 approvals in 2024. Meanwhile, the FDA’s Center for Biologics Evaluation and Research (CBER) has cleared nine biological license applications (including source plasma) versus 19 in 2024. Current-year CBER approvals take the FDA’s year-to-date tally to 47 products, well behind the combined CDER/CBER count of 69 approvals for 2024 and lagging even further the FDA’s score of 80 approvals in 2023.
The CHMP has so far recommended 44 new medicines or vaccines for approval. This is substantially less than the committee’s 64 positive opinions in 2024, and significantly lower than the 55 new medicines or vaccines signed off by the CHMP by November 2024. With one more committee meeting still pending in 2025, though the EMA could feasibly end the year more in line with the 50 approval recommendations for 2023.
In most cases, the European Commission follows the CHMP’s approval recommendations, although there may be several months’ delay before the Commission signs off on them. At the time of writing, 33 of the 44 products recommended by the CHMP had secured European Commission approvals, according to publicly available information.
Both the US and the EU continue to face challenges that may influence the nature and timing of new drug approvals, both now and in the future. Foremost among these is the US clampdown on federal agencies and their workforces, including those of the HHS and the FDA. In March, it was announced that the drug agency would lose 3,500 employees, or 19% of its staff, although some of these employees were subsequently rehired and, as already noted, drug reviewers were spared. The total HHS workforce was cut from 82,000 to 62,000.
Moreover, it was reported that around half of the FDA’s senior leadership had left the agency under the new Trump administration, while 14% of agency staff (more than 2,000 employees) were not working during the US federal government shutdown. These issues could have a knock-on impact on other regulatory agencies around the world, including the EMA, which rely on FDA data and early approvals as well as other channels of communication and collaboration to facilitate their own review efforts.
One potential barrier to US approvals in recent years, now complicated by mixed signals from the restored Trump administration, has been tighter regulatory requirements for accelerated registration. This followed new provisions included in the Food and Drug Omnibus Reform Act of 2022, which gave the FDA authority to improve transparency around accelerated approvals and ensure that applicants complied with post-marketing requirements. A report published in January 2025 by the US Office of Inspector General identified significant flaws in accelerated approval processes for three drugs (Biogen’s Aduhelm/aducanumab, Sarepta’s Exondys/eteplirsen, and Covis Pharma Group’s Makena/hydroxyprogesterone caproate), raising questions over the effectiveness and consistency of the fast track pathway.
On the other hand, FDA Commissioner Makary recently announced a National Priority Voucher (CNPV) scheme, which is a pilot programme aimed at reducing drug review timelines from 10-12 months to one-to-two months following submission of a final approval application addressing US national priorities. These priorities include tackling a major health crisis or unmet public health need, boosting domestic drug manufacturing, or delivering more innovative medicines. The CNPV programme has been used as a bargaining chip in negotiations with US manufacturers on most favoured nation pricing for medicines in their home market.
Also of note has been the introduction of direct price negotiations for high-priced medicines covered by the US Medicare Part D programme, with effect from autumn 2023. This represented a significant disruption of the established US market model, based on free pricing at source and negotiations with insurers or pharmacy benefit managers. Since then, however, the pricing environment in the US market has become far more hostile, with potentially significant global implications for drug approval and launch strategies.
Notably, President Trump has been using the threat of 100% tariffs on pharmaceutical imports into the US to lever most favoured nation (MFN) pricing agreements from leading multinationals such as Pfizer, Eli Lilly and AstraZeneca. The MFN policy, designed to drive down “vastly inflated” drug prices in the US and eliminate “freeriding” by European and other developed nations on American innovations, effectively ties US prices in the Medicaid and Medicare programmes, as well as through other channels, to the lowest levels charged in other developed countries. The pricing formula disclosed by the White House in May 2025 involved matching US drug prices to the lowest price charged in an Organisation for Economic Co-operation and Development (OECD) country with a per capita GDP of at least 60% of US GDP per capita.
Demanding pricing concessions from 17 leading manufacturers in July 2025, President Trump outlined a strategy whereby pharmaceutical companies would have to provide MFN prices for every Medicaid patient, and stipulate that they would not offer other developed countries lower prices for new drugs than those charged in the US. In return, the administration would provide manufacturers with an avenue to “cut out middlemen and sell medicines directly to patients,” providing they did so at a price no higher than the best price available in developed countries. The administration would also use trade policy to support manufacturers in raising their drug prices internationally, if the increased revenues were re-invested directly into lowering prices for US patients and taxpayers.
The MFN strategy effectively encourages companies to compensate for price concessions in the US through higher list pricing in countries that have traditionally exercised more stringent price and reimbursement controls on medicines, including most EU markets. If companies face strong resistance to these pricing offsets outside the US, and decide that only selective launches are economically viable in these markets, the MFP programme could change the balance and complexion of drug approvals and market entries between some of world’s largest and most influential commercial territories. For EU-based pharma operations, this disruption could be further exacerbated by the impact on costs, profit margins, supply-chain strategies and launch programmes of the new and unprecedented 15% drug-import tariffs (excluding generics) agreed between the US and the European Union in July 2025.
The Trump administration’s MFN demands have resulted in banner pricing agreements with companies including Pfizer, AstraZeneca and Merck KGaA. These also incorporate elements such as relief from draconian import tariffs, access to the CNPV programme for accelerated drug approvals, and commitments to invest in US biopharmaceutical manufacturing and research efforts. Most recently, the MFN strategy saw both Novo Nordisk and Eli Lilly strike pricing agreements for Ozempic (semaglutide) and Zepbound (tirzepatide), respectively, aiming to reduce costs for cash buyers through the TrumpRx federal direct-to-consumer (DTC) sales channel, while expanding access to Ozempic and Zepbound through the Medicare and Medicaid schemes.
The radical overhaul of EU pharmaceutical legislation is edging towards a conclusion. The European Parliament generally diluted the provisions of most concern to industry in the proposed pharmaceutical regulation and directive. These included plans to reduce baseline regulatory data protection (RDP) for newly approved medicines from eight to six years (7.5 years in Parliament’s revised version). Parliament also removed a controversial requirement for companies to supply new drugs continuously in all EU Member States within two years of approval, if they wanted to claw back two years of lost RDP.
In the European Council’s compromise position, baseline RDP for newly approved medicines is restored to eight years, with one to two years (two to three years in Parliament’s version) of additional market exclusivity, subject to certain conditions (e.g. an extra year of RDP if the drug addresses an unmet medical need, or if clinical trials are conducted in more than one EU Member State). This effectively reduces the overall baseline protection for new medicines from 10 to nine years, with implications for product launch strategies. A Council amendment granting Member States the authority to insist that marketing authorisation holders supply approved medicines in sufficient quantities to cover the needs of patients in the relevant jurisdiction, via a new article in the pharmaceutical directive, will not be well received by industry.
The Commission’s revisions also included proposals to reduce the time taken to evaluate new medicines centrally in the EU. For example, the EMA would have to assess products within 180 days (150 days for medicines of major public health interest), down from 210 days currently. These adjustments remained unchanged following Parliament’s review of the proposed Directive, as did a provision introducing the concept of regulatory sandboxes, with adaptive frameworks (including real-world data collection) to expedite approvals of novel therapies. There are no indications of these approval-related provisions having been amended in the Council’s consensus position.
The list of new molecular entities approved in 2025 to date continues to honour breakthrough innovation. For example:
Many of the breakthrough medicines listed are indicated for rare diseases, building on the dominant trend in recent years for specialty drug approvals, particularly the sub-category of new medicines for rare diseases. Among the 38 new medicines approved by CDER in 2025 to date, 14 have been for orphan designated medicines (albeit a lower proportion than the 20 out of 40 in the same period of 2024). Of the nine BLAs cleared by CBER in the year to November, three (five of 15 in 2024) were orphan designated. In 2024, 26 or 52% of the 50 new medicines approved by CDER were for orphan-designated indications. Among the 17 BLAs cleared by CBER in 2024, six were orphan designated.
In the EU, the CHMP’s 44 approval recommendations to date in 2025 have included 21 orphan designated products, a higher proportion than the 17 out of 55 recommendations for orphan drugs in the year to November 2024. By the end of 2024, the CHMP’s 64 approval recommendations included 22 orphan drugs.
Some recent trends in drug development and approvals, such as high-profile new-wave therapies for Alzheimer’s disease and obesity, have suggested a tentative return to high-impact new medicines aimed at large patient populations. This shift could gain momentum from regulatory developments such as:
Rebates on above-inflation price increases in Medicare could shift the balance towards more volume-driven drug strategies. With MFP negotiations, the limited grace periods available before MFPs take effect might dampen enthusiasm for current pipeline-in-a-product strategies, geared to cumulative indication-stacking and progressively larger patient populations. On the other hand, MFPs, which have so far stood firm under the Trump administration and resisted legal challenges from manufacturers, could encourage companies to focus more intensely on biologics – and, by implication, specialty medicines – rather than small molecule drugs. This reflects the more favourable grace period (13 years) available to biologics, although companies have lobbied for a level playing field in this respect.
In the EU’s pharmaceutical review, the Commission wanted to replace the current standard 10-year market exclusivity for orphan drugs with a modulated exclusivity. This would mean nine years’ baseline exclusivity and only five years for drugs approved using bibliographic data. An additional year’s exclusivity would be available for drugs addressing a high unmet medical need (HUMN), launching an orphan product in all the Member States within two years of approval, or developing new therapeutic indications for an already authorised therapy.
The compromise position adopted by the European Council in June 2025 incorporated 10 years of orphan market exclusivity, the same as under current legislation, and an improvement on the nine years of baseline orphan market exclusivity proposed by both the Commission and Parliament. It also maintained the possibility for MA holders to extend twice the orphan market-exclusivity period by 12 months, based on approval of a new orphan indication.
At the same time, the Council retained the Commission’s concept of a global MA, so that marketing authorisation holders with more than one product based on the same active substance, but for different orphan indications, would no longer be eligible for separate orphan market exclusivities. The Council also agreed with Parliament that manufacturers of generics and biosimilars should be allowed to apply for, and even secure, approvals before an originator’s orphan market exclusivity period expired, if the initial market exclusivity had less than two years to run.
For all the challenges of regulatory changes in drug approvals, pharmaceutical launch success still ultimately boils down to revenues and profit margins. The Drugs To Watch report published annually by Clarivate includes 11 new medicines either in Phase II/III clinical development, pre-registration/registration or already launched in 2025. These assets were expected to generate annual sales of $1 billion or more within five years and/or to transform treatment paradigms.
Clarivate’s annual Drugs to Watch report for 2024 identified 13 potential blockbusters or game-changers for the next five years on the same basis. Some of the latest year’s crop are more user-friendly or combination versions of existing drugs (e.g. Novo Nordisk’s Awiqli and CagriSema), rather than innovative medicines in the strict sense of new molecular entities (NMEs). This is a salutary reminder that, for all the spectacular advances in basic science and breakthrough treatment options for unmet patient needs, incremental innovation remains pivotal to the industry’s business model.
Source: Clarivate
As these blockbuster candidates either gear up to enter major markets, or work to optimise their launch impact within the crucial first few years of market entry, companies are still investing heavily in recharging their drug development pipelines. Pipeline growth has slowed somewhat of late, though, both on a year-to-year basis and as a general trend over the last 10 years.
Citeline Clinical’s Pharma R&D Annual Review 2025 lists 23,875 pipeline prospects hoping to reshape pharmaceutical market dynamics with breakthrough or improved drug options in the near or more distant future. This is 4.6% more than when Citeline last took the pipeline’s temperature in January 2024.
The annual growth rate is, however, below the 7.2% seen in 2023’s analysis and is the lowest rate of increase recorded over the last decade, falling just short of 4.76% in 2021. Citeline did note that in-house technological disruption during 2024 may have diluted the latest pipeline figures somewhat, although the impact was unlikely to be substantial, while 4,546 new candidates in one year was still a significant lift.
Even with successful drug approvals, and all the effort, attrition and complexity involved in those, companies still have to deal with the frequently rocky transition from regulatory approval to viable market access. This means new drugs being available to the patients who need them, and under conditions (including pricing and reimbursement) that balance payers’ financial challenges with pharmaceutical companies’ commercial imperatives.
Despite the international harmonisation that has levelled the playing field for drug development and approval over the past several years, regulators and payers across markets may have different resources, agendas, methodologies and priorities. A successful drug launch must be designed, planned and executed with sufficient verve, creativity and sustainability to weather the many variations and obstacles of a global marketplace.
In this respect, it helps enormously to have suitable launch and market access management tools, such as SmartLaunch® and SmartAccess™, so that companies can track launch and market access milestones, timelines, opportunities and challenges in real time across countries, internal functional silos and layers of corporate management.
Ensuring these plans and their outputs are 100% visible, aligned and actionable organisation-wide will leverage the hard-won achievements of drug approvals into genuine and long-term launch excellence.