What does 2025 hold for pharma? Hold onto your hat…

By Darcy Grabenstein

It’s that time of year, when we look into our crystal ball to see what the future holds for the pharmaceutical industry. While we see many recurring themes, 2025 promises to be filled with unexpected — and possibly unprecedented — developments. So buckle up and be prepared for a wild ride.

AI

Although we may tire of hearing about artificial intelligence in the life sciences (or any industry, for that matter), we’ll have to get used to it. Don’t look for this topic to go away any time soon.

While there’s a lot of buzz surrounding AI, industry experts emphasize the need for guardrails. As Claire Riches, VP of Clinical Solutions for Citeline, noted at the Citeline Elevate event earlier this year, the risks associated with the application of AI in clinical research are “the elephant in the room.” However, the companies that work best are those taking that risk, combining clinical SMEs with data and AI, Riches said in a panel discussion at Nordic Life Science Days.

Luca Parisi, Citeline Director of Clinical Analytics, says the next phase of AI adoption is already under way in pharma. He cites several examples:

• AI can be employed to develop “digital twins” for enhanced personalized medicine.
• GenAI platforms are accelerating drug discovery by optimizing molecular designs.
• EdgeAI is helping support more decentralized trials with remote patient monitoring through the use of wearable devices.
• Quantum machine learning (ML) can predict alternative treatment pathways and their outcomes.
• Quantum AI can enhance encryption of subject-level data such as real-world data (RWD) and electronic health records (EHR).

Clinical trial diversity

The issue of diversity in US clinical trials was brought to the forefront in 1986 when the National Institutes of Health adopted the Inclusion of Women and Minorities in Clinical Research policy.

More recently, the FDA has drafted a guidance document on diversity actions plans (DAP), designed to improve enrollment of participants from underrepresented populations in clinical studies. The FDA is required to issue final DAP guidance no later than June 2025 (nine months after the comment period closed on the draft guidance).

What was simply a “nice to have” will now become mandatory as the guidance outlines the following requirements for Phase III or other pivotal clinical studies:

• Specify the sponsor’s rationale and goals for clinical study enrollment (separated by the age group, ethnicity, sex, and race of clinically relevant study populations)
• Describe how the sponsor intends to meet those goals

Also expect increased focus on other marginalized groups in clinical trials, including LGBTQIA+, pregnant women, and older individuals.

New administration in US

The election of Donald Trump as the next US president has sent shock waves throughout the pharmaceutical industry. Industry stakeholders were quick to react to the election results and weigh in on the new administration’s potential impact on the life sciences. “We are committed to working with the Trump administration and the new Congress to make our healthcare system work better for patients,” said Pharmaceutical Research and Manufacturers of America (PhRMA) President and CEO Stephen Ubl in a statement.

Trump’s pick to head the FDA is Marty Makary, known for challenging the medical establishment. While Makary has voiced support for evidence-based medicine, he also has diverted from or misconstrued data to advance his positions, so his appointment is being met with cautious optimism. His decisions are likely to come under the microscope.

Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA), told Citeline’s Scrip he does not think the FDA’s expertise will be threatened by the Trump administration.

Trump’s pick for Health and Human Services (HHS) Secretary is Robert F. Kennedy Jr., known for his opposition to vaccines. Former FDA Commissioner Scott Gottlieb and current FDA Commissioner Robert Califf are unsure about the future of the agency.

On a brighter note for the industry, Vivek Ramaswamy, Trump’s choice to co-lead a new Department of Government Efficiency, has advocated for less stringent reviews of drugs and faster approvals.

With a Republican-led administration, the BIOSECURE Act may be closer to passage. The act prohibits entities receiving federal funds from using biotechnology from a company associated with a foreign adversary, such as China. “A Trump win brings uncertainty along a number of fronts for the biopharma industry,” says Pink Sheet Editor in Chief Nielsen Hobbs, “but one bit of clarity is that the BIOSECURE Act seems to have a greater chance of passage. The pharma industry remains committed to the Chinese market and will continue to be so even if BIOSECURE passes.”

In his first term as president, Trump withdrew the US from the World Health Organization (WHO). Biden reinstated the country’s membership, but it would be no surprise if Trump rescinds that order.

Clinical trial disclosure

According to a member of the Drug Information Association (DIA), Europe is driving disclosure at the moment, and Francine Lane, Citeline Vice President of Global Transparency, predicts it will continue to do so. With the full migration to CTIS complete by the end of January (at least officially), Europe will likely continue to shape disclosure in the year to come.

Phase two of the European Medicines Agency Policy 0070 will be enacted in April 2025. Lane says to look for more guidance on Policy 0070 and the clinical trial regulation (CTR) requirement to submit clinical study reports (CSRs) at the time of product approval.

Additionally, the UK announced in October it will finalize this year its highly anticipated post-Brexit clinical trial regulations, anticipated to go into effect in 2026.

Citeline Head of Transparency Operations Thomas Wicks is prepared for a slew of disclosure-related trends in 2025, including:

• Increased demand for plain language summaries (PLS), with the EU and UK leading the way and more countries following suit
• More sophisticated methods for data anonymization and redaction to ensure compliance with GDPR and the upcoming European health data space (EHDS) requirements
• A shift in focus from compliance to quality and consistency of disclosed information, including better data structuring for indications, outcome measures, and eligibility criteria
• The AI trend extends to disclosure, from PLS and trial results summaries to redaction and reporting

A look at APAC

Looking beyond the US and Europe, our APAC experts provide an overview of what the industry can expect. The most impactful life sciences trend in 2025 will be the region’s normalization as a leader in advanced clinical trials on the world stage, says Annie Siu, Citeline Director of APAC Content.

In China, Dexter Yan, senior editor, and Xu Hu, senior reporter, note that Chinese drugmakers will increasingly pursue original innovation, including first-in-class therapies, in addition to their current “fast-follower” R&D strategy. The region will likely see more activity in cell and gene therapy in response to last year’s policy opening this sector to direct foreign investment in four free-trade zones within China. In addition, foreign big pharma companies are anticipated to expand innovative drug development in China. Lastly, M&A activity involving Chinese biotechs and big pharma may pick up amid ongoing fundraising challenges for bioventures.

The election of Trump will likely have a ripple effect in Japan, as it may impact Japanese pharma companies’ large sales in the US, according to Citeline Managing Editor Lisa Takagi and Editor-in-Chief Ian Haydock. Japan also faces political and policy uncertainties around a weakened Liberal Democratic Party after recent national elections there. They expect a continued easing of regulations on rare and infectious diseases to address the “drug loss/drug gap” with other major global markets. As Japan’s rapidly aging society maintains pressure on the National Health Insurance system, there will be regular reimbursement price revisions, while domestic production of essential drugs will be encouraged.

Actions in the US will likely have ramifications for India, whose pharmaceutical market could benefit from the incoming Trump administration. All eyes are on the trajectory of the BIOSECURE legislation, says APAC Executive Editor Anju Ghangurde. The current momentum shows big pharma outsourcing to capability centers in India. India’s revised good manufacturing practices (GMP) regulations will go into effect for smaller firms, which may weed out many that cannot comply. However, there are rumors of a compliance date extension, and clinical trial waivers could help accelerate new drug launches. In terms of innovation, efforts at major Indian firms will be backed by in-licensing to fill portfolio gaps and the partnering of assets.

Few surprises are in store for South Korea, which is in line with global trends. Senior Editor Jung Won Shin says the Korean pharma industry will likely continue to actively seek R&D and assets in new modalities such as antibody-drug conjugates and targeted protein degradation, as well as in obesity/metabolic disorders, given the global growth in these sectors. Korea is also seeing a surge of interest in AI, with AI applications in drug development expected to increase amid new government plans in this area. And the contract development and manufacturing organization (CDMO) industry, particularly for biologics and cell/gene therapies, is expected to continue to grow amid rising demand globally.

Weight-loss drugs

GLP-1 weight-loss drugs have tipped the scales in terms of media coverage, and don’t expect that to lighten up in the near future. That’s because it has been estimated the obesity drug market could be worth $126 billion by 2030, according to Citeline’s sister company Evaluate.

The competition between obesity drugs Zepbound and Wegovy has gone back and forth like a yo-yo diet. Lilly’s Zepbound outperformed Novo Nordisk’s Wegovy in a head-to-head trial in patients with obesity or overweight with at least one comorbidity. In December, Novo Nordisk results from its Phase III trial of CagriSema fell short of expectations. The drug helped people lose up 22.7% of their body weight over the course of the trial. However, Novo executives had hoped for a 25% weight loss, and investors had anticipated up to 27%. Citeline estimates that over 300 drug candidates are in the pipeline and expected to hit the market in the next five or so years.

Amylin agonists may be the next major category to reach the market, according to In Vivo. Amylin, a hormone co-secreted with insulin, is similar to GLP-1: It makes people feel full for longer and inhibits glucagon (a hormone that raises blood sugar). Unlike GLP-1, those on amylin still feel hunger and may be less likely to experience nausea.

In Vivo reports that others looking to suppress appetite without GI side effects are revisiting the cannabinoid system, targeting peripheral cannabinoid (CB1) receptors found in key metabolic organs including the liver, gut, muscle, and fat tissue. This approach may address another weakness of GLP-1s: loss of lean mass.

Other approaches focus on metabolism. Mitochondrial uncoupling is a natural process that turns fat and sugar into heat rather than into the energy-carrying ATP molecule. Also being studied is AMP-activated protein kinase (AMPK) activation, which boosts energy-producing pathways like carbohydrate and fat metabolism and blocks energy storage processes like lipogenesis. Yet other approaches focus on inflammation of the hypothalamus, the brain’s hormone control center, which underpins obesity because of a dysregulated appetite and adipose metabolism.

DCTs making a comeback?

Following their peak during the coronavirus pandemic, decentralized clinical trials (DCTs) seemed to have dropped off the radar for the most part in 2024. With the rise in AI, however, there appears to be a renewed interest in DCTs. Continued efforts to promote diversity and patient-centricity in clinical trials, as mentioned above, favor a decentralized approach.

In September, the FDA issued its final guidance on DCTs. One goal of the guidance is to reduce the burden on clinical trial participants, reflecting a focus on patient-centricity that is certain to remain an industry priority. In fact, the patient-centric approach extends beyond how clinical trials are conducted, such as DCTs, to actual recruitment of patients. Sponsors are looking beyond traditional approaches to take into account patient preferences for everything from trial information to community portals and promotional materials.

COVID’s silver lining??

A Northwestern Medicine study published in The Journal of Clinical Investigation has revealed a connection between COVID-19 infection and cancer regression. Scientists observed that the RNA from the SARS-CoV-2 virus — responsible for COVID-19 — triggered the development of a unique type of immune cell with anti-cancer properties. The discovery holds promise for new approaches to cancer treatment.

With 2024 in the rearview mirror, the pharma industry must be prepared for new twists and turns in the road ahead. Successful organizations will be prepared to shift gears as needed to accommodate the volatile pharmaceutical landscape.