Clinical Research 2023: Highlights and Headlines

Contributing Expert: Nicole Latimer, CEO of Medrio

The past year has brought a series of highs and lows in clinical research. Some news has even captured public interest—things like Ozempic’s new study based on real-world evidence and psychedelic drug MDMA passing a key hurdle on its way to regulatory approval for PTSD treatment. 

Meanwhile, there was a huge amount of other news within clinical research that made a meaningful impact. 

Many new drug types entered the market, including some exciting digital therapeutics (DTx). Regulators also made big announcements, weighing in on topics like accelerated approvals within rare disease research.  

Here, we’re sharing a few of the biggest newsworthy items of 2023 within clinical research.

New Drug Types Enter the Market

Clinical research resulted in some huge wins this year, with many firsts entering the market. These include the first biosimilar to treat multiple sclerosis, the first orally administered fecal microbiota product for C. difficile re-infections, and an accelerated approval for an Alzheimer’s treatment. 

Diabetes research ushered in a new insulin pump with its own algorithm-based software and a new class of medicines for pediatric Type 2 diabetes. 

Women’s health research also saw breakthroughs: the first oral treatment for postpartum depression, the first nonprescription daily oral contraceptive, and a novel drug to treat moderate to severe hot flashes.

In much anticipated post-pandemic news, the FDA approved the first oral antiviral for COVID-19. Both the FDA and the EMA approved the first Respiratory Syncytial Virus (RSV) vaccine. The EMA also recommended granting marketing authorization for Abrysvo, a vaccine for babies and seniors to protect against disease caused by RSV.

New cell and gene therapies

Currently, there are 2,500 cell and gene therapy investigational new drug applications (INDs) on file with the FDA. In 2023, the FDA approved just five new therapies. A handful of approvals may seem small, but these drugs each represent massive advances.

A few weeks ago, regulators in the U.K. approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia. This landmark approval brings in a new era of genetic medicine. Since the initial lab experiments demonstrating CRISPR’s potential as a gene-editing tool were only published in 2012, this approval also shows the accelerated progress of genetic-based clinical research.

Other exciting cell and gene approvals included:

• The first topical gene therapy for treatment of wounds in patients with Dystrophic Epidermolysis Bullosa was awarded to Krystal Biotech.
• The first gene therapy for adults with severe hemophilia A 
• The first cellular therapy to treat patients with Type 1 diabetes
• A therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation

Breakthroughs within rare disease

Too often, rare diseases are fatal or have devastating impacts on patients and their loved ones. But research is gaining momentum. In September 2023, the FDA announced the launch of a pilot program to help further accelerate the development of rare disease therapies. 

They also approved 51 novel drugs in 2023, including several treatments for rare or orphan diseases:

• The first gene therapy for the treatment of certain patients with Duchenne Muscular Dystrophy 
• A new therapy for myelodysplastic syndromes, a rare form of blood cancer
• A two-component therapy for adults living with late-onset Pompe disease

DTx Shows Signs of Maturity

The DTx market is accelerating, and experts project that the digital health market will be worth 426.9 billion (USD) by 2025. In 2023, we caught a glimpse of this market’s potential as:

• Better Therapeutics received authorization for AspyreRx™, a treatment for adults with type 2 diabetes, after spending about a decade developing it. 
• Swing Therapeutics received approval for the first prescription DTx for treating fibromyalgia symptoms after completing multiple randomized controlled trials and real-world studies, including PROSPER-FM, the biggest device trial ever undertaken for fibromyalgia treatment

Some sponsors view DTx as a chance for faster market entry. However, this year emphasized the need to maintain high-quality research standards in this novel category. A just-released study found that “just two prescription digital therapeutics had been evaluated in at least one study that was randomized and blinded and that used other rigorous standards of evidence. Two-thirds of clinical studies of prescription digital therapeutics were conducted on a postmarket basis, with less rigorous standards of evidence than the standards used in premarket studies.” 

In October 2023, the FDA announced a new advisory committee to “help the agency explore the complex, scientific and technical issues related to digital health technologies (DHTs), such as artificial intelligence/machine learning (AI/ML), augmented reality, virtual reality, digital therapeutics, wearables, remote patient monitoring and software.”

Regulators Release New Guidance

This year marks a decade since the International Coalition of Medicines Regulatory Authorities (ICMRA) was established when eight regulatory authorities came together. In 2023, the ICMRA worked to establish key principles for the adaptation of COVID-19 vaccines.

In 2023, the FDA made some significant and groundbreaking moves, including announcing:

• Additional steps to modernize clinical trials. 
• Additional steps to advance decentralized clinical trials. 
• Draft guidance aimed at improving oncology trials for accelerated approvals. 

The agency also created a buzz when it issued its first draft guidance on clinical trials with psychedelic drugs. Interest in the clinical use of psychedelics began in the 1950s but the next two decades brought restrictive laws, effectively shutting down all US clinical research in this area. The recent regulatory movements signal regained momentum in psychedelic research. 

Meanwhile, the EMA also pushed for further progress by:

• Inviting comments on establishing efficacy based on single-arm trials.
• Hosting a workshop to explore clinical trial improvements during public health emergencies.

In Europe, the EMA’s mandate that all new clinical trial applications in the EU use the Clinical Trials Information System (CTIS) took effect on January 1, 2023. This huge change means sponsors can now “apply for authorisations in up to 30 EU/EEA countries at the same time and with the same documentation.” It also includes a public, searchable database.

Mergers and Acquisitions Abound

CROs are expanding their global footprint by acquiring other companies or moving into later-phase trials. For example, in March 2023 ClinChoice acquired CROMSOURCE, thus expanding their reach further into the US, along with seven countries throughout Western and Eastern Europe. In October, Veramed acquired CDRS, a Boston-based data management, biostatistics and medical writing services company. 

In 2023, we also saw new players enter the arena of clinical research. For example:

• In February, Labcorp, a global life sciences company, announced its spinoff clinical development business Fotrea—a new CRO providing Phase I-IV clinical trial management. 
• In March, Premier Health Associates and ObjectiveHealth announced their partnership for an on-site clinical research center named Premier Health Research.

Embracing The Future of Clinical Research

Clinical research is marching forward as it rises to modern challenges and opportunities. Undoubtedly, 2024 will bring even more innovation. 

We’re excited to see how clinical research more fully integrates artificial intelligence, pushing forward the need for data interoperability to support machine learning. We’ll also be watching for trailblazers and innovators chartering new treatments and trial designs. Lastly, we are eager for the development of additional ethical aspects of clinical research, including continued DEI advocacy. 

In 2024 the clinical research community will continue to build momentum in the direction of innovation. Reflecting on this year bolsters our resolve to leverage technology to expedite trials, improve data accuracy, and ultimately, deliver life-changing treatments to those who need them most.