Cancers, Infectious Diseases Get Large Portion of New Drugs

Marcia Frellick

April 24, 2019

A record number of new drugs — 59 in the United States alone — were approved in 2018, according to "The Changing Landscape of Research and Development" report, released Tuesday by the IQVIA Institute for Human Data Science.

Of those new active substances (NASs), 27% are new therapies for cancer, and 20% are for the treatment of infectious diseases.

Murray Aitken, MBA, director of the institute, described to reporters in a press teleconference on Monday "positive momentum" from the amount of investment and research that has resulted in new drug launches.

However, he also pointed to a disappointing 27% drop in productivity over 5 years, broadly defined as "success divided by effort."

"We continue to see stubbornly low levels of success rates and long durations for bringing scientific innovation through the R&D [research and development] process and to patients," Aitken said.

Cancer Drugs Were 40% of the 5-Year Pipeline Increase

The number of new drugs for cancers increased 63% during the past 5 years; new cancer drugs made up 40% of the total pipeline increase. Conversely, the number of vaccines declined during that period by 4%.

In the past year, other areas of therapy that have seen a large boost in new drug options are amyotrophic lateral sclerosis (ALS) and other degenerative musculoskeletal conditions, rare gastrointestinal diseases, and nonnarcotic pain treatments, the report indicates.

Fewer Participants in Studies

In 2018, almost half of new drugs (46%) were approved on the basis of trials that included fewer than 500 persons, reflecting the growing need for specialty, niche, and orphan drugs.

The report also indicated that the drug development process remains slow and risky: 2018 NASs in the United States required an average 13.7 years from patent filing to market. However, that was 2 years faster than drugs launched in the 2 years before and 6 months faster than the average over 5 years.

Number of Clinical Trials Up 9%

The total number of clinical trials that started in 2018 was up 9% from the year before and 35% during the past 5 years. Most of the growth comes from phase 2 trials, the report authors write, up 26% over the prior year and 61% over 5 years. The increase has been fueled by oncology and neurology trials.

The number of trials for gastrointestinal diseases and nonalcoholic steatohepatitis was up 42%, and oncology trials were up 27% during those 5 years. The number of trials of drugs to treat respiratory and endocrine diseases declined.

One Drug Approved in 10 Years for Alzheimer Disease

One of the notable areas of frustration has been in treatments for Alzheimer disease.

Since 2008, for Alzheimer disease, "only one product received regulator approval, while 86 other development projects were discontinued, including four in 2018," according to the report.

However, new treatments are in development.

The report states, "Treatments for nervous system disorders like MS [multiple sclerosis], Parkinson's, ALS, Alzheimer's and other neuromuscular disorders account for 18 next-generation biotherapeutics treatments, up from just five in 2009. Gene therapies are also under investigation for Parkinson's disease, Alzheimer's disease and spinal muscular atrophy."

5-Year Drop in Productivity

According to the report, productivity dropped overall by 27% from 2013 to 2018, largely because of a decrease in productivity in phase 1 trials.

"This speaks to what we recognize are ongoing challenges," Aitken said. "Those are particularly acute in oncology, which has a relatively low level of productivity, in part because it has a high level of complexity. And with the growing share of clinical trial activity being in the oncology space, that's been one factor that has brought the overall level of productivity down in 2018."

Many factors may lift productivity in the next 5 years, Aitken said, including improvement in digital health technologies to enable capture of drug efficacy and safety data remotely and to relieve work burden on trial sites, and an increase in focusing on patient-reported outcomes and use of artificial intelligence.

Databases consisting of data regarding people who agree to make their data available for research, such as those involved in 23andMe, and the Precision Medicine Initiative, are expected to help ensure that trials don't fail because of lack of recruitment, which can lead to shorter trials and faster times to market, the report authors predict.

"We're optimistic that we can foresee some sizeable and measurable improvements in productivity over the next 5 years," Aitken said.

Investment in Research Strong

The report adds that financial support for research is strong and growing. Venture capital firms invested more than $23 billion in new treatments 2018, and the 15 largest pharma companies reported spending more than $100 billion on research and development for the first time, up 32% during the past 5 years.

The report also highlights the importance of emerging biopharma companies, which the authors define as those that spend less than $200 million a year on research and development or have less than $500 million a year in revenue.

Emerging biopharma companies produced 72% of the drugs launched in 2018, and those companies registered almost half of them, Aitken said.

Aitken has disclosed no relevant financial relationships.

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