Examining the Data on the Pipeline Report
Examining the Data on the Pipeline Report
01.25.13 | By Jennifer Wall
Last week we released a new report by Analysis Group examining innovation in the biopharmaceutical pipeline. The report looked at the pipeline from several angles and presented many interesting findings – even some that were surprising.
The pipeline is remarkably robust. There are more than 5,000 drugs in development and of those about 70% have the potential to be first-in-class.
Research in new areas like personalized medicine and therapeutic cancer vaccines, for example, are flourishing.
For those of us who like to dig deep into studies like this, we asked one of the co-authors, Genia Long, managing principal at Analysis Group, to take us behind the numbers to shed light on the methodology:
"There is good reason to celebrate the news that the FDA issued 39 new drug approvals in 2012 – the highest number for 16 years – but what might the future hold over the coming years, as suggested by the pipeline of new therapies in development? That is the question we tried to help shed some light on in the recent report Innovation in the Biopharmaceutical Pipeline: A Multidimensional View.
We focused primarily on medicines in clinical development, those where clinical trials in human volunteers have already begun (i.e., those projects characterized as being in Phase I, II, III, having been filed with the FDA, or having been approved by the FDA but not yet on the market in the U.S.):
- That meant we analyzed information on some 8,000 projects described by a curated proprietary database as being in clinical development, corresponding to some 5,400 distinct medicines in clinical development.
- In addition, some data are presented for another 9,000 projects in pre-clinical development, for approximately 17,000 projects overall.
Strengths and Limitations of New Drug Development Data
Because the data are collected from publicly available sources (e.g., announcements by firms, coverage by industry analysts, government websites) and curated in a proprietary database by the data provider, they reflect the strengths and limitations of the underlying data. To the degree that firms do not announce current information, those data may not be complete or may be out-of-date. Companies may be slow to announce publicly that a development project has begun (particularly in pre-clinical development), or slow to announce that a previously announced but stalled project will not proceed. Likewise, the data on specific development projects, including indication and pharmacologic class, may evolve and become more specific over time. Data were as of December 12, 2011 (unless otherwise noted).
Use of the Terms “Product” Versus “Project”
- We used the term “product” to describe a unique molecule in development (e.g., a particular recombinant protein).
- We used the term “project” to describe unique molecule-indication combinations (e.g., a particular recombinant protein for colorectal cancer, rather than breast cancer).
- In the project totals, a given molecule being investigated in multiple indications is counted once for each indication; multiple companies pursuing the same molecule for the same indication would be counted only once.
- In the product totals, a given molecule would be counted only once, and only if it has not yet been approved by the FDA and is on-market in the U.S.
U.S. Patients and Global Development
While our primary interest was in drugs in development for the U.S. market with the potential to aid U.S. patients, development is inherently a global activity, with clinical sites and laboratory activity located around the world, and it is not possible to know in advance which specific therapies of the thousands in development will surmount the many obstacles to be faced during the clinical development process, and will also be submitted to the FDA for approval for marketing in the U.S. rather than other jurisdictions. Because most medicines are intended for launch in the U.S., the largest drug market in the world, we did not exclude any medicines in development from the totals presented. We did exclude from our counts of products and projects which were in regulatory review or approved but not yet on the market in the U.S. those drugs which were not filed with the FDA (i.e., those listed without an FDA file date).
Focus on Potential New NMEs and New Indications for NMEs in Clinical Trials
We restricted our review to development activity characterized in the data as potentially resulting in new molecular entities (NMEs) if approved, or new indications for previously approved NMEs (when reporting data for projects). We did not review or include in the data either new formulations of existing medicines or post-approval/Phase IV trials, where the molecule had already been approved for the given indication.
In calculating the number of projects in clinical development with the potential to be first-in-class, any molecule in a given new class might have the potential to be first-in-class, and so all projects in development were included in the totals provided. In the end, only one can win that designation and be first-in-class in a given indication, however. If others are approved and marketed in the U.S. for the same indication (if not otherwise precluded by patent or other considerations) they would not be first-in-class; more likely, they would not proceed successfully through clinical testing and other obstacles. It cannot be known in advance which specific molecule, if any, will surmount these many obstacles and be approved first.
Developing a new medicine is a long and complex process, and the vast majority of the thousands of medicines in development will not surmount all the many hurdles that confront them – as previous research has shown, the vast majority of prospects early in the development process (some 70 to 90% of drugs in Phase I) will not demonstrate sufficient safety and efficacy to proceed further in clinical trials and be launched, or will be shelved for other prospects with more favorable profiles. Even a substantial number of agents in Phase III trials, which may encompass thousands of patients and many clinical sites around the world, do not proceed to approval and launch.
Because there are no guarantees in what is inherently a highly risky discovery, development, and review process, this report makes no predictions about which, or even how many, medicines will make their way all the way through the many year-long process. Rather, it highlights the range of possibilities – the medicines in development, some of which will benefit patients, their families, and their physicians in the future."