The Pharmaceutical Research and Manufacturers of America (PhRMA) served up two reports Thursday touting U.S. biopharma's "innovative and robust" pipeline of some 5,400 new molecular entities (NMEs), including more than 800 in Phase III studies. "Innovation in the Biopharmaceutical Pipeline," developed by the Analysis Group Inc. with support from PhRMA, offered a review of therapies that have advanced to the clinic, while an accompanying PhRMA paper, "The Biopharmaceutical Pipeline," highlighted key findings of the larger report.
The reports were long on data but short on analysis, characterizing 70 percent of pharma's pipeline as potentially first in class and silent on the biotech underpinnings of much of big pharma's drug discovery engine, not to mention the peaking patent cliff and lopsided partnering arrangements still bedeviling many early stage companies.
A PhRMA spokeswoman said the organization does not track partnership deals between large biopharma companies and smaller biotechs internally. She cited a March 2012 Burrill & Co. report that noted a growing trend in drug development partnerships, totaling nearly $38 billion in 2011.
In a lively social media exchange following a PhRMA webinar to discuss the reports, industry analysts and observers also questioned some of the findings for example, the 85 cardiovascular compounds cited in Phase III development.
The Analysis Group characterized development activities focused on NMEs as potentially first in class if they were "described by a unique pharmacological class distinct from those of any other marketed products." By that definition, the data collected from the EvaluatePharma commercial database classified 78 percent of projects in Phase I, 69 percent in Phase II and 45 percent in Phase III as potentially first in class. John Castellani, PhRMA president and CEO, suggested in a webinar introducing the report that the breadth of innovation reflected the maturation of science as drug developers learn more about the nature of disease. Cancer, for instance, "is not one disease but a great number of diseases" with correspondingly diverse targets, he observed.
With dozens of compounds in development in certain categories, however, such as the hot phosphatidylinositol-3-kinase inhibitor class, the data seemed to exaggerate the scope of true innovation in drug development.
Indeed, "only one molecule in a given class can eventually win first-in-class designation," the Analysis Group report conceded. "However, it cannot be known in advance which molecule will proceed through clinical testing and be approved first."
The report did not attempt to make those predictions, though it observed that potential first-in-class medicines were especially prominent in neurology (84 percent), psychiatry (80 percent), cancer (80 percent) and diabetes (79 percent). More than 1,600 projects were under way in neurology, alone, according to the Analysis Group.
The Analysis Group report did not address recent failures in high-profile clinical trials, so it wasn't clear whether its findings excluded Phase III Alzheimer's disease misses last year by Eli Lilly and Co. in the double-blind, placebo-controlled EXPEDITION trials of solanezumab or by the Johnson & Johnson/Pfizer Inc.-partnered bapineuzumab, an anti-amyloid beta antibody originally discovered and developed by Elan Corp. plc, along with numerous early and late-stage failures in other neurology indications. (See BioWorld Today, Aug. 8, 2012, and Aug. 27, 2012.)
The reports extolled the number of medicines in the pipeline addressing diseases for which no new therapies have been approved in the last decade. For example, 158 potential medicines are in development for ovarian cancer, 19 for sickle cell disease and 41 for small-cell lung cancer, according to the Analysis Group report.
Personalized medicines also account for a growing proportion of the biopharma pipeline. In addition, the number of potential new medicines for rare diseases averaged 140 per year over the last 10 years compared to 64 in the previous decade, with 1,795 development projects granted an FDA orphan designation as of October 2011, according to the Analysis Group.
The reports did offer interesting snapshots of the scientific approaches drug developers are pursuing. Broad classes of scientific platforms that were readily identifiable in the EvaluatePharma dataset revealed that 245 projects are using cell therapy, 127 are using antisense RNA interference therapy, 102 are using monoclonal antibodies joined to cytotoxic agents and 99 are using gene therapy.
In the webinar, Castellani also sounded a hopeful note on the regulatory front, noting a record high 39 compounds were approved by the FDA in 2012.
The number of new drugs in development has grown by 40 percent since 2005, added John Lechleiter, chairman, president and CEO of Lilly and chairman of PhRMA.
Innovation "doesn't happen in a vacuum," he said, noting the pharma industry has been able to advance scientific discovery due to public policies that supported intellectual property, appropriate regulation and free market access for patients. Going forward, pharmas and biotechs taking development risk want continued assurance they'll be able to profit from innovation.
"We need a regulatory system that is clear and transparent and serves to facilitate the development and approval of new medicines while safeguarding patients," Lechleiter said. "We also need an opportunity to get a fair return based on a benefit these products provide by letting market mechanisms work."
In that regard, changes in the U.S. health care system loom large as a potential obstacle to sustained drug development, Castellani cautioned. Innovation can be considered both a cost and a cost savings, he said, noting that development of effective therapeutics to manage HIV did not bankrupt the U.S. health care system, as some originally feared. Whether the nation will now diminish the long-term value of medicine in an attempt to squeeze dollars in the near term is "the biggest danger to the innovation process," he said.