While the US pharmaceutical industry reports that its R&D expenditures increased 147% during 1993-2004, from nearly $16 billion to close to $40 billion, the number of New Drug Applications (NDAs) which drugmakers submitted to the US Food and Drug Administration during this period rose just 38%, and the NDAs relating to New Molecular Entities (NMEs) went up only 7%, a new government study has revealed.
The FDA approved 76% of all NDAs submitted by drugmakers during 1993-2004, but both the number submitted overall, and those for NMEs, have been declining since 1996, reports the US Government Accountability Office, which has analyzed data on all 1,264 NDAs submitted to the FDA during the period.
Experts consulted by the GAO study on the reasons for the decline in NDAs for innovative new treatments say these may include: limitations on the scientific understanding of how to translate research discoveries into safe and effective drugs; drugmakers’ business decisions; uncertainty over regulatory standards for determining whether a drug should be approved; and certain intellectual property protections, says the Office. To address these issues, it adds, the experts have called for increased collaboration between government, industry and academia in the drug development process and in the development of scientists who can translate scientific breakthroughs into practical results. They also suggest that the government could consider providing additional financial incentives, such as longer patent lives for innovative treatments and shorter patent terms for “me-too” drugs.
However, the three Democratic legislators who requested the study – Representative Henry Waxman and Senators Edward Kennedy and Richard Durbin - say the report contradicts “the myth” that higher research expenditures have resulted in more treatment options for patients and it refutes many industry claims about the drug development process.
Most approvals are for 'me-too' drugs
The findings show that the majority of drug approvals are not for critical breakthrough drugs, with an estimated 60% being for “me-too” drugs but only 32% of applications relating to NMEs - and just 12% are for “priority” NMEs, say the legislators. Moreover, they claim, the study shows that innovation is being reduced by patent law loopholes and also by industry business practices such as the large amount of merger and acquisition activity in the sector and research which focuses on profitable but non-innovative drugs
Most prominently, said Rep Waxman, the study indicates that the link between high research expenditures – “which the industry claims must be driven by high prices - and new drug development is unclear, at best.” It shows that many aspects of the drug development system need to be examined to determine how to encourage research that focuses on breakthrough treatments rather than drug industry profits, he added.
Sen Durbin said the findings “raise serious questions about the pharmaceutical industry claims that there is a connection between new drug development and the soaring price of drugs already on the market. Most troubling is the notion that pharmaceutical industry profits are coming at the expense of consumers in the form of higher prices and fewer new drugs,” he added.
Sen Kennedy noted the importance of ensuring that “every penny of our national investment in health care is well spent, and does not go toward improperly subsidising the marketing and promotional activities of drug companies.”
However, the Pharmaceutical Research and Manufacturers of America said the report confirms that the journey from laboratory to medicine cabinet has become more challenging, risky and expensive than ever before.
“Researchers are tackling increasingly complex diseases using new tools, such as genomics, proteomics and nanotechnology, that will take years to bear fruit. As the cost of discovering new medicines has risen, an increasingly challenging healthcare environment is making it harder for companies to recover these costs,” said PhRMA senior vice president Ken Johnson.
Nevertheless, he added that the pace of scientific discovery had increased exponentially in recent years, and he pointed out that more than 300 new medicines have become available in the last ten years, while over 2,000 are currently in development. These include 646 new treatments for cancer, 146 for heart disease and stroke, 77 for HIV/AIDS and 56 for diabetes, he added.