Good News For Pharma/ Med-Tech Investor: FDA approves 35 tdrugs in 2011

FDA: 35 innovative new drugs approved in fiscal year 2011 Report shows quick approvals of safe and effective medicines occur in the United States before other countries

Over the past 12 months, the U.S. Food and Drug Administration approved 35 new medicines. This is among the highest number of approvals in the past decade, surpassed only by 2009 (37). Many of the drugs are important advances for patients, including: two new treatments for hepatitis C; a drug for late-stage prostate cancer; the first new drug for Hodgkin’s lymphoma in 30 years; and the first new drug for lupus in 50 years.

In a report released today, FY 2011 Innovative Drug Approvals, the FDA provided details of how it used expedited approval authorities, flexibility in clinical trial requirements and resources collected under the Prescription Drug User Fee Act (PDUFA) to boost the number of innovative drug approvals to 35 for the fiscal year (FY) ending Sept. 30, 2011.  The approvals come while drug safety standards have been maintained.

The report shows faster approval times in the United States when compared to the FDA’s counterparts around the globe. Twenty-four of the 35 approvals occurred in the United States before any other country in the world and also before the European Union, continuing a trend of the United States leading the world in first approval of new medicines. ‬‪

“Thirty-five major drug approvals in one year represents a very strong performance, both by industry and by the FDA, and we continue to use every resource possible to get new treatments to patients,” said Margaret Hamburg, M.D., Commissioner of Food and Drugs. “We are committed to working with industry to promote the science and innovation it takes to produce breakthrough treatments and to ensure that our nation is fully equipped to address the public health challenges of the 21st century.”

Among the new drugs approved in FY 2011, a number are notable for their advances in patient care and for the efficiency with which they were approved:

• Two of the drugs – one for melanoma and one for lung cancer – are breakthroughs in personalized medicine. Each was approved with a diagnostic test that helps identify patients for whom the drug is most likely to bring benefits;

• Seven of the new medicines provide major advances in cancer treatment;

• Almost half of the drugs were judged to be significant therapeutic advances over existing therapies for heart attack, stroke and kidney transplant rejection;

• Ten are for rare or “orphan” diseases, which frequently lack any therapy because of the small number of patients with the condition, such as a treatment for hereditary angioedema;

• Almost half (16) were approved under “priority review,” in which the FDA has a six month goal to complete its review for safety and effectiveness;

• Two-thirds of the new approvals were completed in a single review cycle, meaning sufficient evidence was provided by the manufacturer so that the FDA could move the application through the review process without requesting major new information;

• Three were approved using “accelerated approval,” a program under which the FDA approves safe and effective medically important new drugs quickly, and relies on subsequent post-market studies to confirm clinical benefit. For example,  Corifact, the first treatment approved for a rare blood clotting disorder, was approved under this program; and

• Thirty-four of 35 were approved on or before the review time targets agreed to with industry under PDUFA, including three cancer drugs that FDA approved in less than six months.

The Prescription Drug User Fee Act was established by Congress in 1992 to ensure that the FDA had the necessary resources for the safe and timely review of new drugs and for increased drug safety efforts. The current legislative authority for PDUFA expires on Sept. 30, 2012. 

“Before the PDUFA program, American patients waited for new drugs long after they were available elsewhere,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “As a result of the user fee program, new drugs are rapidly available to patients in the United States while maintaining our high standards for safety and efficacy.”

In October 2011, the FDA released a new plan, Driving Biomedical Innovation: Initiatives to Improve Products for Patients, to assist companies engaged in new product development, particularly smaller, entrepreneurial companies.

 

"FDA Impact on U.S. Medical Technology Innovation,”

By: The Advanced Medical Technology Association (“AdvaMed”), prepared by Josh Makower, M.D., consulting Professor of Medicine, Stanford University 
A medical device meeting recently reported that “Growth in the life sciences industries will greatly depend on how the FDA responds to growing complaints that they are stifling medical technologies.  Companies have seen a vast difference in the approval processes in Europe vs. the U.S.  A study released by Stanford University indicated that the cost of bringing a technology to market is dramatically lower in Europe.”
The Advanced Medical Technology Association (“AdvaMed”) recently released the report, prepared by Josh Makower, M.D., consulting Professor of Medicine, Stanford University and Founder, President & Chief Executive Officer of ExploraMed Development, LLC; et al, “FDA Impact on U.S. Medical Technology Innovation,” which garnered responses from more than 200 companies concerning their experiences in working with the FDA.  Participants were also asked about their experiences working with European regulatory authorities in order to offer a comparison between aspects of the two dominant regulatory systems.
“In general, survey respondents viewed current U.S. regulatory processes for making products available to patients as unpredictable and characterized by disruptions and delays,” the results summary states.  Forty-four percent (44%) indicated that part way through the premarket regulatory process they experienced untimely changes in key personnel, including the lead reviewer and/or branch chief responsible for the product’s evaluation.  Thirty-four percent (34%) of respondents also reported that appropriate FDA staff and/or physician advisors to the FDA were not present at key meetings between the FDA and the company.”
The report goes on to highlight that those factors contribute to significant delays in navigating FDA regulatory processes, with premarket process for 510(k) pathway devices (of low-to moderate risk) taking an average of 10 months from first filing to clearance.  Devices requiring a clinical study for low- to moderate-risk devices before making a regulatory submission, the premarket process took an average of 31 months from first communication to being cleared to market while, in comparison, it took an average of 7 months in Europe.
For higher risk devices seeking premarket approvals, responding companies indicated that it took an average of 54 months to work with the FDA from first communication to being approved to market the device.  In Europe, it took an average of 11 months.”
Beyond the time gap comparing FDA and Europe approval processes, the survey also showed that the average total cost for a low- to moderate-risk 510(k) product from concept to clearance was approximately $31 million, with $24 million spent on FDA dependent and/or related activities.  For a higher-risk PMA product, the average total cost from concept to approval was approximately $94 million, with $75 million spent on stages linked to the FDA.
According to the report, these statistics result in a “significant, measurable cost to U.S. patients in the form of a device lag.  Respondents reported that their devices were available to U.S. citizens, on average, nearly 2 full years later than patients in other countries, due to delays with the FDA and/or company decisions to pursue markets outside the U.S. before initiating time-consuming, expensive regulatory processes in their own country.”

Stephen J. Ubl, President & Chief Executive Officer of AdvaMed, says, “This report is a wake-up call for those who want to promote medical innovation and preserve American jobs.  A regulatory environment that is marked by needless delays and inefficiencies makes it harder for medical innovation to thrive and companies to survive.  These delays particularly hurt small companies and their ability to produce next generation technologies.”
To read the full report go to: http://www.advamed.org/NR/rdonlyres/040E6C33-380B-4F6B-AB58-9AB1C0A7A3CF/0/makowerreportfinal.pdf
Ultimately, growth within the life sciences will continue at a quick pace during the next few years, with development of R&D pipelines, alliances, and partnerships being a key factor for success.
As the economy continues to chug back to full force, the only obstacle appears to be the differing of opinions between the medical device manufacturers and the FDA.  Perhaps if they come to a meeting of the minds, these growth projections will not just be projections, but will be the reality of a growing field that is quickly, and effectively, delivering what the U.S. healthcare system requires.”