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Mommy, where do drugs come from?

11.16.2010

Innovation? Unmet needs? Where's the action?

An interesting new paper by Robert Kneller (University of Tokyo, Research Center for Advanced Science and Technology) The importance of new companies for drug discovery: origins of a decade of new drugs.

Here’s the abstract:

Understanding the factors that promote drug innovation is important both for improvements in health care and for the future of organizations engaged in drug discovery research and development. By identifying the inventors of 252 new drugs approved by the US Food and Drug Administration from 1998 to 2007 and their places of work, and also classifying these drugs according to innovativeness, this study investigates the contribution of different types of organizations and regions to drug innovation during this period. The data indicate that drugs initially discovered in biotechnology companies or universities accounted for approximately half of the scientifically innovative drugs approved, as well as half of those that responded to unmet medical needs, although their contribution to the total number of new drugs was proportionately lower. The biotechnology companies were located mainly in the United States. This article presents a comprehensive analysis of these data and discusses potential contributing factors to the trends observed, with the aim of aiding efforts to promote drug innovation.

Some relevant facts:

* Of the 252 new drugs approved by the FDA from 1998 to 2007

o 58% from pharmaceutical companies

o 18% from biotech companies

o 16% from universities, transferred to biotech

o 8% from universities, transferred to pharma

* 123/252 (49%) new drugs approved by FDA from 1998 to 2007 were for an unmet medical need

* 118/252 (46%) new drugs approved by the FDA from 1998 to 2007 were scientifically novel

* 24/252 (21%) new drugs approved by the FDA from 1998 to 2007 had an orphan designation

The full paper can be found here.

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Paul Krugman: For Death Panels before he was against them

11.15.2010

The media’s favorite economist Paul Krugman is under fire for these comments he made this past weekend on ABC regarding federal spending on health care.

“Some years down the pike, we’re going to get the real solution, which is going to be a combination of death panels and sales taxes.”

Krugman is now trying to correct the record, or so he says:

I said something deliberately provocative on This Week, so I think I’d better clarify what I meant (which I did on the show, but it can’t hurt to say it again.)

So, what I said is that the eventual resolution of the deficit problem both will and should rely on “death panels and sales taxes”. What I meant is that

(a) health care costs will have to be controlled, which will surely require having Medicare and Medicaid decide what they’re willing to pay for — not really death panels, of course, but consideration of medical effectiveness and, at some point, how much we’re willing to spend for extreme care

This is merely a euphemistic way of saying the government must decide who lives and who dies.

Do we really want Medicare bureaucrats determining the “medical effectiveness” of any given treatment or medication?

Sounds like a very slippery slope.

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Drug Makers Brace for Possible Cuts to FDA

11.15.2010

Via this week’s edition of Ad Age:

Drug Makers Brace for Possible Cuts to FDA

After Midterm Elections, Pharma Wary of Congressional Appetite for Reduced Spending on Federal Agency

The Nov. 2 elections that reshaped the structure of the U.S. House of Representatives are causing a headache in the pharmaceutical industry, which could lose millions as a hamstrung FDA leads to a clogged up drug pipeline.

With many congressional candidates riding into office on a platform of reduced spending, drug makers are bracing for what they believe could be cuts to government agencies, particularly to the Food and Drug Administration.

Astra Zeneca CEO David Brennan and FDA Commissioner Dr. Margaret Hamburg last week both told the Reuters Health Summit in New York that cuts to the agency would be detrimental. "I don't think that would be good for our industry. It doesn't look to me like that would speed things up," Mr. Brennan told Reuters, speaking of the time it takes the FDA to review new products, where delays of several months can cost pharma companies millions.

Ms. Hamburg cautioned the new Congress to think twice about FDA cuts.

"Not every function of government can be cut to the same degrees using the same tools. I think we should proceed with real care," she told Reuters. "It should be recognized if we can't do our job and do it well, there isn't any other entity that will backstop behind us. What we do really matters to health."

"I think the threat to cut FDA funding is real and it's a signal that FDA has to deliver on the promises that it made during the last PDUFA negotiations," said former FDA associate commissioner Peter Pitts, referring to the Prescription Drug User Fee Act, or the fees that pharmaceutical companies pay to the FDA. "It's a shot across the bow."

For the rest of the story, see here.

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Quote of the week

11.12.2010

Joel Ario of HHS’ Office of Insurance Exchanges comparing Mitt Romney’s health system overhaul in Massachusetts to the federal health care law:

“You could say Obamacare was Romneycare before it was Obamacare.”

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CMPI at Columbus Circle

11.12.2010

CMPI recently hit the streets of New York City to ask people if they expected the health care law to help them.

Watch the video below to hear their answers:

CMPI at Columbus Circle from CMPI on Vimeo.

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MIT enjoying NEWDIGS "over there"

11.12.2010

The European Medicines Agency (EMA) and MIT’s Center for Biomedical Innovation (CBI) and Center for International Studies (CIS) are launching a collaborative research project with a focus on enhancing regulatory science in pharmaceuticals.

Specific questions addressed by this project include how to:

* adapt current regulatory requirements to support the efficient development of safe and effective drugs;

* incorporate patient valuation of health outcomes and benefit-risk preferences into regulatory decision-making;

* implement 'staggered' and 'progressive' approaches to drug approval;

* improve fulfillment of post-marketing regulatory requirements.

The project will explore the feasibility of, priorities for and practical considerations of implementing demonstration projects on some of the issues addressed during the course of the research.

Sound familiar? It's what the FDA's Critical Path program was designed to do -- that is until it hit Congressional treacle embodied by a certain member who will shortly lose her majority status.

The data and recommendations from this project are expected to link to implementation of the Agency's Roadmap to 2015 and the CBI's New Drug Development Paradigms (NEWDIGS) research program.

The project is scheduled to be completed by December 2011.

Hopefully with a new chair at the helm of the House Agriculture (and FDA) Appropriations Committee, funding for the Reagan/Udall Center can be released and FDA can, once again, take a leadership role in developing the tools for 21^st century regulatory science.

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Remembering Our Veterans

11.11.2010

Reagan's D-Day Speech at Normandy Beach still resonates and recalls the courage mustered by everyday Americans to preserve freedom.

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Death rates in U.S. and England

11.11.2010

A new study conducted by the Institute for Fiscal Studies in London and RAND Labor and Population offers an interesting look at both the British and American health care systems.

From the study:

In the new study, researchers examined the prevalence of illness among those 55 to 64 and 70 to 80. They also looked for the first time at the onset of new illnesses in those age groups in the United States and England during the years spanning 2002 to 2006. Finally, researchers examined trends in death rates in each country.

The findings showed that both disease prevalence and the onset of new disease were higher among Americans for the illnesses studied -- diabetes, high-blood pressure, heart disease, heart attack, stroke, chronic lung diseases and cancer. Researchers found that the higher prevalence of illness among Americans compared to the English that they previously found for those aged 55 to 64 was also apparent for those in their 70s. Diabetes rates were almost twice as high in the United States as in England (17.2 percent versus 10.4 percent) and cancer prevalence was more than twice as high in the United States (17.9 percent compared to 7.8 percent) for people in their 70s.

In spite of both higher prevalence and incidence of disease in America, death rates among Americans were about the same in the younger ages in this period of life and actually lower at older ages compared to the English.

Researchers say there are two possible explanations why death rates are higher for English after age 65 as compared to Americans. One is that the illnesses studied result in higher mortality in England than in the United States. The second is that the English are diagnosed at a later stage in the disease process than Americans.

"Both of these explanations imply that there is higher-quality medical care in the United States than in England, at least in the sense that these chronic illnesses are less likely to cause death among people living in the United States," Smith said.

"The United States' health problem is not fundamentally a health care or insurance problem, at least at older ages," Banks said. "It is a problem of excess illness and the solution to that problem may lie outside the health care delivery system. The solution may be to alter lifestyles or other behaviors."

Megan McArdle has further analysis of the study here.

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A Tangled Webb

11.10.2010

Virginia Senator Jim Webb on his conversation with President Obama about the health care bill:

"I told him this was going to be a disaster. The president believed it was all going to work out."

Yet Senator Webb voted in favor of the bill. Apparently talking out of both sides of one’s mouth never goes out of vogue in Congress.

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Long Time Companion (Diagnostics)

11.09.2010

Industry is anxiously awaiting guidance from FDA on the development of companion diagnostics for drugs that will inform efforts to move towards personalized medicine. The agency has vowed to release a draft guidance on companion diagnostics by the end of the year. But the initial document will address development of companion diagnostics for drugs already on the market, not simultaneous drug/diagnostic development for new drugs.

According to Vicki Seyfert-Margolis, FDA’s senior advisor for science innovation and policy:

“One challenge is that multiple centers have to be involved – CDRH and CDER/CBER. The challenge is internal to determine the mechanism for how the applications will come in and how the review is done. I think we are well on our way to figuring out that process between the different centers. With respect to how one designs and qualifies a diagnostic versus a clinical trial, the study design aspects are another challenge.”

“We are also concerned that we will have markers and use patient selection strategies in a clinical trial and then that marker may turn out to be a disease prognostication marker and you may have selected a set of patients that have different metabolisms. There are a myriad of possibilities one could think of that may or may not completely relate to the drug. We just have to be very thoughtful. I think we will evaluate a lot on a case-by-case basis, but we are trying to at least get some information out about what the paths are.”

And then there’s the issue of the “open kimono.”

Ms. Seyfert-Margolis: “One thing I think would be a big win in helping drive personalized medicine and co-development and companion diagnostics will be to open the data. We need to open the data in-house and also strive to get industry to be more transparent and work with us on this. If we took rheumatoid arthritis, for example, and looked across all the trials that have been done with TNF-alphas and evaluated who are the true responders, who are the non-responders, is there anything we can find in all that data that might point to some marker or some diagnostic that could be used, then we could begin to get at least hints about things that could open up new scientific areas for predictive diagnostics.

Amen. Sounds like a good way to involve the Reagan/Udall Foundation.

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Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.