The Cross-Border Biotech Blog

Biotechnology, Health and Business in Canada, the United States and Worldwide

Tag Archives: Comparative Effectiveness

Biotech Trends in 2011: Comparative Effectiveness and Personalized Medicine

When this blog was launched in 2009, comparative effectiveness and personalized medicine were fairly new features in the North American landscape. Our initial argument that they were related topics — determining which treatment is best depends on which patient is being treated – was soon bolstered by the comparative effectiveness provisions in the U.S. stimulus bill and new personalized medicine data via the FDA.

The proposition has since become common knowledge, culminating in statements by Francis Collins and at BIO 2010 and discussed in the New York Times. Personalized medicine is now a key strategy for 12-50% of current drug pipelines, according to a recent Tufts study, and is a significant driver for DNA sequencing technology companies. If anything, the pendulum has swung a bit too far towards ‘hype,’ and as Matthew Herper reminds us, there are still non-personalized potential blockbusters in the pipeline.

The two concepts have even merged in a motto:

“the right drug to the right patient at the right time,”

which I still don’t like as much as “Personalized Effectiveness” (my neologized mash-up), but seems to be sticking. We’re just going to call it “Personalized Medicine” for now and will continue to follow major developments. You can too, on this page.

This post is the second in a series briefly outlining the biotech industry trends we’ve been following on the blog and noting some recent developments, plus directions for 2011.

BIO Panel on Comparative Effectiveness Research Notes “Silver Lining” of Personalized Medicine

Speakers Daniel Todd, from EMD Serono, and Steve LaPierre, from Boston Scientific, were led by Foley Hoag lawyer Jayson Slotnik in a discussion of the final CER legislation and predictions about implementation. The overall tone was skeptical — the panel noted the potential for CER data to ultimately contribute to CMS coverage decisions, and worried about the cost of prospective randomized trials and about potential impacts on the FDA approval process.

They were, however, optimistic about the role of personalized medicine in the CER implementation. Steve LaPierre expects it to be helpful, and Daniel Todd advocates using the reference to molecular and genetic subtypes in the legislation to push for personalized analysis if a product is selected for a CER study. He calls personalized medicine a silver lining.

The panel also noted positive structural aspects, including helpful governance provisions in the CER legislation. Specifically, they were impressed with the public reporting and audit provisions and the availability of comment periods to allow private sector input.

In looking at how CER will shake out, the panel expected progressive adaptation of the program over the next 3-7 years.

Daniel Todd emphasized the importance of picking a first recommendation to establish credibility. The controversy this year about breast cancer screening recommendations shows the loss of credibility that can come from a debatable result, so they expect CER to ramp up over time.

He also predicted that dissemination of CER results through social media may drive bottom-up adoption of findings, and that defensive medicine may also contribute to adoption.

Bookmark and Share

Biotech Trends at BIO 2010

As I’m preparing for the BIO conference in Chicago next week, I’m excited to see that several of the biotech trends we’ve been following on the blog are showing up as conference sessions.

  • Interested in “A New Kind of Non-Dilutive Financing and Fundraising: Partnering With Not-for-Profits”? Get an early start at our trends page on Commercialization by non-profit foundations!
  • Does “Comparative Effectiveness Research and the Government Role” or “Transforming Health Care Through Personalized Medicine” catch your eye? Check out the stories we’ve highlighted on Comparative Effectiveness and Personalized Medicine!
  • Of course, with the new regulatory pathway created by Health Reform legislation in the U.S., Follow-on Biologics (aka Biosimilars) are all the rage at BIO this year.
  • and the whole thing kicks off with Lilly’s General Counsel speaking on “Leveraging IP to Spur Global Biotechnology Innovation, Investment and Jobs” – emphsizing the link between IP Constituencies and Global Innovation that we have been following for some time.

Stay tuned for news from these and other sessions as we hit the conference next week!

Bookmark and Share

Comparative Effectiveness and Personalized Medicine are “Part of the Same Question” Collins Confirms

In a very informative Kaiser Health News interview (via GenomeWeb), Francis Collins says that

“personalized medicine strategy and CER strategy are part of the same question. … There will often be more than one therapeutic intervention, so you have to compare them. But you also want to know what’s different about the individual that might have an influence on that answer.”

Couldn’t have said it better myself.

Bookmark and Share

Biotech Trends Update: A Personalized Critique of Comparative Effectiveness Misses the Mark

As the U.S. and Canada move to invest and rely more on comparative effectiveness research (CER), lack of personalization has been the loudest and most frequent objection.  That is why we have been following the interaction between comparative effectiveness and personalized medicine as a key industry trend.

Yesterday, an opinion piece in the WSJ by Leonard Zwelling, a professor of medicine and pharmacology at M.D. Anderson, came out strongly in opposition to CER.  Zwelling takes a number of swipes at CER, but most of the time he is focused on the inability of CER to personalize treatment – for a patient’s genes, age or preferences – in identifying a “best” treatment.  For example:

“If decisions based on CER inhibit the progress of personalized medicine—or in any way interfere with a meaningful interaction between doctor and patient to individually tailor the most appropriate therapy—no one is helped.”

Zwelling’s argument sets up more than a few straw men — that CER uses only retrospective data, that it will ignore qualify of life, and that treatment options change too quickly for CER to provide timely advice — but the main problem is that he assumes that CER and personalization are incompatible.

As this blog has noted on many occasions, CER is at its best when coupled with personalized medicine.  This post about KRAS genotyping is a great example.  This point is not lost on the Obama administration, which is aware of and sensitive to the need to account for personalized treatment in CER.  Zwelling himself opens with a quote from relevant legislation that says the current CER funding is supposed to find out

“what works best for which patient under what circumstances.”

The clause “for which patient” is a built-in acknowledgement of the importance of personalized approaches to CER. 

My bottom line: Both comparative effectiveness and personalized medicine are critical to medically effective and financially sustainable medical care and drug development.

Biotech Trends Update: Costs Savings from Personalized Medicine Sought by PBMs, Employers, Pharma Face Legal and Privacy Hurdles

When AstraZeneca announced a companion diagnostics collaboration recently, their head of oncology development said the goal was to get “the right treatment, to the right patient, the first time,” a nice turn of phrase* that is becoming a chorus in the healthcare industry.

This week, giant PBM Medco purchased DNA Direct, saying “[o]ur whole thing at Medco is to get people on the right drug the first time.”  DNA Direct uses its research on 2,000 available tests to help physicians, health insurance companies and patients understand how to use personalized medicine.  This is a good move — we said last month that education is key to expanding the personalized medicine market

AstraZeneca, Medco and other providers, employers and insurers would all like to use information on individuals’ health risks in order to reduce their costs, and as the Wall Street Journal reports, they are willing to provide incentives to their employees to mitigate those risks.  However, some of these efforts conflict with barriers put in place by the Genetic Information Nondiscrimination Act (GINA), which prohibits the intentional acquisition of genetic information about applicants and employees, and imposes strict confidentiality requirements on data that is acquired. (H/T @genomicslawyer)

In addition to legal barriers (some still being erected), AMA and other advocacy groups have also reportedly expressed concern.  I agree there is risk inherent in putting the decision of what the “right drug” is in the hands of manufacturers or payors, neither of whom is neutral in the outcome.  Medco, in particular, does not seem a neutral player here (at least based on their approach to Plavix and Effient, though I invite comments if I’m misinterpreting that study).

Still, a solution is required.  As I have been saying for over a year, personalized approaches to treatment have the potential to benefit all participants in the healthcare system, as the KRAS-Erbitux story has proven.  As Procter & Gamble said when investing in Navigenics’ funding round this week, “Personalized genetic testing can have significant meaning in helping consumers focused on prevention and wellness live better, healthier lives.” 

My bottom line:  A large part of the problem here is the low level of trust from the public, which even limits governments’ ability to act.  That’s particularly unfortunate, because government is the closest thing we have to a neutral funding source for comparative effectiveness and personalized medicine research (despite also being a payor). This is a problem much bigger than just personalized medicine, but until trust is restored, valuable cost savings and health benefits will go unrealized.

Bookmark and Share

* A concept I’ve been trying to call “personalized effectiveness” — tell your friends.

Biotech Trends Update — Personalized Medicine: The Case for Diagnostics Focuses on Cost and Effectiveness

A report in FierceBiotech today distilled the views of three life science VCs on trends to watch in 2010.  Along with other worthwhile observations (and I’d encourage you to read the whole thing) was this bullet pointing out the value of personalized medicine in addressing comparative effectiveness concerns:

“Interest in molecular diagnostics is heating up. It’s one of the most attractive areas because physicians are increasingly demanding test that can tell them which treatments have the best chance of working before expensive medicines are issued. And diagnostics fit well with the healthcare reform efforts. Bloch adds that any technology that improves the efficacy of how care is delivered will be attractive to investors.”

The business case is eminently obvious.  Earlier this week AstraZeneca announced a collaboration with Dako Denmark A/S that will see Dako developing companion diagnostics for products in AstraZeneca’s oncology pipeline.  Key quotes from the announcement highlight the companies’ focus on “health care costs” and “reimbursable products”:

“Targeted treatment with personalized medicine is the future, and … is also a significant contributive factor in cutting health care costs” (Dako CEO)

“This agreement … will enable us to develop novel, reimbursable products that … predict which patients are most likely to respond to treatment, ensuring that we are giving the right treatment, to the right patient, the first time.” (AZ Head of Oncology Development)

The economic case for personalized medicine was one of this blog’s top biotech trends in 2009 and looks to continue at a strong pace through 2010.  To reach its full potential, though, the industry will have to convince policy makers and clinicians that personalized medicine can live up to its promise.

Bookmark and Share

Top Four Biotech Trends of 2009

These may not all be consensus picks (and don’t miss the IVB’s year-end deal-centric fun) but I’m sticking with these four trends as the ones that have really shaped the year that was:

  1. Follow-on Biologics. Call them what you want (we like “biosimilars”, but we’re internationalist like that), there’s no denying that biosimilars were a major force in the industry and in politics this year.  Some in the press are calling the 12-year exclusivity period a done deal, but I say don’t count your chickens ’til the fat lady sings.
  2. Comparative Effectiveness and Personalized Medicine (not a two-fer, a trend of convergence). How much of comparative effectiveness variation will turn out to be an artifact of genetic sub-populations (each with binary responses to the drugs in question)?  Nobody knows, but as money pours into both fields, the truth will set us (and drug pricing) free.
  3. Shifting IP Constituencies. What do you get when you cross generics-hungry pharma companies with innovation-hungry Asian countries?  A whole new world of collaboration that will ultimately change the face of TRIPS and pharma R&D.
  4. Electronic Medical Records. Not really biotech (if you want to be picky), but it will have a massive impact on the way physicians, patients and payors interact with each other and with drug companies over the coming years.  Plus, with billions allocated to electronic medical records in Canada and the U.S., the pace of innovation and implementation really took off.

Bookmark and Share

Preventing Bias in Comparative Effectiveness Research

Comparative effectiveness research has the potential to avoid wasteful spending and create net benefits for patients if approached properly, but it’s expensive.  Many of the large-scale comparative effectiveness studies include industry funding, and benefits managers are no strangers to the game, but giving those partners a say in study design risks introducing bias. 

An interesting example comes from today’s report that pharmacy benefits giant Medco is planning a head-to-head study of nearly-off-patent Plavix versus brand-new Effient.  The interesting tweak here is that the study will exclude people with a genetic variant (of the CYP2C19 polymorphism) who can’t metabolize Plavix.

This seems like another great example of personalized medicine informing a comparative effectiveness decision.  But, as the In Vivo Blog pointed out in an August post about Plavix and Effient, the effect of the CYP2C19 polymorphism on Effient efficacy is unknown.

So the PBM, with cost-saving incentives, is setting up a study to make payment decisions in which the efficacy of the (cheap) generic is boosted by excluding patients with the CYP2C19 polymorphism, with the validity of the comparison based on the untested assumption that there is no systematic bias to the branded product’s efficacy in the excluded population.  Am I missing something here?

The moral of the story: fund comparative effectiveness research through neutral parties and keep a careful eye on genetic and phenotypic subgroups to maximize the value of these important studies.

Bookmark and Share

Trends Update — Personalized Medicine: Montreal CRO ethica Licenses Artificial Intelligence Data Analysis Product for Stratification

B&W_DNA_sequenceethica Clinical Research acquired a worldwide exclusive license to Matrix Pharma’s  artificial intelligence (AI) data analysis platform.  Neither the form of consideration nor payment structure (up-front vs royalty etc.) was disclosed, but the deal is “valued at CAD1.25 Million.”  The companies say the AI can:

“extract interdependencies, correlations, and predictive models from complex data sets that conventional statistical tools are unable to detect.”

In other words, it’s an approach designed for stratification of clinical trial data to identify personalized subgroups for whom a drug may be particularly effective.

ethica says the product, which will be marketed as “eidyia™” (pronounced “idea”)

“has already yielded successful results in a series of clinical applications such as identifying biomarkers, optimizing the predictive power of biomarkers, identifying responder/non-responder indices, genotype-phenotype linkages, elaborating models for early diagnosis, and for classifying participants in Phase II clinical trials for the optimization of Phase III clinical trials.”

I don’t know if  “eidyia” is (or isn’t) the best platform for these tasks; but I am convinced that in many cases, the ability to stratify patient populations will be key to demonstrating the effectiveness of new drugs and winning approval and reimbursement.

Bookmark and Share

Trends Update — Comparative Effectiveness and Personalized Medicine: Is Canada Ahead of the U.S. In the Use of HER2 Testing for Personalized Breast Cancer Treatment?

B&W_DNA_sequenceFor the 20%-30% of breast cancer patients with tumors that overexpress HER2, treatment with Herceptin (an antibody drug from GenetechRoche) is highly effective.  That’s why this article in the journal Cancer is so shocking.  The authors gathered data from a variety of published sources and estimate that:

“up to 66% of eligible patients had no documentation of testing in claims records, up to 20% of patients receiving trastuzumab were not tested or had no documentation of a positive test, and 20% of HER2 results may be incorrect.”

I asked a friend, who is a genetic counsellor in Toronto, if she thought the gaps here were as bad.  She said no, and that she would be extremely surprised not to see a HER2 result in a patient’s file.

Of course it’s not valid to draw conclusions about national health care from a comparison of the Cancer study to my anecdote.  Among other reasons, the authors admit their data is flawed and dated, and my friend works at one of Canada’s top teaching hospitals.  Nevertheless, the possibility that many U.S. patients could be falling through the cracks is extremely disturbing.

If clinicians can’t adopt and maintain near-universal use of a personalized medicine approach like HER2-Herceptin with recognized and measurable benefits, the future of pharmacogenomics is in some big trouble and we will never generate truly useful comparative effectiveness data.

H/T @FiercePharma.

Bookmark and Share

Trends Update — Comparative Effectiveness: Where Data Shows No Difference, Tie Should Go To the Patient

A post by Scott Hensley on the NPR Health Blog yesterday has some good food for thought in the comparative effectiveness debate: what to do when comparative effectiveness studies show no statistically significant difference between treatments.

The post notes that insurance coverage will be a factor in these decisions, but that:

“in the end, it might be you and your gut feeling.”

In one of this blog’s prior posts, I noted that it will be hard to distinguish between treatments that show different effectiveness because of personal differences between patients and those that would show different results even if the patients were identical. 

Hensley’s post illustrates that no matter how much data we gather, there will be gray areas where doctors and patients have to make subjective calls.  I hope payors will be extremely cautious about second-guessing these decisions.  Tie goes to the patient.

Bookmark and Share

Trends Update — “Personalized Effectiveness”: Amgen Gets Prospective Data to Back KRAS-Vectibix Plan

B&W_DNA_sequenceA few weeks ago, when the FDA changed the labeling on anti-EGFR drugs, Amgen was pretty enthusiastic about “avoiding unnecessary treatments in patients [with a specific genetic marker] who are unlikely to benefit” from Vectibix.  Avoiding these patients leaves more reimbursement available for patients who would benefit from Amgen’s product.

Now Amgen has even better data to support its personalized approach to colorectal cancer treatment: their study of Vectibix as a first-line treatment tracked the KRAS genetic status of participants and showed “significantly prolonged progression-free survival” for the wt-KRAS group.

In patients with mutated KRAS, Vectibix wasn’t just “unnecessary,” it actually showed worse outcomes than the control group, meaning genetic testing of all colorectal cancer patients will be a top priority.

A second important note for companies thinking about companion diagnostics and personalized effectiveness is that the Amgen study was a prospective study that will support much more robust conclusions.  H/T @ldtimmerman.

In contrast, recall that the CMS decision not to reimburse genetic testing for Warfarin dosing specifically cited the lack of prospective data on which to base a decision.

This new data from Amgen will:

  1. Drive tumor genotyping as a standard of care; and
  2. Help make the economic case for companion diagnostics.

Share Button

Trends Update — Comparative Effectiveness and Personalized Medicine: Genetic Test Identifies Patient Subpopulation for Benefit, Avoids Wasted Money and Time for Others on Erbitux, Vectibix

B&W_DNA_sequenceThis is exactly how personalized medicine and comparative effectiveness can interact to benefit patients, pharma companies and payors:

  • data shows that patients with KRAS mutations don’t benefit from anti-EGFR antibody meds Erbitux or Vectibix;
  • the FDA approves a labeling change identifying the patients who won’t benefit;
  • payors see costs savings from eliminating pointless prescriptions;
  • patients without the KRAS mutations have added incentive to take the drugs, benefiting themselves and the pharma companies who make the drugs.

As the WSJ Health Blog says:

“Erbitux and other expensive cancer medicines have faced repeated questions about whether drugs that prolong life for short periods of time are worth the high costs… Not using Erbitux as a first-line treatment for [KRAS-variant] patients could save about $600 million a year.”

The manufacturers Bloomberg’s reporter spoke to are fully supportive:

“‘The inclusion of KRAS as a biomarker in the Erbitux labeling helps physicians to better understand the most appropriate use of the drug in the management of patients with metastatic colorectal cancer,’ said Fouad Namouni, an oncology executive for Bristol-Myers …

Physicians can eliminate Vectibix and Erbitux for colon cancer patients with the KRAS mutation and ‘redirect those patients to alternative therapies, avoiding unnecessary treatments in patients who are unlikely to benefit,’ said Sean Harper, Amgen’s chief medical officer.”

Share Button

Bailout Update: New UK Life Sciences Blueprint Aims to Promote Innovation

world_map_2002The UK has a new Life Sciences Blueprint that sets as a goal the creation of an internationally-recognized life sciences cluster.  Here’s the press release and here’s the full report (pdf). 

Innovation Pass and Changes at NICE:

The Blueprint kicks off an “Innovation Pass” program under which certain novel medicines (criteria TBD) will be available for a 3-year period without the otherwise mandatory predicate of review by the National Institute for Health and Clinical Excellence (NICE).  A further report is due from Sir Ian Kennedy next week (July 22) that aims “to identify the aspects of value and innovation which NICE should take into account in its work.”  If innovation stimulus is considered a part of the comparative effectiveness analysis, who knows how widely the door may open even after the Innovation Pass. 

Between these changes and the recent report on genomic medicine from the House of Lords Science and Technology Committee, which recommends that the purview of NICE be extended to “include a programme for evaluating the validity, utility and cost-benefits of all new genomic tests for common diseases, including pharmacogenetic tests,” big changes may be heading NICE’s way.

Also notable:

  • The Government will invest £150 million alongside private sector investment, with the aim of leveraging enough private investment to build a £1bn, 10-year Venture Capital Fund.  This is Lord Drayson’s idea that we held up at the time as a model for technology-agnostic lobbying;
  • Along with a variety of educational initiatives and programs, the Society of Biology will begin to accredit undergraduate bioscience degrees to help ensure that graduates leave with the core skills and competencies required by employers;
  • A reassessment of the UK’s various R&D tax incentive programs (A little tax joke for you there as a prize for making it this far down the post.);
  • An £18 million program for regenerative medicine R&D; and of course
  • A marketing initiative to make sure everyone knows all the great stuff they’re doing.

Share Button

Trends Update — Comparative Effectiveness and Personalized Medicine: Study by CAMH in Toronto will Integrate Genetics, PET Brain Imaging and Pharmacology

CAMH logoToronto’s Centre for Addiction and Mental Health (CAMH) will use a $2.8 million grant from the Canada Foundation for Innovation, along with expected Ontario matching funds, for their ambitious neuroIMAGENE initiative.  The neuroIMAGENE program aims

“to combine the power of genetics and sophisticated brain imaging to personalize treatment … for common psychiatric conditions like major depression, bipolar disorder, schizophrenia, as well as addictions.”

Specifically, CAMH will compare DNA characteristics across 18 different psychiatric conditions and integrate that data with PET scans showing individuals’ neurochemical changes induced by drug therapies. The idea is to create tools to identify the medication that will work best for each individual’s brain chemistry and genotype,

“helping to avoid trial-and-error prescribing, treatment failure, relapse, and serious side effects.”

Bookmark and Share

Post-Vacation Brain Dump: VC and Business News

Some suggestions for things to do:

And some suggestions for things not to do:

Trends Update — Comparative Effectiveness and Personalized Medicine: Patient-Centered Outcomes Research Act of 2009 Increases Personalized Medicine Focus

story on GenomeWeb yesterday takes a close look at the Baucus-Conrad Comparative Effectiveness Bill and notes that the influence of personalized medicine that we’ve flagged as a trend in 2009 has shown up in this year’s verison of the bill as

language specifying research approaches such as “molecularly informed trials” and “genetic and molecular sub-typing.”

This year’s version of the bill also

includes more emphasis on involvement with the diagnostics community and calls for an expert in genomics to serve on a methodology committee.

In addition to a focus on personalized medicine, the changes to Baucus’ bill incorporate another idea from Sen. Kyl’s amendment in April – adding some hurdles before CER results could be used (by CMS) for coverage decisions. 

Even so, the bill continues to meet procedural impediments and substantive objections, resisting Baucus’ efforts to re-brand the concept.

Bookmark and Share

Trends Update — Comparative Effectiveness: To Head-to-Head or Not To Head-to-Head?

Goats_butting_heads_in_GermanyOne of the challenges of comparative effectiveness implementation is figuring out when to look for comparative data. Two developments this week shed light on current comparative effectiveness thinking by regulators and pharma:

  1. Vanda’s new antipsychotic, iloperidone (Fanapt) was approved by the FDA last week without any head-to-head comparison against competitors (Zyprexa, Risperidol).  This approval indicates that so far, as the In Vivo Blog points out, the FDA is sticking to the view expresed by Bob Temple last year to the RPM Report that “[i]n most settings, especially for symptomatic treatments, we [the FDA] do not get or ask for comparative data and are perfectly willing to approve a drug that is shown effective.” (emphasis added).
  2. Nevertheless, reports today indicate that AstraZeneca decided to find out at Phase III whether its heart drug Brilinta is more effective than Plavix.  AZ conducted one of the largest head-to-head studies ever undertaken before regulatory approval.  FierceBiotech says that “offers a clear indication of the path developers will be more willing to take as they angle for market share among increasingly cost-conscious payers.”

Would Vanda have had an easier time if it had done a head-to-head trial? Could they have sustained the company long enough to find out? Maybe the only moral of these two trials is to look for comparative data as soon as you can afford to.

Bookmark and Share

About the Delayed Sebelius Confirmation

Contrary to expectations, Secretary of HHS-designate Kathleen Sebelius did not receive her Senate confirmation before the Congressional recess on April 3, due to the objection of one Senator.  Is Sebelius using the break to make greater assurances to Republican lawmakers before her vote is taken up again during the week of April 20? 

In her confirmation hearing, Sebelius addressed Republican concerns about comparative effectiveness research leading to “rationed care.”  Mike Enzi (R-Wyoming), top Republican on the Senate Health, Education, Labor and Pensions Committee questioned whether or not Sebelius would support a budget reconciliation measure opposed by Republicans to expedite Congressional approval of health reform. Republicans have also raised strong opposition to the idea of a government-run insurance plan to compete with private insurers.  The recess has provided more time for Republicans to press Sebelius on these matters and dangle them in front of her Senate confirmation.  Let’s see if this leads to more detailed commitments on comparative effectiveness and health reform!

Bookmark and Share

Wednesday Brain Dump: Things that Might Surprise You Edition

Things that surprised me this week:

Bookmark and Share

Trends in 2009: Comparative Effectiveness Meets Personalized Medicine in the Senate

Yesterday Senator Kyl (R-AZ) introduced a “comparative effectiveness amendment” (SA 793) to the budget which would have:

  1. required that legislation resulting from the health care reserve fund not use data obtained from comparative effectiveness research to deny coverage under Federal health care programs; and
  2. ensured that comparative effectiveness research accounts for advancements in genomics and personalized medicine, the unique needs of health disparity populations, and differences in the treatment response and the treatment preferences of patients.

As I noted in our earlier Trends post, I believe the comparative effectiveness advocates in the Obama administration are perfectly on board with point (2), a point I made again with further examples when the U.S. Stimulus allocated $1.1 billion to comparative effectiveness research.  It’s point (1) that most likely caused the amendment to be rejected, 44-54. 

Bottom line:  until the research is done, we won’t know how much of what appears to be “comparative effectiveness” is actually accounted for by “personalized medicine” (i.e., individual, genetically-based response variation to the subject medication/treatment), as compared to different “effectiveness” (i.e., response to a medication/treatment in a (relatively) homogeneous population). 

My guess is that the vast majority of apparently differential effectiveness will boil down to underlying genetic differences in patient subpopulations. 

But ultimately, there may be some treatments that are genuinely less effective than others in comparable populations.  As a patient and as a taxpayer, I’d like to know what those are and avoid taking or paying for them.

Bookmark and Share

Wednesday Brain Dump: Around the World Edition

Comparative Effectiveness Stimulus Stimulates Reactions

The $1.1 billion in the stimulus bill for comparative effectiveness research has, not surprisingly, generated a good deal of public attention.  Friday’s Washington Post and the front page of today’s New York Times both have stories covering the political jockeying.

Although both pieces focus on potential problems from the lack of individualization, either from libertarian or advocacy perspectives, neither has picked up our strain that personalized medicine, also favored by the Obama administration, will change the shape of the entire comparative effectiveness debate.
More on a recent example after the jump…

U.S. Stimulus Compromise (Updated)

Initial reporting (NYT, WSJ) on the bill coming out of the House-Senate conference committee this evening indicates that the $789 billion package will include most of the Bio-related provisions:

Update 10am: More details from ScienceInsider based on Pelosi’s fact sheet:

  • The Department of Energy’s Office of Science, which supports US physical science, will receive $1.6 billion.
  • $400 million will be provided to fund a new mini agency within DOE called the Advanced Research Project Agency-Energy. Obama’s energy secretary, Steve Chu, is a fan. Now congress has bestowed their blessing — and big money –on his dream energy program.
  • NASA will get $1 billion including $400 million for climate change research.
  • The National Institute of Standards and Technology will receive $580 million.
  • The National Science Foundation would receive the full $3 billion increase that the House had passed last month. That’s a 50% boost to its $6 billion budget. The breakdown of that number is not clear, but the House version contained $2 billion more for research grants; $900 million for three infrastructure programs, including a revived $200 million extramural facilities competition; and $100 million for two education programs.

Update 5pm:

  • The $3 billion in NSF funding breaks down as follows: $2.5 billion for research (peer-reviewed proposals); $100 million for the university research instrumentation program; and $400 million for national labs.
  • The NIST money is for infrastructure: $220 million for lab equipment and $360 million for facilities.
  • USDA gets $850 million for infrastructure.
  • NOAA gets $600 million for facilities and equipment.

Bookmark and Share

Wednesday Brain Dump: February 4, 2009

Some good news on the gene therapy front in adenosine deaminase-deficient SCID patients and in rheumatoid arthritis.

But mostly bad news on the job front at GSK, AstraZenecaAbbott,  GenVecPatheon, and others.

Other good news on the approvals front for Parusgel (despite process concerns), KapidexLamictalGelnique and Taxus Liberte.

Really small news: Nanomaterials may be heading for increased regulation in Canada, with a mandatory reporting program reportedly pending and a new guide from IRSST in Quebec (pdf) (although the IRSST guide doesn’t mention bio-materials).

Just NICE news: Comparative Effectiveness may be headed for some changes in the UK, where NICE is working on a review.

Positively Biblical news: The lion lies down with the lamb (or something equally unlikely)

Bookmark and Share

Personalized Medicine: Local to Global

Two local developments in personalized medicine in Canada, one at the forefront of global efforts, one making recommendations on how to play catch-up:

Read more of this post

Trends in 2009: Random Gloating

Remember way back Monday when we identified Comparative Effectiveness as a trend to watch in 2009? Well, here it is, showing up as part of the bailout bill. More on the bailout bill’s Bio provisions to come. AAAS is running continually updated coverage of the bill and its R&D provisions here.

Trends in 2009: Comparative Effectiveness and Personalized Medicine

Two potentially conflicting trends may see a dramatically increased profile in 2009: Government Bailouts and Free-Market Capitalism Comparative Effectiveness and Personalized Medicine. Both have been highlighted by the incoming Obama administration.  Details and analysis after the jump…

Follow

Get every new post delivered to your Inbox.

Join 130 other followers