Are some medical treatments too expensive to justify?

Monty Burnz

up for anything wrote: »

That's a tough one but yes, I'd see ten A&Es closed down to extend the life of one of my children if they were dying but could be given extra time and I'd sell my house (and my extended life children) to keep my mother alive.

Fortunately for all the other people's loved ones your choice would kill, you're not in a position to close our A&Es...

up for anything

I was asked a question and answered it! I'm sad for all the people that might die because of my decision but that's life or rather death.

Noreen1

Monty Burnz wrote: »

Fortunately for all the other people's loved ones your choice would kill, you're not in a position to close our A&Es...

Why do you assume that closing A&Es is the only option in obtaining funds?
Don't you believe that any other cost savings can be achieved?

Chuck Stone

Although modern pharmaceuticals are supposed to represent the practical payoff of basic research, the R&D to discover a promising new compound now costs about 100 times more (in inflation-adjusted dollars) than it did in 1950. (It also takes nearly three times as long.) This trend shows no sign of letting up: Industry forecasts suggest that once failures are taken into account, the average cost per approved molecule will top $3.8 billion by 2015. What’s worse, even these “successful” compounds don’t seem to be worth the investment. According to one internal estimate, approximately 85 percent of new prescription drugs approved by European regulators provide little to no new benefit. We are witnessing Moore’s law in reverse.

http://www.wired.com/magazine/2011/12/ff_causation/all/1

Maybe the estimate that '85 percent of new prescription drugs approved by European regulators provide little to no new benefit' is the reason the pharmaceutical companies spend so much on sales and advertising.

yore

Chuck Stone wrote: »

Although modern pharmaceuticals are supposed to represent the practical payoff of basic research, the R&D to discover a promising new compound now costs about 100 times more (in inflation-adjusted dollars) than it did in 1950. (It also takes nearly three times as long.) This trend shows no sign of letting up: Industry forecasts suggest that once failures are taken into account, the average cost per approved molecule will top $3.8 billion by 2015. What’s worse, even these “successful” compounds don’t seem to be worth the investment. According to one internal estimate, approximately 85 percent of new prescription drugs approved by European regulators provide little to no new benefit. We are witnessing Moore’s law in reverse.

http://www.wired.com/magazine/2011/1...ausation/all/1

Maybe the estimate that '85 percent of new prescription drugs approved by European regulators provide little to no new benefit' is the reason the pharmaceutical companies spend so much on sales and advertising.

Yeah.....I'd reckon the lack of cheap victims clinicial trial subjects from our now over-zealous orphanages have pushed up those costs. God be with the days when the companies could simply walk into the orphanage, inject all the kids with various cocktails and then come back in a week and see which ones didn't have adverse reactions.

Monty Burnz

Noreen1 wrote: »

Why do you assume that closing A&Es is the only option in obtaining funds?
Don't you believe that any other cost savings can be achieved?

I'm replying to a statement made by another poster that they would happily kill others to save their loved one, or extend their life for a bit.

Daveysil15

Yes they are. Scientists in Canada found a cure for cancer but they won't produce it because the pharmaceutical companies won't make a profit on it.

Min

We can bail out sick banks but not sick people?

Teyla Emmagan

Daveysil15 wrote: »

Yes they are. Scientists in Canada found a cure for cancer but they won't produce it because the pharmaceutical companies won't make a profit on it.

Proof?

The state must make realistic decisions as to how it spends our limited funds. 85k to potentially extend the life of one person is just not justifiable. If you want the treatment, raise the funds yourself. Harsh, but fair, IMO.

Monty Burnz

Daveysil15 wrote: »

Yes they are. Scientists in Canada found a cure for cancer but they won't produce it because the pharmaceutical companies won't make a profit on it.

Conspiracy theories board, tbh. Total nonsense.

Monty Burnz

Min wrote: »

We can bail out sick banks but not sick people?

That's a child's level of understanding of government to be fair.

laoch na mona

its disgusting the way companies profit off life saving drugs

Monty Burnz

laoch na mona wrote: »

its disgusting the way companies profit off life saving drugs

Why would companies make any drugs at all if they made no profit? Again, people have the brains of two-year olds.

I suppose you spend all your time helping disabled people for free? Oh no wait, you don't, you're a huge hypocrite.

bleg

The answer is: it depends.

There is a whole science dealing with this called pharmacoeconomics.

http://en.wikipedia.org/wiki/Pharmacoeconomics


Drugs cost a lot of money . Some substances can cost millions or hundreds of millions per gram. These are the most valuable things by weight in the world ever.

Think about the 1000s of people employed in the R&D of these drugs. Add in regulatory, quality, production, administrative staff, light, heat, electricity, distribution (in a cold chain), education, marketing and you've got a lot of mouths to feed. Drugs aren't becoming cheaper and simpler, they're becoming unbelievably complex and expensive and sometimes what they can achieve is amazing.

Look at the amount of mergers and acquisitions and divestments that is going on in the pharma industry at the moment. This is an industry facing a crisis, not a cash cow.

laoch na mona

Monty Burnz wrote: »

Why would companies make any drugs at all if they made no profit? Again, people have the brains of two-year olds.

I suppose you spend all your time helping disabled people for free? Oh no wait, you don't, you're a huge hypocrite.

surely people should want to do whatever they can to help people

and by the way I do lots of charity work so don't call me a hypocrite :mad:

Monty Burnz

laoch na mona wrote: »

surely people should want to do whatever they can to help people

So people should work for free all the time, because it's 'helpful'?

laoch na mona wrote: »

and by the way I do lots of charity work so don't call me a hypocrite :mad:

Do you exclusively do charity work for free? Why bother with an education even - you should do whatever you can to help! Seeing as you want pharma companies to work for nothing...

Let me guess: ULA supporter?

laoch na mona

Monty Burnz wrote: »

So people should work for free all the time, because it's 'helpful'?

Do you exclusively do charity work for free? Why bother with an education even - you should do whatever you can to help! Seeing as you want pharma companies to work for nothing...

Let me guess: ULA supporter?

I'm not going to debate with what I can only assume from the nature of your arguments is a typical keyboard warrior
slán:P

bleg

laoch na mona wrote: »

surely people should want to do whatever they can to help people

I do, but until I can eat air I need to get paid.

Monty Burnz

laoch na mona wrote: »

I'm not going to debate with what I can only assume from the nature of your arguments is a typical keyboard warrior
slán:P

The nature of my arguments being 'correct', I suppose. Slán agat.

PoleStar

Stiffler2 wrote: »

rewind to 100 years ago when this technology did not exist and cancer also did not exist.

I think you have missed one important fact: age is the single most significant risk factor for most cancers.

100 years ago, people didn't die of cancer because the life expectancy was less. In addition, 100 years ago, when people were ill and dying, there was no diagnosis, so many may have died of cancer but without a diagnosis. Nowadays when someone is ill, they get investigated and in most (not all) cases a concrete diagnosis is made. I am sure you would agree that is quite different to 1912.

lividduck

smash wrote: »

The sad thing is that there is no possible way it should cost anywhere near €85,000.

I agree, Pharmacuetical companies should spend tens of millions researching and developing drugs and then just give them away, i'm sure their shareholders would be fully in favour! Doh

bnt

I should also mention the effect that the USA situation has. The costs of medical treatment are passed on to the patients via insurance companies and HMOs, with no government in the loop to control prices. The nightmare for drug companies operating in the USA is the grey import market, from Canada & Mexico in particular, so they are not very flexible on price anywhere in the world.

For example, the new multiple sclerosis therapy Gilenya (fingolimod) is fully approved in Ireland, and the govt. review board called it "cost-effective", but it's still not being prescribed, solely due to cost. MS Ireland are, to put it politely, unhappy about this. But Novartis aren't going to be allowing any major reductions in price, since they don't have much competition, and they don't want any country's price to undercut any other's - especially not the USA. If Gilenya cost half as much here in Ireland, someone would make a profit selling it on to the USA market.

bnt

re Cancer: a few minutes on Wikipedia would have told you that cancer was a recognised condition, and given its name, over 2,000 years ago. There's nothing "new" about it at all.

Cancer is definitely increasing in incidence as people get older, but it's not only an old person's disease. Just yesterday, the news was reporting the death of one of the Beastie Boys from cancer at the age of 47. I didn't watch "Big Brother", but I couldn't avoid the headlines about Jade Goody, and her death from cervical cancer at the age of 27. The picture is statistical, and we're playing the odds, not crossing some imaginary line, as we age.

DonQuay1

smash wrote: »

The sad thing is that there is no possible way it should cost anywhere near €85,000.

Pardon me for asking a silly Q ... but how do you know that it shouldn't cost anywhere near that??

When 'Interferon' - for cancer - was first on the market it cost the budget of a county council to provide it!! until economies of scale brought the price down to relatively cheap levels.
If you buy an Ipod ... it costs a fortune for the lucky first few buyers .... then when it takes off and factories are retooled over time ... price comes down.
Economics - it puts food in the shops and doctors in hospitals.

Usual ridiculous comment without any thought behind it.

Chuck Stone

Monty Burnz wrote: »

Why would companies make any drugs at all if they made no profit?

Here's another valid question.

How do we ensure the profit motive doesn't supersede the goal of improving the health of consumers?

Dr. Ben Goldacre talks about how the pharmaceutical industry distorts the evidence it gives to doctors and patients.

Go to 7:28

This game is rigged, man. We like the little bitches on a chessboard.

The Wire.

bleg

Chuck Stone wrote: »

How do we ensure the profit motive doesn't supersede the goal of improving the health of consumers?

it doesn't. Most of the people in the healthcare industry are there to make the world a better place, from researcher to doctor, you get a profound feeling that what you're doing is actually helping them. Running these companies like a business ensures efficiency and allows people to earn money whilst making the world healthier.

It would be great if all pharma companies were run as a trust like Leo pharma but the majority of the major companies were founded as businesses by pharmacists and evolved and flourished in the corporate environment.

Pushtrak

Chuck Stone wrote: »

Maybe the estimate that '85 percent of new prescription drugs approved by European regulators provide little to no new benefit' is the reason the pharmaceutical companies spend so much on sales and advertising.

I'd like to know more about that particular estimate.

According to one internal estimate, approximately 85 percent of new prescription drugs approved by European regulators provide little to no new benefit. We are witnessing Moore’s law in reverse.

One internal estimate? What was this estimate based on? Was it after a thorough study or just someone making an educated guess? An uneducated guess?

Monty Burnz

Chuck Stone wrote: »

Here's another valid question.

How do we ensure the profit motive doesn't supersede the goal of improving the health of consumers?

Whose goal is that though? Surely that's the goal of the medical profession, not the pharma companies.

Chuck Stone

Pushtrak wrote: »

I'd like to know more about that particular estimate.

I had a quick look and couldn't get the source. Will try a bit more.

Monty Burnz wrote: »

Whose goal is that though? Surely that's the goal of the medical profession, not the pharma companies.

Are they not both in the medical profession/business?. I guess doctors have to trust the pharma corporations to do the right thing and if Goldacre is to be taken seriously they are manipulating the information released about the efficacy of drugs.

Monty Burnz

Chuck Stone wrote: »

Are they not both in the medical profession/business?. I guess doctors have to trust the pharma corporations to do the right thing and if Goldacre is to be taken seriously they are manipulating the information released about the efficacy of drugs.

I've no doubt some skullduggery goes on in the pharma industry, but that's what regulation is for. I've a lot of time for Goldacre.

Chuck Stone

Monty Burnz wrote: »

I've no doubt some skullduggery goes on in the pharma industry, but that's what regulation is for. I've a lot of time for Goldacre.

I would like to see us (humans) experiment with a different model where there is no medical patent (monopoly of production) as a major driving force of innovation. I think we can all agree that monopolies are almost never good for consumers.

I've read about suggestions that a minuscule tax on all medicine pooled in a prize fund would accumulate billions of dollars pretty quickly.

With billions of dollars in prize funds the profit motive would remain but patent-free medicines could be produced competitively by whoever wanted which would drive down the costs for consumers.

Monty Burnz

Chuck Stone wrote: »

I would like to see us (humans) experiment with a different model where there is no medical patent (monopoly of production) as a major driving force of innovation. I think we can all agree that monopolies are almost never good for consumers.

I've read about suggestions that a minuscule tax on all medicine pooled in a prize fund would accumulate billions of dollars pretty quickly.

With billions of dollars in prize funds the profit motive would remain but patent-free medicines could be produced competitively by whoever wanted which would drive down the costs for consumers.

There's a lot of research into innovation if you are interested. One of the main reasons why technological development exploded in the 20th century was the implementation of patent law.

Prize funds would make investment in drug research a lottery - and investors don't like lotteries, otherwise they'd just buy lottery tickets and cut out the need to manage a complex business.

Chuck Stone

Monty Burnz wrote: »

One of the main reasons why technological development exploded in the 20th century was the implementation of patent law.

I'm not entirely convinced that patents are the main driver of technological development. Some people believe patents stifle innovation.

Here's one famous case.

Here's another.

Here's one where the 'inventor' needs a boot up the hole.

Prize funds would make investment in drug research a lottery - and investors don't like lotteries, otherwise they'd just buy lottery tickets and cut out the need to manage a complex business.

I wouldn't describe it as a lottery. More like a game of poker. You can be good at poker but there's no such thing as being good at the lotto.

c_man

Chuck Stone wrote: »

there's no such thing as being good at the lotto.

http://simpsonswiki.net/w/images/7/74/Hot_Lotto_Picks_Weekly.png

Monty Burnz

Chuck Stone wrote: »

I'm not entirely convinced that patents are the main driver of technological development. Some people believe patents stifle innovation.

You may not be convinced, but both history and researchers in the field of innovation are.

The patent system certainly isn't perfect, but like democracy, it's the best of a lot of bad solutions.

Red Crow

Chuck Stone wrote: »

I'm not entirely convinced that patents are the main driver of technological development. Some people believe patents stifle innovation.

Here's one famous case.

Here's another.

Here's one where the 'inventor' needs a boot up the hole.



I wouldn't describe it as a lottery. More like a game of poker. You can be good at poker but there's no such thing as being good at the lotto.

I'd absolutely agree 100% with you. Monopolies on drugs are the biggest problem.

Monty Burnz

Eli Manning wrote: »

I'd absolutely agree 100% with you. Monopolies on drugs are the biggest problem.

But without the monopoly (for a quite limited time) how do companies recoup the money spent on R&D?

Chuck Stone

Monty Burnz wrote: »

You may not be convinced, but both history and researchers in the field of innovation are.

The patent system certainly isn't perfect, but like democracy, it's the best of a lot of bad solutions.

If you take a look at figures #1 & #2 in this PDF (page 2) released by the FDA it appears that in spite of increasing money spent on R&D the amount of new products being approved is in steady decline.

I can't see how researchers would be completely convinced that innovation is doing fine in the face of evidence to the contrary.

ismisekatie

lazygal wrote: »

There's a new cancer drug costing 85,000 per patient being approved. http://www.irishtimes.com/newspaper/ireland/2012/0504/1224315592242.html It doesn't cure the disease apparently, but does offer some help. There's also cases of people being sent to other countries to avail of expensive treatments not available here and the HSE funding it. Is 85,000 for treatment for one person too expensive? Or is life something for which money shouldn't be a factor?

was that the lady on the late late last night?

Monty Burnz

Chuck Stone wrote: »

If you take a look at figures #1 & #2 in this PDF (page 2) released by the FDA it appears that in spite of increasing money spent on R&D the amount of new products being approved is in steady decline.

I can't see how researchers would be completely convinced that innovation is doing fine in the face of evidence to the contrary.

There are plenty of possible reasons why the numbers of new products being approved is declining - here are a couple off the top of my head:

1. Stricter standards by the FDA
2. Less R&D investment by drugs companies
3. Fewer new compounds available to research
4. Current pharma technology reaching limits of what it can do
5. Consolidation in pharma industry meaning fewer projects make it to trials

Those are suggestions from someone who knows little about the industry. I imagine an insider would come up with more/more accurate possibilities.

I'd also point out that falling numbers of approved drugs suggests that the incentives for pharma companies are too low, not too high.

Chuck Stone

Monty Burnz wrote: »

There are plenty of possible reasons why the numbers of new products being approved is declining - here are a couple off the top of my head:

1. Stricter standards by the FDA
2. Less R&D investment by drugs companies
3. Fewer new compounds available to research
4. Current pharma technology reaching limits of what it can do
5. Consolidation in pharma industry meaning fewer projects make it to trials

Those are suggestions from someone who knows little about the industry. I imagine an insider would come up with more/more accurate possibilities.

I'd also point out that falling numbers of approved drugs suggests that the incentives for pharma companies are too low, not too high.

Yeah, well...

I'm out.

Daveysil15

Teyla Emmagan wrote: »

Proof?

The state must make realistic decisions as to how it spends our limited funds. 85k to potentially extend the life of one person is just not justifiable. If you want the treatment, raise the funds yourself. Harsh, but fair, IMO.

It was in the news a while ago. There have probably been several cures made for cancer over the years, but there are powerful people concerned with population control. Look at the recent scandal over the Swine Flu vaccines. There's a lot going on that we don't know about.

28064212

Daveysil15 wrote: »

It was in the news a while ago. There have probably been several cures made for cancer over the years, but there are powerful people concerned with population control. Look at the recent scandal over the Swine Flu vaccines. There's a lot going on that we don't know about.

So it was in the news, but it's still being covered up? That's some pretty crap covering up

Pushtrak

Chuck Stone wrote: »

If you take a look at figures #1 & #2 in this PDF (page 2) released by the FDA it appears that in spite of increasing money spent on R&D the amount of new products being approved is in steady decline.

I can't see how researchers would be completely convinced that innovation is doing fine in the face of evidence to the contrary.

That was a study from 2004. Studies are routinely done to see the state of affairs in a given field, and what ought to be done to correct it. So, that being 2004, has anything happened?
http://www.acrohealth.org/fda-testimony-on-clinical-trials-modernization.html

The Association of Clinical Research Organizations (ACRO) represents the world's leading clinical research organizations (CROs). Our member companies provide a wide range of specialized services across the entire spectrum of development for new drugs, biologics and medical devices, from discovery, pre-clinical, proof of concept and first-in-man studies through post-approval and pharmacovigilance research. With more than 75,000 employees engaged in research activities around the world, ACRO advances clinical outsourcing to improve the quality, efficiency and safety of biomedical research. Each year, ACRO member companies conduct more than 11,000 clinical trials involving nearly two million research participants in 115 countries. On average, each of our member companies works with more than 500 research sponsors annually, and we have a broad and unique understanding of the roles, responsibilities and behavior of all the stakeholders – sponsors, investigators, IRBs, research volunteers and ancillary providers of all types – that are part of the research enterprise.

In March 2004 the FDA released the report titled, “Challenge and Opportunity on the Critical Path to New Medical Products.” The white paper posited the need for “new tools to get fundamentally better answers about how the safety and effectiveness of new products can be demonstrated, in faster time frames, with more certainty, and at lower costs.” The report called for a joint effort by industry, academia and the FDA to identify key problems and develop targeted solutions along the “critical path,” noting that most of the recent cost increases in the process of getting to new medical products are in the ‘development’ phase – from pre-clinical discovery work through the end of human clinical trial testing, between discovery and launch, in other words. The Critical Path Initiative was intended to be a cross-Center effort that would address scientific and regulatory barriers or hurdles to new biomedical product development; as the white paper stated, “A new product development toolkit… is urgently needed to improve predictability and efficiency along the critical path.”

Having solicited feedback from industry and other stakeholders, in March of 2006 the FDA announced a “Critical Path Opportunities List” that laid out 76 opportunities across six broad topic areas[1] for innovation in the evaluation, testing and manufacture of new products. On the topic of today’s hearing, “streamlining clinical trials”, the Opportunities List enumerated a dozen potential innovations relating to encouraging new trial design, developing new trial endpoints, standardizing data handling and reporting, and the like.

To advance the Critical Path Initiative, the FDA has supported and continues to support, either financially (to the tune of several million dollars each year) or with personnel or both, a number of public-private collaborations, including the Critical Path (C-Path) Institute, the Clinical Data Interchange Standards Consortium (CDISC) and the Clinical Trials Transformation Initiative (CTTI) and there has been some real progress in development of the “tool box” that supports the biomedical product development paradigm. For instance, CDISC has more than a dozen data standards and innovations developed, tested and rolled out for use, and C-Path has submitted to the FDA for review more than 60 potential biomarkers, disease models and patient-reported outcomes. Similarly, the NIH Biomarkers Consortium, in which several of our member companies participate, has undertaken data sharing and data mining projects; one recent project, for instance, analyzed aggregated placebo data from large, industry-funded trials to determine whether the protein adiponectin is useful as a predictive biomarker of glycemic control.

From Tool Development To Transformation

So, there has been some progress in developing basic buildings blocks for the development enterprise. But actual product development remains costly, slow and unproductive. As was true in 2004, it still takes over a billion dollars across a timeline that can take up to 15 years to bring a new biomedical product to market.

In terms of the Critical Path, the next question might be posed as: How do we move from (investigator-initiated) basic research and tool development to translational research? (In another context, we might say, When is this ready to test in humans?) Given its name, one might look to the Clinical Trials Transformation Initiative (CTTI) for direction here. Unfortunately, in our view CTTI has yet to consider undertaking any project that might qualify as ‘transformative’. Instead, its approach is one of doing “research on research” which may well produce white papers and other publications, recommendations, meetings and educational initiatives but is unlikely, we believe, to facilitate any significant change to current practices, let alone transformation of the enterprise. Surveys of current practices in regard to monitoring, site initiation, SAE reporting and the like may be interesting but, even if augmented by critical assessments and recommendations for future practice, are very unlikely to actually change those practices. Simply, understanding that certain practices are wasteful or ill-advised will not, on its own, change those practices. (As a clinical psychologist I would be happy to point you to a vast literature that supports the finding that merely ‘understanding’ dysfunctional behavior very often does not lead to changing such behavior.) To illustrate by example: as long as Federal or industry research grants flow to institutions where completion of IRB review averages over two years, without any negative consequences, you can expect that the timeline for IRB review at those institutions will not improve more than marginally.

To quote an epigraph from Goethe that was used to open the IOM’s recent report on transforming the clinical trials enterprise: “Knowing is not enough; we must apply. Willing is not enough; we must do.” In regard to CTTI per se, ACRO suggests that the focus of this agency-supported public-private collaborative should be on innovation and on generating data that would support innovation. What is needed, we believe, is an agenda of studies that: 1) compare current and innovative approaches; and/or 2) pilot or demonstration projects agreed to by the FDA (and other regulators, as may be necessary,) that have the potential to demonstrate actual savings in development time and/or cost for a given product.

A few topics that might be considered for comparison studies or in vivo demonstration projects that might be considered include, (this is a non-exhaustive list, of course):

Incorporating more modern regulatory science into clinical trial design so that a sponsor can generate the data necessary for product approval with fewer subjects;
Simplifying the informed consent process so that the information provided to potential participants is more intelligible and meaningful, and thus encourages truly informed clinical trial participation and eases the burden on research sites and investigators – via projects that may compare approaches to IC or demonstrate the utility of technology-facilitated or other improvement to the IC process without comparison;
Testing, across therapeutic areas, the use of a single, central IRB on a country level to improve the quality of independent review, to make more consistent operational practices, and thus to accelerate trial initiation – including assessment of the impact of the use of a central IRB on predetermined metrics, such as the quality of Informed Consent, the occurrence of inclusion/exclusion errors, etc.;
Approaches to risk-based oversight of sites/investigators by sponsors, including statistical and other sampling, remote and other technology-enabled monitoring;
Expanding regulatory cooperation and information sharing for multi-regional clinical trials to limit or eliminate duplicative audits, examinations and reviews.


All such studies or projects should be assessed against the question of whether the findings to be generated have the potential to “transform” the current clinical trial paradigm – to reduce costs or development time or increase the number of potentially approvable products – and, we believe, undertaken with clear deliverables and timelines. We believe, also, that the Critical Path Initiative should not be yoked to any political agenda, like ‘saving’ the current U.S. research enterprise – its goal is to facilitate a new model that can produce more products in less time at less cost, and it should be focused on the needs of patients, not companies, regulators, academic institutions, CROs, IRBs, or anyone else.

What the FDA Can Do

Eight years into the Critical Path Initiative, why have we yet to see an actual test of scientific, operational and/or regulatory oversight innovation applied to the development, review and approval of a product?

Since 2004, in open forums senior FDA staff have consistently indicated openness to demonstration and pilot projects, proof of concept initiatives, and the like intended to advance innovation in product development and the agency’s capacity to improve its approach to regulatory science. We note that the FDA has made successful use of an Accelerated Approval process for new drugs but lacks a similarly focused “Accelerated Development” mechanism to shorten the period between discovery and New Drug Application (NDA).

Because there is no formalized process whereby industry can bring proposals for innovative drug development projects to the FDA, nor any possibility of securing enforceable agreements with the agency concerning the approval, execution, oversight and evaluation of experimental approaches, industry has remained reluctant to pursue innovative approaches based only on the informal assurances offered by FDA officials – and actual testing of new approaches to the science of drug development has been stymied. Simply, no research sponsor or CRO partner wants to be “the first one out there” (for example, by undertaking a clinical trial project with significantly reduced, risk-adapted monitoring) lest some unforeseen event(s) take place and the company be subject to untoward consequences during a later agency inspection or review. In effect, the devil we know may be unnecessarily expensive and time-consuming, but the devil we don’t know could be even worse; a cancelled trial, a rejected product application, etc.

ACRO suggests two actions that would help to re-focus the Critical Path Initiative:

Engage a consulting firm or other 3rd party to review and prepare a report to the agency on the following question –

How can the FDA more aggressively support innovation by regulated industry and embrace scientific/regulatory innovation in-house? Specifically, assess the status of the Critical Path Initiative, including agency activities and contracts or other support provided to other parties. In regard to agency-supported public-private collaborations: describe how the collaboration addresses topics numbered 1 through 76 outlined on the Critical Path Opportunities List; assess the governance and productivity of each funded collaboration, including the number of ‘products’ delivered, demonstration or pilot projects undertaken, the potential for each activity undertaken to impact (“transform”) the cost, speed or efficiency of the current drug development paradigm; describe how the agency-supported collaborative establishes project priorities and timelines, produces deliverables in a timely fashion, and is evaluated by the FDA at least annually.

Appoint (or seek Congressional approval to appoint) a Chief Innovation Officer for the FDA who shall be responsible for overseeing the Critical Path Initiative and will –

a) identify promising new scientific and regulatory approaches to ensure the rapid development, testing, and review of new drugs and devices, which may include the validation and qualification of biomarkers, the adoption of novel models or methodologies to enhance clinical trial design, clinical trial conduct, clinical data evaluation, or predictive toxicology, and the coordination and optimization of efficient review processes for drugs, and devices;

b) solicit from industry and other stakeholders specific proposals for innovative approaches to biomedical product development in the form of demonstration and pilot projects; approve research projects that test and/or compare current and innovative approaches to clinical trial testing of products; permit temporary trial-specific alterations to FDA regulations and requirements; and facilitate such other initiatives as may demonstrate the potential to reduce the cost or time required to develop and test new biomedical products;

c) identify personnel and processes whereby industry and other stakeholders may pursue initiatives such as those identified in b) under formal agreements with the agency which may modify existing regulatory requirements, including agency audits, inspections, data requirements and such other modifications as may be advisable;

d) ensure that agreements entered into under c) are recognized throughout the agency, both vertically and horizontally;

e) provide to the Commissioner and the Congress an annual report regarding specific, quantifiable progress toward the objectives of the Critical Path Initiative – a faster, less costly, more productive biomedical product development paradigm – with supporting metrics.

So, clearly there is still work to be done in the area. There has been some progress, too. It isn't as if the situation is stagnant.

Capt'n Midnight

BBC recent news

http://www.bbc.co.uk/news/health-17925581 Aspirin is as 'good as warfarin' for most heart failure patients

Obviously it's not best for the all as individual circumstances vary, but Aspirin is a lot cheaper, money that could be used elsewhere.


and this is WTF
Novartis suing the NHS for using cheaper drugs

http://www.bbc.co.uk/news/health-17956425 Using Avastin for eye condition wet AMD 'could save NHS £84m'

NHS-funded research says both Lucentis and Avastin have a similar effect in preventing loss of sight when used for wet age-related macular degeneration.

Lucentis costs about £700 an injection, and Avastin £60 - but Avastin is not officially approved for eye conditions.

Novartis, which markets Lucentis in the UK, is taking legal action against four NHS trusts for using the cheaper drug.

Newer drugs may be better but there is the whole problem of diminishing returns. It doesn't matter how good drugs are against placebos, what matters is how good they are against the generic / lower cost / alternative treatments.