Latest Drugwonks' Blog
In a recent tweet, Senator Bernie Sanders made it clear he’s upset about high drug prices. Less clear is that his righteous wrath will put patients in harm’s way. He proved yet again he’s uninformed about the facts and unconcerned about the unintended consequences of his actions.
The target of Senator Sanders terrible tweet was a small innovative pharmaceutical company (Ariad Pharmaceuticals) and its innovative drug Iclusig (for chronic myeloid leukemia treatment). The company raised the price – and Senator Sanders can’t see why. He should open his eyes.
A few facts that are worth sharing. The first is that Iclusig serves a population of approximately 1,000 to 2,000 patients. And these patients have limited options. Ariad Pharmaceuticals, works to ensures that no patient is prevented from treatment due to price. (They provide a robust support program for patients who have accessibility and affordability concerns.)
Second, these types of ultra-orphan disease cancer patient population programs require large investments and face tremendous odds. Ariad sure doesn’t look profitable. They’ve invested more than $1.3 billion in R&D and accumulated losses of approximately $1.4 billion since the company was founded. But they’re betting on innovation. In 2015, Ariad generated $119 million in total revenue and invested $171 million, or 143% of revenue, in R&D alone.
Third and most importantly, Iclusig works. It significantly increases 10-year survival rates for patients with no other hope or option except for expensive stem cell transplants and draconian radiation treatments.
Attention Senator Sanders. It’s not just a question of an ecosystem-driven price. It’s also about investment and value – precisely the rationale behind the Orphan Drug Act -- to encourage investment in treatments for small and in this case, tiny, populations. “Facts,” as John Adams quipped, “are pesky things.
The target of Senator Sanders terrible tweet was a small innovative pharmaceutical company (Ariad Pharmaceuticals) and its innovative drug Iclusig (for chronic myeloid leukemia treatment). The company raised the price – and Senator Sanders can’t see why. He should open his eyes.
A few facts that are worth sharing. The first is that Iclusig serves a population of approximately 1,000 to 2,000 patients. And these patients have limited options. Ariad Pharmaceuticals, works to ensures that no patient is prevented from treatment due to price. (They provide a robust support program for patients who have accessibility and affordability concerns.)
Second, these types of ultra-orphan disease cancer patient population programs require large investments and face tremendous odds. Ariad sure doesn’t look profitable. They’ve invested more than $1.3 billion in R&D and accumulated losses of approximately $1.4 billion since the company was founded. But they’re betting on innovation. In 2015, Ariad generated $119 million in total revenue and invested $171 million, or 143% of revenue, in R&D alone.
Third and most importantly, Iclusig works. It significantly increases 10-year survival rates for patients with no other hope or option except for expensive stem cell transplants and draconian radiation treatments.
Attention Senator Sanders. It’s not just a question of an ecosystem-driven price. It’s also about investment and value – precisely the rationale behind the Orphan Drug Act -- to encourage investment in treatments for small and in this case, tiny, populations. “Facts,” as John Adams quipped, “are pesky things.
As seen in the Daily Caller
Fact Checking Hillary Clinton On Drug Pricing
Both Donald Trump and Hillary Clinton get a lot of flak for lying. But in Secretary Clinton’s latest speech on healthcare reform, she didn’t lie — she just got all her facts wrong.
Clinton used her speech to demonize pharmaceutical companies. She argued that greedy firms are gouging consumers and that government-imposed price controls are needed to protect us from them. Her rhetoric ignores reality and her proposals would harm the patients she wants to help.
According to Mrs. Clinton, Americans are “paying the highest price” for medicines, compared to citizens of other developed nations. She implied that drugcompanies are overcharging Americans just because they can. It’s not so simple.
The real reason medicines are more expensive in the United States is that the socialized medicine systems in other countries cap prices on innovator drugs, while also rationing their use. Many foreign governments threaten to break ‘drug patents if firms don’t agree to sell their products at below-market rates. As a result, American consumers shoulder a disproportionate share of the world’s research and development burden. That’s “free-riderism” and it’s not fair. It’s also important to note that generic drugs, which account for 85 percent by volume of all the medicines used in the United States, are cheaper here than in Europe or Canada.
But the answer isn’t to impose our own price caps. That would only discourage research into new medicines. Here’s a fact that Clinton didn’t mention — America invents more than half of new medicines in the world. Stifling U.S. research would lead to vastly fewer medicines here and across the globe. It’d be smarter economically, and better for patients, to negotiate stronger trade protections to prevent other nations from freeloading off American investments.
Secretary Clinton told her audience that their tax dollars fund drug safety evaluations. They do not. All of the complex and costly clinical trials that must be done to bring a new medicine to market are fielded and funded — 100 percent — by the pharmaceutical industry. They are then reviewed by the Food and Drug Administration. And industry pays for that privilege through “user fees” the FDA collects from pharmaceutical companies.
She also slammed the Medicare Part D drug benefit, touting a doctor’s claim that he “can’t prescribe certain drugs that [his] patients need” because government health programs won’t pay for them. But when it comes to Medicare, that assertion is simply false. Medicare’s prescription drug plans cover, on average, 191 of the 200 medicines most used by seniors. That’s more than most Obamacare exchange options.
In her assault on capitalism and private enterprise, Secretary Clinton singled out the price of hepatitis C drugs. She claimed that makers of these cures — which are vastly more effective than previous therapies — are gouging Americans.
The truth is radically different – and highly documented. Hepatitis C drugs are now cheaper in the United States than in Western Europe, thanks to a price war between competing manufacturers. Clinton inadvertently picked an example that proves the free market yields better, cheaper medicines than socialist systems and fix prices and ration care.
Bashing the companies that research and produce the world’s most groundbreaking medicines might give Clinton a bump in the polls. But her reality-free rhetoric has dangerous consequences.
Clinton’s attacks on drug makers have prompted a sell-off of biotech stocks multiple times over the past year. If companies can’t raise funding from investors, they’ll have to limit new research projects. That means fewer drugs down the road. What’s political expediency worth?
And if Clinton reaches the White House and actually implements price controls, it’s statistically certain Americans will lose out on lifesaving drugs. Price controls in other countries depress research spending by up to $8 billion each year — the equivalent of three or four new drugs, according to a Department of Commerce study.
For a super policy wonk, Secretary Clinton got an awful lot wrong in her recent speech. If she really wants to help patients, perhaps she should pay a little less attention to her focus groups and a little more attention to the facts.
Peter J. Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest.
Fact Checking Hillary Clinton On Drug Pricing
Both Donald Trump and Hillary Clinton get a lot of flak for lying. But in Secretary Clinton’s latest speech on healthcare reform, she didn’t lie — she just got all her facts wrong.
Clinton used her speech to demonize pharmaceutical companies. She argued that greedy firms are gouging consumers and that government-imposed price controls are needed to protect us from them. Her rhetoric ignores reality and her proposals would harm the patients she wants to help.
According to Mrs. Clinton, Americans are “paying the highest price” for medicines, compared to citizens of other developed nations. She implied that drugcompanies are overcharging Americans just because they can. It’s not so simple.
The real reason medicines are more expensive in the United States is that the socialized medicine systems in other countries cap prices on innovator drugs, while also rationing their use. Many foreign governments threaten to break ‘drug patents if firms don’t agree to sell their products at below-market rates. As a result, American consumers shoulder a disproportionate share of the world’s research and development burden. That’s “free-riderism” and it’s not fair. It’s also important to note that generic drugs, which account for 85 percent by volume of all the medicines used in the United States, are cheaper here than in Europe or Canada.
But the answer isn’t to impose our own price caps. That would only discourage research into new medicines. Here’s a fact that Clinton didn’t mention — America invents more than half of new medicines in the world. Stifling U.S. research would lead to vastly fewer medicines here and across the globe. It’d be smarter economically, and better for patients, to negotiate stronger trade protections to prevent other nations from freeloading off American investments.
Secretary Clinton told her audience that their tax dollars fund drug safety evaluations. They do not. All of the complex and costly clinical trials that must be done to bring a new medicine to market are fielded and funded — 100 percent — by the pharmaceutical industry. They are then reviewed by the Food and Drug Administration. And industry pays for that privilege through “user fees” the FDA collects from pharmaceutical companies.
She also slammed the Medicare Part D drug benefit, touting a doctor’s claim that he “can’t prescribe certain drugs that [his] patients need” because government health programs won’t pay for them. But when it comes to Medicare, that assertion is simply false. Medicare’s prescription drug plans cover, on average, 191 of the 200 medicines most used by seniors. That’s more than most Obamacare exchange options.
In her assault on capitalism and private enterprise, Secretary Clinton singled out the price of hepatitis C drugs. She claimed that makers of these cures — which are vastly more effective than previous therapies — are gouging Americans.
The truth is radically different – and highly documented. Hepatitis C drugs are now cheaper in the United States than in Western Europe, thanks to a price war between competing manufacturers. Clinton inadvertently picked an example that proves the free market yields better, cheaper medicines than socialist systems and fix prices and ration care.
Bashing the companies that research and produce the world’s most groundbreaking medicines might give Clinton a bump in the polls. But her reality-free rhetoric has dangerous consequences.
Clinton’s attacks on drug makers have prompted a sell-off of biotech stocks multiple times over the past year. If companies can’t raise funding from investors, they’ll have to limit new research projects. That means fewer drugs down the road. What’s political expediency worth?
And if Clinton reaches the White House and actually implements price controls, it’s statistically certain Americans will lose out on lifesaving drugs. Price controls in other countries depress research spending by up to $8 billion each year — the equivalent of three or four new drugs, according to a Department of Commerce study.
For a super policy wonk, Secretary Clinton got an awful lot wrong in her recent speech. If she really wants to help patients, perhaps she should pay a little less attention to her focus groups and a little more attention to the facts.
Peter J. Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest.
The Wikileaks disclosure of John Podesta's emails reveals that the Clinton campaign considered attacking FDA Commissioner Robert Califf for ties to drug companies as part of a broader effort to divert attention from Secretary Clinton's email scandal.
FDA Commissioner and Clinton Campaign Target Robert Califf, MD
It start with an email from long-time Clinton adviser Mandy Grunwald to Clinton policy adviser Ann O Leary. The email contains a link to a NY Times article on acting FDA Commissioner Rob Califf who had been nominated by President Obama to become the full time commish.
On Sep 21, 2015, at 10:33 PM, Mandy Grunwald sent an email to Clintion policy advierse Jake Sullivan and Ann O'Leary, Joel Benenson a strategist and pollster for the presidential campaign., press secretary Brian Fallon and Communications Director Jennifer Palmieri and asks: Do we want to weigh in on this?
Jake Sullivan responds: What do you think?
On Mon, Sep 21, 2015 Mandy Grunwald replies: I don't know anything about the guy. If we weren't hitting the Administration with Keystone this week, I might be tempted, but I think that probably makes it a bad idea. Lets keep an eye on it and see about those Pharma ties.
Then Anne O Leary: Interesting. I'll do some asking around to see what folks in the public health world think.
On Sat, Sep 26, 2015 at 12:53 PM, Brian Fallon : Any update on this? As we consider fights that fit into the larger themes we are trying to promote, this seems like a good fight to have. Plus, the VP would be in a box of having to support this nominee.
On Sat, Sep 26, 2015 at 12:59 PM, Jake Sullivan wrote: This is ultimately a political call, not a policy call. I don't really like the idea of bashing this White House's nominees, knowing their vetting process and standards. But if you guys want to do it, I cannot identify a policy reason not to -- you all know the facts. Ann, any other intel?
On September 26, 2015 at 1:13 pm Anne O'Leary wrote:
Califf the Obama nominee does have real ties to the drug industry - Chris Jennings is calling a few people for me to learn more so we don't tip our hand directly. We are clean on Clinton Admin FDA Commissioner - it was David Kessler, an academic who had run a teaching hospital - and best known for taking on big tobacco. We could certainly signal that we want someone willing to stand up to Pharma (in the same way Kessler stood up to Tobacco). BUT - I want to do a little more digging and due diligence before we hit this guy. Having been through a nomination fight with my husband (in which he lost), this is personal and messy and horrible on the person nominated and their families - so I don't take attacking this guy lightly. Do you want to do it on Meet the Press?
That was follwed by an email from Podesta on Sep 26, 2015, at 2:27 PM, who wrote: " I think we will pay a huge price with the WH on this one. Worries me."
Nothing transpired. But the back and forth about whether to trash Califf came down to politics, not policy differences. And it had NOTHING to do with Califf as a person, a physician, a researcher and public servant. It was clear that Team Clinton was interested only in dirt that was newsworthy. It was simply a matter of whether attacking Califf on Meet The Press would be worth the headache of taking on an Obama nominee. How lovely.
Rob Califf should remain FDA commissioner regardless of who is elected president because of his qualifications and commitment to accelerating access to safe and effective medicines. I am concerned that the Clinton team -- which exults about going to war against Pharma -- will seek to replace Dr. Califf. That will trigger a prolonged political war that will undermine the FDA.
The emails reveal a Team Clinton eager to find ways to smash up people for short term political gain without tipping their hand that they are behind the hit job. Like it's war on pharma, it has less to do with policies and more to do about the benefits of creating and attacking enemies.
FDA Commissioner and Clinton Campaign Target Robert Califf, MD
It start with an email from long-time Clinton adviser Mandy Grunwald to Clinton policy adviser Ann O Leary. The email contains a link to a NY Times article on acting FDA Commissioner Rob Califf who had been nominated by President Obama to become the full time commish.
On Sep 21, 2015, at 10:33 PM, Mandy Grunwald sent an email to Clintion policy advierse Jake Sullivan and Ann O'Leary, Joel Benenson a strategist and pollster for the presidential campaign., press secretary Brian Fallon and Communications Director Jennifer Palmieri and asks: Do we want to weigh in on this?
Jake Sullivan responds: What do you think?
On Mon, Sep 21, 2015 Mandy Grunwald replies: I don't know anything about the guy. If we weren't hitting the Administration with Keystone this week, I might be tempted, but I think that probably makes it a bad idea. Lets keep an eye on it and see about those Pharma ties.
Then Anne O Leary: Interesting. I'll do some asking around to see what folks in the public health world think.
On Sat, Sep 26, 2015 at 12:53 PM, Brian Fallon : Any update on this? As we consider fights that fit into the larger themes we are trying to promote, this seems like a good fight to have. Plus, the VP would be in a box of having to support this nominee.
On Sat, Sep 26, 2015 at 12:59 PM, Jake Sullivan wrote: This is ultimately a political call, not a policy call. I don't really like the idea of bashing this White House's nominees, knowing their vetting process and standards. But if you guys want to do it, I cannot identify a policy reason not to -- you all know the facts. Ann, any other intel?
On September 26, 2015 at 1:13 pm Anne O'Leary wrote:
Califf the Obama nominee does have real ties to the drug industry - Chris Jennings is calling a few people for me to learn more so we don't tip our hand directly. We are clean on Clinton Admin FDA Commissioner - it was David Kessler, an academic who had run a teaching hospital - and best known for taking on big tobacco. We could certainly signal that we want someone willing to stand up to Pharma (in the same way Kessler stood up to Tobacco). BUT - I want to do a little more digging and due diligence before we hit this guy. Having been through a nomination fight with my husband (in which he lost), this is personal and messy and horrible on the person nominated and their families - so I don't take attacking this guy lightly. Do you want to do it on Meet the Press?
That was follwed by an email from Podesta on Sep 26, 2015, at 2:27 PM, who wrote: " I think we will pay a huge price with the WH on this one. Worries me."
Nothing transpired. But the back and forth about whether to trash Califf came down to politics, not policy differences. And it had NOTHING to do with Califf as a person, a physician, a researcher and public servant. It was clear that Team Clinton was interested only in dirt that was newsworthy. It was simply a matter of whether attacking Califf on Meet The Press would be worth the headache of taking on an Obama nominee. How lovely.
Rob Califf should remain FDA commissioner regardless of who is elected president because of his qualifications and commitment to accelerating access to safe and effective medicines. I am concerned that the Clinton team -- which exults about going to war against Pharma -- will seek to replace Dr. Califf. That will trigger a prolonged political war that will undermine the FDA.
The emails reveal a Team Clinton eager to find ways to smash up people for short term political gain without tipping their hand that they are behind the hit job. Like it's war on pharma, it has less to do with policies and more to do about the benefits of creating and attacking enemies.
Written by Rafael Fonseca MD and Robert Goldberg PhD
Two recent articles in the NEJM (1) e) and Annals of Internal Medicine (2) propose that restrictions or eliminations of co-pay assistance programs by pharmaceutical companies are needed since they distort the market and therefore, while few benefit, many suffer in the form of higher healthcare expenditures. They are factually correct but the conclusions point to a larger problem; the third party payer system in health care. When consumers (in this case patients) are detached from the price consequence of transactions they will be less careful in their selections. However, the proposal presented in these articles are, at least in the short-term, patient unfriendly and immediate implementation of their recommendations would limit, severely, access; particularly for patients with serious or life-threatening conditions. The Annals article is notorious in that it fails to mention the increased cost-sharing pressure that patients are facing from the payers.
So what are the solutions? Ideally, healthcare should be divided between the routine care and the catastrophic care. Routine care should involve much more “skin in the game.” In my view, regular care should not be covered under traditional heath insurance but be bought directly, with compassionate support tools for those in need. In this type of care I could envision more direct pressure in the selection of older medications for which many alternative exist. There are many options for anti-hypertensive medications, statins, etc. In the absence of a third party payer system the Epipen would never cost $600. This is where consumer pressure, with co-pay requirement as a discriminator, should be employed; vigorously, if you may. I would not disagree that if an equivalent generic exists, a true equivalent, then having the patient paying more for a branded product is fine. This is not to say that branded products and generics are always the same. Sometimes production quality may suffer or sometimes an alternative may not be the same as the original, similar and yet not the same product. But if you want the branded statin maybe you should pay a bit more. Who could argue with that?
However, for more expensive medications, medications used for serious, chronic severe or catastrophic illnesses the pressure seems misguided. For instance preventing co-pay assistance for patients with cancer seems inhumane. Dusetzina and colleagues have documented that lack of supplemental insurance delays initiation of treatment for CML (3). Should we disincentivize the use of medications that can be lifesaving for a cancer patient so they consider inferior treatments? Should we place additional burdens in someone who is at high risk for bankruptcy? Someone who cannot work or whose caregiver cannot work? That seems inappropriate. Furthermore, for many of these newer and expensive medications there are no suitable alternatives (4). Dr. Bach and colleagues were able to reduce the price of a competing monoclonal antibody, but that is a rarity in oncology and more of an exception. Should myeloma patients forgo lenalidomide in favor or thalidomide and face certain peripheral neuropathy? Or should a diabetic myeloma patient be forced to be treated with bortezomib instead of carfilzomib and again face neuropathy? To create a disincentive as this for people facing serious medical illness appears to me as a fundamental violation of medicine’s stance of benevolence. Rather this has the appeal of altruism, sacrifice the few for the benefit of the many.
Lastly, government regulations that eliminate this co-pay assistance are demonstrably patient unfriendly. Commercially insured patients can receive this directly and in another study by Dusetzina she shows that the average co-pay for specialty medications is only $35 (5). In another study it was shown that 90% of myeloma patients pay less than $100 per month for lenalidomide, a backbone for the treatment of this disease. In the meantime, Medicare beneficiaries struggle to find indirect support with funds provided by the pharmaceutical companies to third parties. I’ve written about this topic before, but again is a built-in disincentive to lessen the use of medications (6). What if these medications save dollars by preventing hospitalizations and decreasing expenses elsewhere? Once again it is pound foolish to only concentrate on the price of drugs.
References
1. Dafny LS, Ody CJ, Schmitt MA. Undermining Value-Based Purchasing - Lessons from the Pharmaceutical Industry. The New England journal of medicine. 2016. Epub 2016/10/13. doi: 10.1056/NEJMp1607378. PubMed PMID: 27732125.
2. Ubel PA, Bach PB. Copay Assistance for Expensive Drugs: A Helping Hand That Raises Costs. Annals of internal medicine. 2016. Epub 2016/10/11. doi: 10.7326/M16-1334. PubMed PMID: 27723893.
3. Winn A, Keating N, Dusetzina S. Factors Associated With Tyrosine Kinase Inhibitor Initiation and Adherence Among Medicare Beneficiaries With Chronic Myeloid Leukemia. Journal of Clinical Oncology. 2016;10.1200/JCO.2016.67.4184.
4. Fonseca R. 2016. Available from: https://tmblr.co/Z0_1wo2CgPnw1.
5. Dusetzina SB. Share Of Specialty Drugs In Commercial Plans Nearly Quadrupled, 2003-14. Health Aff (Millwood). 2016;35(7):1241-6. Epub 2016/07/08. doi: 10.1377/hlthaff.2015.1657. PubMed PMID: 27385240.
6. Fonseca R. 2016. Available from: https://tmblr.co/Z0_1wo2D7lHbj.
From the pages of today's edition of the Boston Herald:
Insurers not covering new cancer treatments
Poor insurance coverage is causing cancer patients to miss out on cutting-edge technologies that use gene analysis to determine the best treatments — a fact a former commissioner of the Food and Drug Administration is calling another example of Obamacare’s failure to provide Americans with high-quality health care.
“There’s a degree of dishonesty about what the Affordable Care Act provides, and that is starkly clear when a patient has a serious type of cancer,” said former FDA Associate Commissioner Peter J. Pitts, who now serves as president of the Center for Medicine in the Public Interest. “The soundbite of the ACA is, many more Americans now have health insurance. But the health insurance isn’t worth the paper it’s written on.”
Pitts said companies are covering the old-fashioned, less-effective chemotherapy regimens rather than more sophisticated approaches and prescription benefit managers are opting not to reimburse the more innovative, expensive treatments. Instead, they are negotiating rebates with pharmaceutical companies that they pocket, rather than passing the savings along to the patient.
“It’s time for insurance companies and prescription benefit managers to step up and do the right thing by putting patients first,” Pitts said. “When they choose not to reimburse for a product because it doesn’t earn them enough money, even though it could save a patient’s life, I think it’s despicable.”
And coverage for the treatments tends to vary by tens of thousands of dollars nationwide, depending on the company and geographic region, according to a University of Texas MD Anderson Cancer Center study published Monday in the journal Cancer.
The study found that insurance costs varied by as much as $47,000 for women on a chemotherapy plan that included the drug Herceptin, which is used to treat breast and stomach cancers by keeping cell growth at bay — and out-of-pocket costs range from $2,700 to $3,400.
The extra costs caused by these variations leads to an additional $1 billion spent to treat breast cancer in the U.S. each year, the study found. But cancer patients will often be covered for a treatment if it has been proven effective by the FDA, according to Dr. Harold Burstein, a breast cancer specialist at Dana-Farber Cancer Institute, who said: “The business of cures not being covered is extraordinarily uncommon.”
Many of the additional costs for the patient, he said, come in the form of lost income and child care.
“Those things are harder to measure than direct hospital bills,” he said, “and they generally have more of an impact.”
Insurers not covering new cancer treatments
Poor insurance coverage is causing cancer patients to miss out on cutting-edge technologies that use gene analysis to determine the best treatments — a fact a former commissioner of the Food and Drug Administration is calling another example of Obamacare’s failure to provide Americans with high-quality health care.
“There’s a degree of dishonesty about what the Affordable Care Act provides, and that is starkly clear when a patient has a serious type of cancer,” said former FDA Associate Commissioner Peter J. Pitts, who now serves as president of the Center for Medicine in the Public Interest. “The soundbite of the ACA is, many more Americans now have health insurance. But the health insurance isn’t worth the paper it’s written on.”
Pitts said companies are covering the old-fashioned, less-effective chemotherapy regimens rather than more sophisticated approaches and prescription benefit managers are opting not to reimburse the more innovative, expensive treatments. Instead, they are negotiating rebates with pharmaceutical companies that they pocket, rather than passing the savings along to the patient.
“It’s time for insurance companies and prescription benefit managers to step up and do the right thing by putting patients first,” Pitts said. “When they choose not to reimburse for a product because it doesn’t earn them enough money, even though it could save a patient’s life, I think it’s despicable.”
And coverage for the treatments tends to vary by tens of thousands of dollars nationwide, depending on the company and geographic region, according to a University of Texas MD Anderson Cancer Center study published Monday in the journal Cancer.
The study found that insurance costs varied by as much as $47,000 for women on a chemotherapy plan that included the drug Herceptin, which is used to treat breast and stomach cancers by keeping cell growth at bay — and out-of-pocket costs range from $2,700 to $3,400.
The extra costs caused by these variations leads to an additional $1 billion spent to treat breast cancer in the U.S. each year, the study found. But cancer patients will often be covered for a treatment if it has been proven effective by the FDA, according to Dr. Harold Burstein, a breast cancer specialist at Dana-Farber Cancer Institute, who said: “The business of cures not being covered is extraordinarily uncommon.”
Many of the additional costs for the patient, he said, come in the form of lost income and child care.
“Those things are harder to measure than direct hospital bills,” he said, “and they generally have more of an impact.”
Anthem Health has issued a decision deny coverage of Exondys 51, the first drug to treat a form of Duchenne’s muscular dystrophy. As STAT’s Ed Silverman reports : Anthem “claims that the drug is “not medically necessary” and that “the clinical benefit … has not been demonstrated.” Duchenne is a rare disease that confines boys to wheelchairs and condemns them to an early death.
The decision was followed by this: "Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members," Anthem spokeswoman Leslie Porras said in an emailed statement on Friday.
Not exactly. Anthem conveniently ignored the fact that the FDA does not approve drugs that have no medical benefit or more precisely, the FDA approves drugs that are likely to promote treatment of specific conditions. Anthem claims it is denying coverage because a multitude of limitations cast further doubt on the reliability of dystrophin levels as a surrogate endpoint for clinical efficacy in DMD (FDA, 2016). But these limitations were considered and approval was given. In fact, in ignoring the FDA’s decision Anthem is taking refuge in the statements of FDA reviewers who raised concerns about clinical trials but were overruled by Janet Woodcock and Robert Califf, the FDA commissioner.
I wonder if Anthem would have a problem with doctors and its utilization review munchkins ignoring the company’s medical policy on any treatment based on evidence ultimately rejected by Anthem. And I wonder what a disability rights attorney will do with the fact that Anthem is willfully misapplying the term medically necessary.
In fact, Anthem has no problem covering acupuncture, massage therapy, spine manipulations and naturopathy.
Compare Anthem’s demand for more evidence before covering Exondys with the statement accompanying it’s decision to cover acupuncture:
"Acupuncture as a therapeutic intervention is widely practiced in the United States. There have been many studies of its potential usefulness. However, many of these studies provide equivocal results because of design, sample size, and other factors. The issue is further complicated by inherent difficulties in the use of appropriate controls, such as placebo and sham acupuncture groups…Further research is likely to uncover additional areas where acupuncture interventions will be useful.”
Anthem covers treatments that have less medical evidence of benefit for less severe ailments than DMD. And it covers treatments based on the it’s belief that research will find acupuncture useful” (though not clinically effective.) That’s because they attract millions of fairly healthy consumers.
There are about 17000 boys with DMD. Providing the drug also means paying for all the other supportive services, emergency care, hospitalizations that such kids may require. To Anthem, spending money to help boys with DMD stay independent and alive is a money loser.
That’s the only evidence Anthem cares about.
The decision was followed by this: "Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members," Anthem spokeswoman Leslie Porras said in an emailed statement on Friday.
Not exactly. Anthem conveniently ignored the fact that the FDA does not approve drugs that have no medical benefit or more precisely, the FDA approves drugs that are likely to promote treatment of specific conditions. Anthem claims it is denying coverage because a multitude of limitations cast further doubt on the reliability of dystrophin levels as a surrogate endpoint for clinical efficacy in DMD (FDA, 2016). But these limitations were considered and approval was given. In fact, in ignoring the FDA’s decision Anthem is taking refuge in the statements of FDA reviewers who raised concerns about clinical trials but were overruled by Janet Woodcock and Robert Califf, the FDA commissioner.
I wonder if Anthem would have a problem with doctors and its utilization review munchkins ignoring the company’s medical policy on any treatment based on evidence ultimately rejected by Anthem. And I wonder what a disability rights attorney will do with the fact that Anthem is willfully misapplying the term medically necessary.
In fact, Anthem has no problem covering acupuncture, massage therapy, spine manipulations and naturopathy.
Compare Anthem’s demand for more evidence before covering Exondys with the statement accompanying it’s decision to cover acupuncture:
"Acupuncture as a therapeutic intervention is widely practiced in the United States. There have been many studies of its potential usefulness. However, many of these studies provide equivocal results because of design, sample size, and other factors. The issue is further complicated by inherent difficulties in the use of appropriate controls, such as placebo and sham acupuncture groups…Further research is likely to uncover additional areas where acupuncture interventions will be useful.”
Anthem covers treatments that have less medical evidence of benefit for less severe ailments than DMD. And it covers treatments based on the it’s belief that research will find acupuncture useful” (though not clinically effective.) That’s because they attract millions of fairly healthy consumers.
There are about 17000 boys with DMD. Providing the drug also means paying for all the other supportive services, emergency care, hospitalizations that such kids may require. To Anthem, spending money to help boys with DMD stay independent and alive is a money loser.
That’s the only evidence Anthem cares about.
From today's edition of Morning Consult ...
21st Century Pharmacovigilance and the Role of Artificial Intelligence
Artificial intelligence has an unimaginable potential. Within the next couple of years, it will revolutionize every area of our life, including medicine — and pharmacovigilance. Usually, we make sense of the world around us with the help of rules and processes that build up a system. The world of Big Data is so huge that we will need artificial intelligence to be able to keep track of it.
With the evolution of digital capacity, more and more data is produced and stored in the digital space. The amount of available digital data is growing by a mind-blowing speed, doubling every two years. In 2013, it encompassed 4.4 zettabytes, however by 2020 the digital universe — the data we create and copy annually — will reach 44 zettabytes, or 44 trillion gigabytes.
In a world increasingly driven by outcomes reporting and Big Data, more patient-level information from individual consumers is not always synonymous with better information. The good news is that artificial intelligence will facilitate what the pharmacovigilance ecosystem lacks today — a coordinated and efficient systems for developing actionable evidence on safety and effectiveness.
Today, the absence of these capabilities significantly affects the public health by creating obstacles for patients and clinicians to receive the meaningful information they need to make informed decisions, perpetuating unnecessarily long delays and gaps in effective and timely safety communications and recall management, hindering the timely development of new and innovative treatment options, and increasing the overall costs and inefficiency of the healthcare system.
In considering the role artificial intelligence can play in the both the near and long-term future of outcomes centricity, we need to discuss the concept of Design Thinking which requires cross-examination of the filters used in defining a problem and to revise the potential opportunities before developing strategies and tactics. Design Thinking requires cross-functional insights into a problem by varied perspectives as well as constant and relentless questioning. In Design Thinking observation takes center stage. In the “Sciences of the Artificial,” Herbert Simon has defined “design” as the “transformation of existing conditions into preferred ones.”
Unlike Critical Thinking, which is a process of analysis, Design Thinking is a creative process based on the creation of action-oriented ideas. AI can be a revolutionary tool to develop those action-oriented ideas. And, just for the record, “action-oriented” and “pharmacovigilance” are not mutually exclusive terms.
There is so much data to utilize: patient medical history records, treatment data — and lately information coming from wearable health trackers and sensors. This huge amount of data must be analyzed not only to provide patients who want to be proactive with better suggestions about lifestyle, but also to serve providers with instructive pieces of information about how to design healthcare based on the needs and habits of patients, and provide regulators not just with more data, but better date in context. Can AI usage for adverse event reporting and prediction be far behind?
Artificial intelligence is already found in several areas in health care, from data mining electronic health records to helping design treatment plans, from health assistance to medication management.
Artificial intelligence will have a huge impact on genetics and genomics, helping to identify patterns in huge data sets of genetic information and medical records, looking for mutations and linkages to disease. There are companies out there today inventing a new generation of computational technologies that can tell doctors what will happen within a cell when DNA is altered by genetic variation, whether natural or therapeutic. Imagine the predictive capabilities for pharmacovigilance.
But making knowledge actionable requires the application of proven analytical methods and techniques to biomedical data in order to produce reliable conclusions. Until recently, such analysis was done by experts operating in centers that typically restricted access to data. This “walled garden” approach evolved for several reasons: the imperative to protect the privacy and confidentiality of sensitive medical data; concern about the negative consequences that could arise from inappropriate, biased, or incompetent analysis; and the tendency to see data as a competitive asset. Regardless of the specific reason, the result has been the same: widespread and systemic barriers to data sharing.
If we are to reverse these tendencies and foster a new approach to creating evidence, we must bear in mind that there must be a common approach to how data is presented, reported and analyzed and strict methods for ensuring patient privacy and data security.
Rules of engagement must be transparent and developed through a process that builds consensus across the relevant ecosystem and its stakeholders. To ensure support across a diverse ecosystem that often includes competing priorities and incentives, the system’s output must be intended for the public good and be readily accessible to all stakeholders.
For any of this to work — and especially in the world of pharmacovigilace, we must view artificial intelligence through the lens of 21st century interoperability: the idea that different systems used by different groups of people can be used for a common purpose because those systems share standards and approaches.
And as Philip K. Dick wrote, “Reality is that which, when you stop believing in it, doesn’t go away.”
Will our socio-economic “technology gap” lead to a more pronounced “adherence/compliance gap?” It’s an important question. That’s why it’s crucial we remember there is no one-size-fits all solution. But that mustn’t mean we disregard the reality of the growth and pervasiveness of apps, mobile apps. Let’s face it, when it comes to mobile phones, any gap is rather narrow.
As the American industrialist Walter O’Malley once opined, “The future is just one damned thing after another.” Much depends not just on infrastructure, but also on capabilities, and trust.
The end goal is the same for all stakeholders — ensuring optimal use of resources for health care systems; improving access to value-adding medicines for patients; and appropriate reward for innovation.
As management guru W. Edwards Deming once quipped, “Change is not required. Survival is not necessary.”
Artificial Intelligence is here. Fasten your seat belts.
21st Century Pharmacovigilance and the Role of Artificial Intelligence
Artificial intelligence has an unimaginable potential. Within the next couple of years, it will revolutionize every area of our life, including medicine — and pharmacovigilance. Usually, we make sense of the world around us with the help of rules and processes that build up a system. The world of Big Data is so huge that we will need artificial intelligence to be able to keep track of it.
With the evolution of digital capacity, more and more data is produced and stored in the digital space. The amount of available digital data is growing by a mind-blowing speed, doubling every two years. In 2013, it encompassed 4.4 zettabytes, however by 2020 the digital universe — the data we create and copy annually — will reach 44 zettabytes, or 44 trillion gigabytes.
In a world increasingly driven by outcomes reporting and Big Data, more patient-level information from individual consumers is not always synonymous with better information. The good news is that artificial intelligence will facilitate what the pharmacovigilance ecosystem lacks today — a coordinated and efficient systems for developing actionable evidence on safety and effectiveness.
Today, the absence of these capabilities significantly affects the public health by creating obstacles for patients and clinicians to receive the meaningful information they need to make informed decisions, perpetuating unnecessarily long delays and gaps in effective and timely safety communications and recall management, hindering the timely development of new and innovative treatment options, and increasing the overall costs and inefficiency of the healthcare system.
In considering the role artificial intelligence can play in the both the near and long-term future of outcomes centricity, we need to discuss the concept of Design Thinking which requires cross-examination of the filters used in defining a problem and to revise the potential opportunities before developing strategies and tactics. Design Thinking requires cross-functional insights into a problem by varied perspectives as well as constant and relentless questioning. In Design Thinking observation takes center stage. In the “Sciences of the Artificial,” Herbert Simon has defined “design” as the “transformation of existing conditions into preferred ones.”
Unlike Critical Thinking, which is a process of analysis, Design Thinking is a creative process based on the creation of action-oriented ideas. AI can be a revolutionary tool to develop those action-oriented ideas. And, just for the record, “action-oriented” and “pharmacovigilance” are not mutually exclusive terms.
There is so much data to utilize: patient medical history records, treatment data — and lately information coming from wearable health trackers and sensors. This huge amount of data must be analyzed not only to provide patients who want to be proactive with better suggestions about lifestyle, but also to serve providers with instructive pieces of information about how to design healthcare based on the needs and habits of patients, and provide regulators not just with more data, but better date in context. Can AI usage for adverse event reporting and prediction be far behind?
Artificial intelligence is already found in several areas in health care, from data mining electronic health records to helping design treatment plans, from health assistance to medication management.
Artificial intelligence will have a huge impact on genetics and genomics, helping to identify patterns in huge data sets of genetic information and medical records, looking for mutations and linkages to disease. There are companies out there today inventing a new generation of computational technologies that can tell doctors what will happen within a cell when DNA is altered by genetic variation, whether natural or therapeutic. Imagine the predictive capabilities for pharmacovigilance.
But making knowledge actionable requires the application of proven analytical methods and techniques to biomedical data in order to produce reliable conclusions. Until recently, such analysis was done by experts operating in centers that typically restricted access to data. This “walled garden” approach evolved for several reasons: the imperative to protect the privacy and confidentiality of sensitive medical data; concern about the negative consequences that could arise from inappropriate, biased, or incompetent analysis; and the tendency to see data as a competitive asset. Regardless of the specific reason, the result has been the same: widespread and systemic barriers to data sharing.
If we are to reverse these tendencies and foster a new approach to creating evidence, we must bear in mind that there must be a common approach to how data is presented, reported and analyzed and strict methods for ensuring patient privacy and data security.
Rules of engagement must be transparent and developed through a process that builds consensus across the relevant ecosystem and its stakeholders. To ensure support across a diverse ecosystem that often includes competing priorities and incentives, the system’s output must be intended for the public good and be readily accessible to all stakeholders.
For any of this to work — and especially in the world of pharmacovigilace, we must view artificial intelligence through the lens of 21st century interoperability: the idea that different systems used by different groups of people can be used for a common purpose because those systems share standards and approaches.
And as Philip K. Dick wrote, “Reality is that which, when you stop believing in it, doesn’t go away.”
Will our socio-economic “technology gap” lead to a more pronounced “adherence/compliance gap?” It’s an important question. That’s why it’s crucial we remember there is no one-size-fits all solution. But that mustn’t mean we disregard the reality of the growth and pervasiveness of apps, mobile apps. Let’s face it, when it comes to mobile phones, any gap is rather narrow.
As the American industrialist Walter O’Malley once opined, “The future is just one damned thing after another.” Much depends not just on infrastructure, but also on capabilities, and trust.
The end goal is the same for all stakeholders — ensuring optimal use of resources for health care systems; improving access to value-adding medicines for patients; and appropriate reward for innovation.
As management guru W. Edwards Deming once quipped, “Change is not required. Survival is not necessary.”
Artificial Intelligence is here. Fasten your seat belts.
The FDA has announcing a public meeting to discuss scientific and technical issues relating to formulation development and pre-market evaluation of opioid drug products with abuse-deterrent properties. The meeting is intended to give FDA the opportunity to discuss, and seek public input from stakeholders on, the approach to testing FDA recommended in its draft guidance “General Principles for Evaluating the Abuse Deterrence of Generic Solid Oral Opioid Drug Products.” The meeting will also provide an opportunity to discuss FDA’s efforts to develop standardized in vitro testing methodologies for evaluating the abuse deterrence of opioid drug products. FDA is seeking input from all stakeholders, including patients, health care providers, health care payers, the pharmaceutical industry, patient advocates, academics, researchers, and other government entities.
The FDA may hold one or more additional meetings in the future to discuss the risk-benefit paradigm for opioid drug products to ensure that FDA is appropriately considering the full public health impact of prescription opioid drug products and the post-market impact (“real world effects”) of abuse-deterrent opioid drug products.
The public meeting will be held on October 31, 2016, from 8:30 a.m. to 4:30 p.m. and November 1, 2016, from 8:30 a.m. to 4 p.m.
Full details can be found here.
The FDA may hold one or more additional meetings in the future to discuss the risk-benefit paradigm for opioid drug products to ensure that FDA is appropriately considering the full public health impact of prescription opioid drug products and the post-market impact (“real world effects”) of abuse-deterrent opioid drug products.
The public meeting will be held on October 31, 2016, from 8:30 a.m. to 4:30 p.m. and November 1, 2016, from 8:30 a.m. to 4 p.m.
Full details can be found here.
Amy Stein Harvey
My cousin Amy Stein Harvey passed away after living with ovarian cancer for 7 years. She was diagnosed at Stage III C and was told she would likely die within 3 years. Not only did she "beat the odds" in addition to holding down a job and raising kids, Amy become a powerful advocate for cancer research and for legislation requiring new oral cancer drugs to have the same cost sharing as injectible. She lived in Michigan, still one of only 8 states that have refused to enact oral parity. Three days before she died, her friends and supporters participated in a walk in support of oral parity and research. Amy's Army, as they call themselves will continue her fight.
Her last post on Facebook (see below) is beyond inspirational. She teaches us that the source of our urge to live longer is the desire to love and deepen our capacity to love.
Amy, we will complete the mission you started. We miss you and will always love you. Give my mom a kiss for me...
"Today marks another milestone for me. Today is my 7 year cancerversary! I have been fighting this awful disease for 7 long years. Some days are better than others, but I know that the next day things will turn around. They say that women diagnosed with stage IIIC ovarian cancer don't have a good survival rate. Well, take that stupid cancer. I have beaten the odds!! This past year has had more downs than ups, but I am hopefully on the right track now. I have gone to Washington DC and Lansing to let my voice be heard and put a face to a stastic, I have taken a fabulous cruise to Alaska, and I was able to see Benjamin finish elementary school. Cancer, you listen to me... you're never going to get me down! I am a fighter from the word go and I am going to win this war!!! So, I want to thank everyone for all they have done for our family and most importantly standing next to me in the fight of my life!!!"
A new report in the British Medical Journal “reveals” that many U.S. government regulators who review the safety of prescription drugs later go on to work for the pharmaceutical industry.
As opposed to – the automotive industry?
The authors identified individuals working at the FDA who were charged with reviewing hematology-oncology drugs from 2001 to 2010. They identified 55 reviewers. They found that 49 per cent were still employed with the FDA. Of the half that left, 58 per cent had moved on to jobs in the biopharmaceutical industry or as consultants to the industry. They could not find employment information for about 31 per cent of the reviewers who no longer work for the FDA. According to authors Jeffrey Bien and Vinay Prasad, “The transition from regulator to advising companies seems logical, but it raises concern as to whether regulators indefatigably act in the public interest.”
Hogwash.
Here’s what the FDA has to say:
"Federal laws and FDA ethics rules cover issues like outside employment, avoiding real and apparent conflicts of interest, recusals, disclosure requirements, protecting confidentiality, a ban on gifts from regulated industry, and avoiding conflicts should a federal employee choose to seek or negotiate outside employment," the spokesperson said. "Furthermore, past federal employees are bound by additional rules protecting the confidentiality of information they worked on while in federal service, a cooling-off requirement for senior employees, and other important rules against switching sides, contacting former employees, and contacting agency leaders."
And as the Pink Sheet reports:
“Peter Pitts, President of the Center for Medicine in the Public Interest and a former FDA associate commissioner, also slammed the findings of the report. He echoed FDA's comments, while also describing its authors as "ignorant."
"Former FDA employees understand the difference between sharing their expertise with industry and trying to unfairly influence their former colleagues," Pitts told the Pink Sheet. "They are the first to point out their jobs aren't to influence the agency but rather to share how best to properly communicate relevant information, and completely within appropriate limits. Former FDA employees understand the difference between sharing their expertise with industry and trying to unfairly influence their former colleagues. They are the first to point out their jobs aren't to influence the agency but rather to share how best to properly communicate relevant information, and completely within appropriate limits." Pitts added that he has never met a former FDAer "who ever asked for a short cut or an inappropriate edge. Shame on those who would allude otherwise." He further said that people should be applauding the fact that industry, like FDA, is looking to attract "the best and the brightest" to spur innovation.
Any former FDAer who tried to inappropriately influence an agency decision on behalf of a client would become persona non grata – and rightly so. What the authors seem not to understand is that there’s a world of difference between expert navigation and conflicts of interest.
As opposed to – the automotive industry?
The authors identified individuals working at the FDA who were charged with reviewing hematology-oncology drugs from 2001 to 2010. They identified 55 reviewers. They found that 49 per cent were still employed with the FDA. Of the half that left, 58 per cent had moved on to jobs in the biopharmaceutical industry or as consultants to the industry. They could not find employment information for about 31 per cent of the reviewers who no longer work for the FDA. According to authors Jeffrey Bien and Vinay Prasad, “The transition from regulator to advising companies seems logical, but it raises concern as to whether regulators indefatigably act in the public interest.”
Hogwash.
Here’s what the FDA has to say:
"Federal laws and FDA ethics rules cover issues like outside employment, avoiding real and apparent conflicts of interest, recusals, disclosure requirements, protecting confidentiality, a ban on gifts from regulated industry, and avoiding conflicts should a federal employee choose to seek or negotiate outside employment," the spokesperson said. "Furthermore, past federal employees are bound by additional rules protecting the confidentiality of information they worked on while in federal service, a cooling-off requirement for senior employees, and other important rules against switching sides, contacting former employees, and contacting agency leaders."
And as the Pink Sheet reports:
“Peter Pitts, President of the Center for Medicine in the Public Interest and a former FDA associate commissioner, also slammed the findings of the report. He echoed FDA's comments, while also describing its authors as "ignorant."
"Former FDA employees understand the difference between sharing their expertise with industry and trying to unfairly influence their former colleagues," Pitts told the Pink Sheet. "They are the first to point out their jobs aren't to influence the agency but rather to share how best to properly communicate relevant information, and completely within appropriate limits. Former FDA employees understand the difference between sharing their expertise with industry and trying to unfairly influence their former colleagues. They are the first to point out their jobs aren't to influence the agency but rather to share how best to properly communicate relevant information, and completely within appropriate limits." Pitts added that he has never met a former FDAer "who ever asked for a short cut or an inappropriate edge. Shame on those who would allude otherwise." He further said that people should be applauding the fact that industry, like FDA, is looking to attract "the best and the brightest" to spur innovation.
Any former FDAer who tried to inappropriately influence an agency decision on behalf of a client would become persona non grata – and rightly so. What the authors seem not to understand is that there’s a world of difference between expert navigation and conflicts of interest.
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.
