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The Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) proposed by Senator Kay Hagen (D,NC) would create two new FDA approval pathways. Drugs would be eligible for "progressive" or "exceptional" approval if they provide meaningful advances in the treatment of an unmet serious or life threatening condition. FDA could also approve drugs based on approval in the EU as well as in Australia, Canada and some other countries.
TREAT would permit progressive approval based on data "reasonably likely" to predict clinical benefit, the standard currently used for accelerated approval. Unlike accelerated approval, drugs could receive progressive approval without data from a surrogate endpoint. Exceptional approval could be granted when the data necessary to satisfy the standard for approval "cannot ethically, feasibly or practicably be generated." would also relax conflict of interest restrictions for advisory committee members, create a fixed term for the FDA commissioner, and revise FDA's mission statement to emphasize the promotion of biomedical innovation.
The draft bill can be found here.
http://washingtonexaminer.com/opinion/columnists/2011/11/david-salzmans-valiant-fight-what-truly-matters
David Salzman's valiant fight for what truly matters
By: Diana Furchtgott-Roth | 11/10/11 8:05 PM
It was December 2006, just before Christmas, when my friend David Salzman called. "I have bile duct cancer, and they've given me only three months to live."
That was almost five years ago. At 1 am on Saturday morning, David, age 53, a Maryland entrepreneur, succumbed to an infection, dying at Johns Hopkins Hospital in Baltimore, with his mother, Nancy Salzman, next to him.
He was one of the longest-surviving patients with bile duct cancer. As well as his mother, he leaves his wife, Beth Kevles; two children, Michael, 16, and Joel, 14; and his brothers, Andy and Jimmy.
For David, our health insurance system worked. As an entrepreneur, he had small-business insurance through Blue Cross Blue Shield. It didn't drop him and never questioned his care. David had only praise for Blue Cross.
I met David in Washington, D.C., in 1986. With two degrees in physics, a B.S. from Yale and a Ph.D, from the University of Chicago, he could have taken the usual academic route. Instead, he founded startups to develop new products.
One of his first companies was Polychip, where he pioneered proximity chip-to-chip communication, which dramatically increases the data rate between chips by stacking them on top of each other.
David sold that patent to Sun Microsystems, later bought by Oracle, which now has a government contract using the technology to develop a new generation of supercomputers.
Another of his companies, LightSpin Technologies, uses new ways to manipulate, detect, and generate light. When David died, he was developing ways to use light waves to find land mines. He considered this vital because of the danger posed by hidden mines.
One of his partners, Eric Harmon, told me, "You shake the ground with a loud noise, and then use the pattern of the sound waves to find the land mines."
Right until his death, David was active in community meetings about the new Montgomery County rail line, the Purple Line. He was intensely concerned with the way politics were overcoming science, and showed how studies justifying the project were artificially inflating ridership and energy savings and minimizing the noise levels.
David was more than just a scientist, he was a modern Renaissance man, able to enjoy literature, opera, and politics with equal knowledge and gusto. That's why my husband and I asked him to be the godfather to our third son, Godfrey, hoping a little magic would rub off.
"There wasn't anything that could be learned that David didn't want to learn," his mother told me on Wednesday.
This extended to his cancer treatments. David's college buddy, Phil Schiff, organized a team of friends to drive David to Hopkins for his weekly treatments so that Beth could spend more time with the boys. I was honored to be on the team.
At the hospital, David would go over the printouts of the blood tests, questioning the nurses and technicians. He chose the most aggressive treatments possible, overcoming pain and side effects, anything to have more time with his family.
Thoughts of his family spurred him on. David kept going by trying to survive until specific events such as Michael's bar mitzvah, Joel's bar mitzvah, and then, the final goal, Michael's 16th birthday on Sept. 23.
Beth told me on Thursday, "David did not want to be remembered for his cancer. Cancer was what he had, not what he was." On the drives to Hopkins we'd talk not about cancer treatments, but about how our various children were doing in school and what they would do over the summer.
Because, in the end, that's what matters.
The Digital Health Council (DHC) was created to serve as the collective public voice and provide a national public forum for the discussion of current and future issues relevant to digital and electronic marketing of healthcare products and services. I am honored to serve on the Council’s advisory board.
On October 13, 2011 at the Marketing to the Digital Consumer conference in Fairfield, NJ (hosted by DTC Perspectives), I participated on a panel with some of my fellow DHC advisory board members. My fellow panelists were:
Mark Bard, Co-founder of the Digital Health Coalition
Joan Mikardos, Senior Director, Digital Center of Excellence, Sanofi
Jeremy Shane, President and Chief Operating Officer, HealthCentral
Jay Goldman, VP Strategy, Klick
Gautam Gulati, Chief Medical Officer, Physicians Interactive Holdings
My opening comments commenced as follows:
In my opinion, social media must be viewed primarily as a way to advance public education, public health, adherence, compliance – all the things we say are really important – but always come second to selling product. We’re not going to advance the use of social media as long as we’re stuck in “sell” mode. When we move towards advancing the public health we begin to revive the mission of the pharmaceutical industry.
A transcribed version of the panel discussion has been created as a white paper and can be found here. No punches were pulled.
The National Federation of Independent Business is announcing a new report this morning that says the health insurance premium tax (levied on insurance plans) will cut as many as 249,000 jobs in the private sector by 2021 and cause a loss of $18 billion to $30 billion in sales. The drop would be a result of rising premium costs for employers and consumers. Just last week America's Health Insurance Plans released a similar report that said the tax would increase premiums by as much as 3.7 percent. The White House pushed back on the AHIP report and said it ignored "provisions that will increase transparency and bring costs down."
Read the NFIB report here.
Kansas Gov. Sam Brownback announced a major overhaul of the state’s Medicaid program today, which would put nearly all Medicaid recipients into private, managed-care plans. While low-income families are currently in such plans, elderly and disabled Kansans receive care through a fee-for-service system.
The state has drafted a request for proposals from private contractors willing to provide comprehensive health, mental health and long-term health care services at a fixed rate per person. State officials say they expect to select three vendors, who will compete for clients.
At a news conference today, Lt. Gov. Jeff Colyer, a physician and the administration’s point man on the Medicaid reform effort, predicted the changes could slow the growth in Medicaid spending by nearly one percent a year. That would save the state more than $350 million over the next five years and would save the federal government $500 million at the same time.
Read the full story here.
A new report in the Chicago Sun-Times raises an important question in the looming debate over generic interchangeability – the non-active ingredients in pharmaceuticals.
Just as there are bioeqivalance issues between innovator and generics – so too are there important differences between generics of the same molecule. And an important issue in both cases is the variety of fillers, colors and additives in these products that have nothing to do with the reason it is prescribed but can have unintended and undiagnosed consequences that can range from stomach pains to headaches to hyperactivity.
“It’s frequently overlooked by medical professionals and patients because, when we prescribe medication, we’re prescribing the active ingredient,” said Dr. John Saran, who specializes in internal medicine with Edward Medical Group. “(But) there may be 15 other things in there.”
While patients generally are asked if they have allergies to any medications, the allergy refers to the drug, not necessarily the fillers in it.
For instance, lactose is a common filler in pills and capsules. If a patient is lactose intolerant, the patient might believe they cannot tolerate the drug, when in fact, it is the filler that is causing cramping. Neither the doctor nor the patient may be aware that the pill contained lactose because, according to Saran, many times the additives and filler information are not included on the handout from the pharmacy.
According to the Sun-Times report, “Common additives … can lead to asthma attacks, anaphylaxis, runny nose and adversely affect children with learning disorders. Artificial sweeteners can affect those who have hyperactivity. Vegans may not want capsules because they are gelatin based. Even dye-free versions of medicine may still contain preservatives that can cause reactions. Children’s medications are especially a problem because they are flavored and colored to make them more palatable."
As if adverse event reporting wasn’t tough enough already.
According to BioCentury, Lisa Barclay has replaced Molly Muldoon as chief of staff for Commissioner Margaret Hamburg. Barclay, a partner at the law firm of Zuckerman Spaeder, worked at FDA in the 1990s in the Commissioner's Office of Policy.
The 4th Annual Risk Management and Drug Safety Summit is over. But the reverberations will be felt for some time to come.
After event chair (me) opened with a challenge for industry and regulators to step up to the challenge of “the responsibility of risk, the esteemed presenters were, to put it mildly – feisty.
(My complete opening comments on the “responsibility of risk” can be found here and many of the presentations from the Summit can be found here.)
The first keynote presenter at the summit was Janet Woodcock, who said that “advancing the science of safety is a shared effort.” She also shared the agency’s relief (shared by the majority of summiteers) that MedGuides shall now exist outside of a REMS context.
When MedGuides were in safety’s land. Let my REMS plans go.
Janet also made it clear that the outcomes data bases now available to the agency’s Sentinel program will not be used for comparative effectiveness purposes.
She then addressed the issue (also part of the PDUVA V discussion) or a benefit/risk assessment tool. Specifically, Janet laid out five “key considerations”: (1) analysis of condition, (2) unmet medical need, (3) clinical benefit, (4) risk and (5) risk management.
Sounds like a plan. Well – almost.
Dr. Woodcock then discussed a pilot program that most in the room (myself included) had never heard of before. Janet shared that CDER has begun a pilot program (with six unnamed NMEs) wherein the various sectors of review teams will fill out their own benefit/risk assessments (based on the five criteria mentioned above) to explain how they arrived at their relative positions. She didn’t mention whether or not these findings would be made public.
(Did somebody say, “transparency?”)
Janet also talked about the agency’s continuing and crucial struggle to advance PMI (patient medical information). The goal of CDER’s current initiative to create a one-pager for Rx products more akin to the Nutrition Facts Panel (aka, “the food label”) or an OTC “drug facts” box.
A noble effort – but the devil is certainly in the details. For example – would this document be progressive, or would existing products need to create them as well. If progressive, would this single sheet be part of the initial label negotiation process? And if retroactive – can the agency use its FDAAA directive labeling authority to create the page itself -- and, if so, based on what social science? Where would the boundaries be between product education and promotion? How would this document be distributed (hard copy, websites, social media, etc.)? Would generics use the same information and, if so, what about narrow therapeutic index products? Janet didn’t have all of the answers – but it’s certainly a provocative topic worth pursuing.
Next up was Sir Alasdair Breckenridge (Chairman, MHRA), who turned heads by saying that, “We need to stop talking about safety. Safety should be removed from our lexicon. We must focus on benefits and harms.”
Sir Alasdair also discussed the difficulties of regulating in an environment where EU- level directives add additional burdens to national level regulatory authority. Specifically, he shared that mandarins in Brussels have altered the definition of “adverse reaction.” The new definition includes:
“… noxious and unintended effects resulting not only from the authorized use of a medicinal product at normal doses, but also from medication errors and uses outside the terms of the marketing authorization, including misuse and abuse of the medicinal product.”
How, Sir Alasdair, asked, can any agency address adverse reactions based on medical errors and product abuse? Are they signals or noise?
Brussels sprouts. Alasdair doubts.
He also cited an interesting study (Golder, S., et al, PLoS Medicine, May 2011) on the issue of adverse effects data derived from RCTs as compared to observational studies. The conclusion of this paper is that:
“Empirical evidence indicates that there is no difference, on average, in the risk estimate of adverse effects of an intervention derived from meta-analyses of RCTs and meta-analyses of observational studies. This suggests that systemic reviews of adverse effects should not be restricted to specific study types.”
This opens up a big can of worms relative to the considered value of observation studies. But, as Alexandre Dumas said, “All generalizations are dangerous – even this one.”
Picking up on Sir Alasdair’s point about “benefits and harms,” Dr. Tim Franson (former regulatory chief at Eli Lilly & Co, current President of the USP Convention and an SVP at B&D Consulting) asked a smart question, should we be talking about risk at all – or about benefit risk? shared a timely quote from Edward Tenner’s treatise, Why Things Bite Back: Technology and the Revenge of Unintended Consequences, “Any technology powerful enough to improve life radically is also capable of abuse and prone to serious unanticipated side effects. Mix new technologies with the wide variations in how organizations and individuals behave and you often have a recipe for explosion.”
That passage deals with nuclear power. Discuss.
Dr. Franson concluded his remarks reminding the audience that, when it comes to global benefit/risk management, “We all share in the responsibility.”
Day Two of the summit featured a keynote address by John Lechleiter, Chairman, President and CEO of Eli Lilly and Co. who commented:
“We’d like to see the FDA adopt systematic, transparent Benefit/Risk assessment methods consistently across review divisions and the Office of Surveillance and Epidemiology. This would support more balanced regulatory decision-making … and enable the Agency to clearly communicate the rationale for its decisions to industry, providers and the public at large. I note here FDA’s support for medication adherence in 2011 – which we applaud. But a more balanced approach to communicating both the benefits and risks of a drug would also aid in the effort to improve adherence.
FDA should accelerate efforts to adopt and apply the best scientific methods and also incorporate the perspectives of affected patients – which can form the basis of consistent, transparent, reproducible decision-making.
Here are some things that I believe FDA could do right now to accelerate the benefit-risk agreement outlined in PDUFA:
Identify external benefit-risk experts as key consultants. FDA has acknowledged the need for systematic benefit-risk assessment tools … and has engaged external experts sporadically over the past several years. To accelerate progress, FDA should identify and pull together the leading academicians, clinicians, and thought leaders in the field now to augment their internal practical experiences in drug review.
Engage other major regulators in this effort. For example, FDA could advance discussions with EMA and other agencies to develop a harmonized approach to benefit risk assessment that would enrich decision-making and enable effective communications. This is important, as there’s potential for discord as regulators globally develop different tools and approaches. Adopting globally harmonized assessment of benefits and risk could alleviate regulatory confusion and uncertainty and help advance the public health.”
(John’s compete remarks can be found here.)
The theme of shared responsibility ran through the entire event. But talk is cheap. And if we all believe that to be true – then it must also instruct our rhetoric. For example – should ETASU (Elements to Assure Safe Use) be changed to ETASU (Elements to Assist Safe Use)? After all, (and to brutally frank here) nothing can ever assure safe use, but if we all assist in the endeavor, well, there’s a much higher chance for success.
Shared responsibility. If you can’t say it, you can’t do it.
Which brings us back to where we started – risk as a shared responsibility facilitated relationships built on trust. Trust between regulator and regulated. Trust between physician and patient. Trust enhances perception and, as the saying goes, perception is reality.
Generally, when you think about President Obama’s “core constituencies,” blue-collar unions are at or near the top of the list.
Consider, then, the following groups and their united position against the President’s plan to impose additional mandatory rebates on the pharmaceutical industry:
(And let’s call it what it is – a tax. More precisely, an excise tax imposed by Uncle Sam on drug sales – and not a single penny goes towards lower costs for a single patient. Not one. The cash goes into the general fund.)
· The International Brotherhood of Electrical Workers
· The International Brotherhood of Boilermakers
· The International Association of Police Associations
· Sheet Metal Workers’ International Association
· International Association of Fire Fighters
· International Association of Bridge, Structural, Ornamental, and Reinforcing Iron Workers
And the soliDarity is for a good reason – according to a new study by the Battelle Technology Partnership Practice, the President’s new tax on Medicare Part D would:
· Increase Medicare prescription drug premiums by up to 40%
· Increase annual out-of-pocket spending for almost 18 million seniors by as much as $208 annually
· Increase yearly total out-of-pocket drug costs for seniors by up to $3.7 billion.
The bottom line is the bottom line: As Yale Economist Fiona Scott Morton plainly states, “Applying the Medicaid rebate rule to Medicare Part D would likely result in higher prices for consumers in the private sector.”
That is not what the union movement signed on for when they supported the passage of the Affordable Care Act.
But wait, it gets worse.
The President’s tax a job killer. According to the Battelle Report, the tax could cause the elimination of between 130,000 and 260,000 jobs – many of them in the construction industry -- and hence the union revolt.
And, to add insult to injury, the President’s tax would also stifle life science innovation.
It’s hugely disappointing that the same man who (as a United States Senator) once said that …
“Realizing the promise of personalized medicine will require continued federal leadership and agency collaboration; expansion and acceleration of genomics research; a capable genomics workforce; incentives to encourage development of genomic tests and therapies; and greater attention to the quality of genetic tests, direct-to-consumer advertising and use of personal genomic information."
… is now advocating a policy that would result in precisely the opposite.
After speaking (during the State of the Union and a widely quoted op-ed in the Wall Street Journal) about the need for America to embrace innovation – President Obama is trying to make it more difficult, specifically when it comes to the desire to invest in pharmaceutical innovation – a sure bet under no circumstances.
If innovation is one of the key answers to our national economic recovery, then the President should abide by what he said, “Our economy is not a zero-sum game. Regulations do have costs; often, as a country, we have to make tough decisions about whether those costs are necessary. But what is clear is that we can strike the right balance. We can make our economy stronger and more competitive, while meeting our fundamental responsibilities to one another.”
As Harvard University health economist (and Obama healthcare advisor) David Cutler has noted: "Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost."
Let’s keep our eye on the prize. No, not ill-considered budget reduction on the backs of working Americans and seniors – the real prize: better access to smarter healthcare for all Americans. Rather than wasting time on spin, let’s redouble our efforts on innovation. Then, when we succeed through brainpower and teamwork (and, hopefully some civil bipartisanship), the circus surrounding the President’s tax will be but a footnote in the history of American healthcare.
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.
