Latest Drugwonks' Blog
Too often medical innovation against cancer has been both undervalued and even identified as a problem to be eradicated. That's one reason changes necessary to truly accelerate progress have preserved the status quo
Most people don't know that since 1990 new cancer therapies generated 43 million additional life years for people living with cancer. Those additional life years created $4.7 trillion in economic value. Every dollar we spend on new cancer medicines reduce spending on hospitals and doctors by 7 dollars. Such innovative treatments are less than two percent of total health care spending. They are the leading source of longer life, lower health care costs and greater economic growth.
Personalized therapies are the leading edge of this progress. Unfortunately it takes longer than ever for new medicines to get FDA approval. Health plans are requiring people to pay more and wait longer for innovations that save lives and reduce what consumers and they spend of health care. The reboot will be the result of patients, doctors and researcher knowing and sharing their genomic information about cancers using online apps and social networks. These virtual cure communities will define what treatments and mutations to study and use.
The American inventor Buckminster Fuller said: “You never change things by fighting the existing reality. To change something, build a new model that makes the existing model obsolete.”
We hope to demonstrate that the existing approach to medical innovation, while serving us well, is outdated and to support the building of new model that can lead to a world free from cancer.
The big news is the Supreme verdict that human genes cannot be patented.
And it was unanimous.
Is the decision good for patients – and what that does that mean? Does it mean prices (now driven by competition) will decrease? Does it mean that tests (being developed by multiple companies) will become more accurate? Does it mean payers will see the light?
Will “open genes” help or hinder innovation? Well, on the one hand, more companies (at least in theory) will be able to develop new products. But will lower profit margins, without patentable genes, be attractive to investors? Will competition drive tests that are “better” (more accurate, faster, etc.)? Or will it result in “me-too” tests at parity pricing. (Think “statins.”)
Will it increase patient access? Just as with generic drugs, competition will drive down costs. But by how much? Competition isn’t likely to result in plummeting prices. After all, process patents are still vey much alive.
Perhaps the most relevant question is whether or not the Court’s decision will in some way influence how payers view such tests. And then there's the issue of how they are regulated.
And, speaking of ever-greening -- there’s the interesting question of plant-derived medicines. Are natural compounds next?
This is not the end of the discussion. Nor is it the beginning of the end. It is, however, the end of the beginning.
Dr. Jerry Avorn (in his one-sided and self-congratulatory New York Times op-ed) misses a crucial piece of the argument on academic detailing – fairness.
Government detailing programs aren’t neutral. Just like detailing programs run by pharmaceutical companies, there is an inherent "interest."
According to the Agency for Health Research and Quality (AHRQ) the government’s top priority is ‘‘high volume’’ practices across 150 Metropolitan Statistical Areas. So, rather than focusing on offices with disproportionately high negative patient outcomes, the government is directing its efforts against those doctors who are high prescribers—which is a pretty good indicator about what government detailing is all about—decreasing cost rather than improving care.
When AHRQ’s ‘‘outreach experts’’ phone physicians to request appointments, they are allowed to entice physicians with the promise of free Continuing Medical Education (CME) credits. Would a pharmaceutical company be permitted to offer such an enticement? No.
Government-funded detailers must be held to the same high standards as industry representatives. This simple step would ensure providers are receiving accurate, unbiased information on the best treatment options available to improve patient care. When it comes to academic detailing, fairness is the proper yardstick. The general lack of rules or oversight stands in stark contrast to the extreme scrutiny with which industry “detailers” are subject in their interactions with physicians.
In Utah, the recently passed Information on Pharmaceutical Products Act makes the common sense point that without public scrutiny there is no guarantee that government-sponsored detailing will present information that is unbiased, peer-reviewed, and aligned with the existing evidence base. Bravo. Without clear guidelines, there is nothing to prevent government detailers from using their outreach to advocate for lower costs instead of educating for better care.
Per the new Health Affairs study on Consumer Directed Health Plans (CDHPs), here is the article.
As to the study design – there is no discussion of outcomes. As to the reason – is it unfair to say that the benefit model is being pushed for short-term economic benefit to … employers and payers?
Just sayin’.
For more on this, see The Cost/Quality Conundrum.
The study found that members in one large employer’s health plan filled significantly fewer prescriptions following a transition from a preferred-provider organization PPO plan to a consumer-directed health plan. The study followed more than 13,000 individuals for five years.
The article cites a Towers Watson employer survey finding that the percentage of employers offering only CDHPs to employees increased from 5% in 2007 to 8% in 2012 and is expected to rise to 13% in 2013.
Enhanced quality? Better outcomes? No, lower cost.
Regulations pertaining to the tax treatment of health reimbursement arrangements and HSAs as well as ACA’s excise tax on high-cost health plans that takes effect in 2018 “make CDHPs more attractive to employers because they may keep costs below the threshold that triggers the tax.”
Outpatient physician visits showed a significant decline and emergency department (ED) visits showed a significant increase for the study group over the four years following the switch to a CDHP. “When it comes to the initial decline in prescription drug use, this may be the result of fewer outpatient office visits or simply the introduction of the CDHP,” the article says. “However, it is unknown whether people only reduced unnecessary prescriptions or reduced the use of necessary pharmaceutical services. If the latter occurred, it may explain the longer-term increase in ED use.”
“The increase in ED use might stem from the long-term implications of reductions in physician office visits and prescription drug use after the CDHP was implemented. Fewer physician office visits may lead to the writing of fewer prescriptions, which could in turn mean that individuals with chronic conditions are less adherent to recommended medication therapy,” the article says. However, the article adds that more study is needed to determine the cause for the increase in ED visits.
It will be interesting to analyze the paper to see the intricacies of the benefit structure and whether that played a role in the drops in outpatient office visits and RX use and increased ED visits.
The FDA's Endocrinologic and Metabolic Drugs and Drug Safety and Risk Management Advisory Committees voted 20 of 26 to recommend removing or modifying rosiglitazone's highly restrictive label and distribution system. Five voted to keep the product's risk evaluation and mitigation strategy (REMS) as it is now.
"I find no substantial evidence or information that rosiglitazone is unsafe," Arthur Moss, MD, cardiology professor at the University of Rochester School of Medicine in New York, said, adding its ability to lower blood sugar without causing hypoglycemia proves it could be of use.
Sanjay Kaul, MD, director of cardiovascular fellowship training at Cedars-Sinai Medical Center in Los Angeles, said there's not enough evidence to support or blame rosiglitazone's safety and therefore physicians should be able to choose it if they desire.
Why is this important so far after the fact?
First of all, it’s important because cardiologists need to have faith in the products that they have in their armamentarium.
Second it’s important to set the record/RECORD straight. After the 2010 media circus, the Congressional carnival, and the endless posturing from the Cleveland metropolitan area – yesterday’s adcomm vindicated both the process and the people at the FDA. In 2010 the pressure was withering. In 2013 there was a degree of redemption.
(As Mark Twain quipped, “The rule is perfect: in all matters of opinion our adversaries are insane.”)
Thirdly, the adcomm discussion and vote is important relative to the FDA’s evolving views on the future development and review of diabetes medicines.
Let me embrace thee, sour adversity, for wise men say it is the wisest course.
-- William Shakespeare
A Portland, Maine, physician announced on April 1 that he would cut the middle man and deal directly with his patients, no longer accepting insurance in any form.
"I’ve been able to cut my prices in half because my overhead will be so much less," Dr. Michael Ciampi told the Bangor Daily News. Before, Ciampi charged an existing patient $160 for an office visit addressing one or more complicated health problems. Now, he charges $75.
Ciampi lost a few hundred of his 2,000 patients who had insurance and didn't want to deal with the hassle of paperwork for reimbursement, but he expects to make up the loss by attracting the self-employed, the young and others without insurance or with prohibitively high deductibles.
Read more.
Anna Gorman of the L.A. Times on the drive to enroll young Americans on insurance plans:
Arsine Sargsyan is 23 years old, healthy and uninsured. She chooses to forgo coverage for one simple reason: "I never get sick."
Despite her reluctance, Sargsyan is exactly the type of person insurance plans, states and the federal government are counting on to make health reform work.
As the clock ticks toward the 2014 launch of the Affordable Care Act, health leaders across the nation are embarking on a tough task: persuading young adults like Sargsyan to enroll. Their participation will be critical to balance out older, sicker patients more likely to sign up for health insurance as soon as they are able.
Read more.
Tom Miller of the American Enterprise Institute on the ACA’s health flexible spending account provisions negative impact on older Americans:
The central provisions of the Affordable Care Act require younger and healthier Americans to buy insurance policies that will, in essence, subsidize the healthcare of older and sicker Americans. But one of Obamacare's hidden taxes — a new limit on contributions to health flexible spending accounts, or FSAs — will hit older and chronically ill individuals hardest.
Starting this year, the healthcare law imposes a $2,500 annual cap on an individual's contribution to an FSA that is part of an employer's "cafeteria" benefits plan. Such contributions, diverted directly from one's paycheck, are not subject to federal income and payroll taxes. The money in an FSA can then be used to pay for qualified medical expenses such as deductibles, co-insurance and co-payments, as well as services not covered by insurance.
Read more.
The percent and absolute number of colonoscopies and other procedures performed on colon cancer patients in hospitals has declined. Also, the length of hospital stays and number of in hospital deaths have fallen by 30 percent. Indeed, colon cancer treatment costs in America are growing more slowly than in Europe.
Yet, over the past 30 years, death from colon cancer has declined faster in America than in Europe. Nearly 65 percent of all Americans will live 5 years or longer with colon cancer compared to 59 percent of Europeans.
The difference? Americans get screened and treated faster with newer medicines and diagnostics. The way to saving money and lives is faster innovation, not more regulation.
Yesterday I had the pleasure of participating on the keynote panel at the World Pharma Congress. My fellow panelists were Karim Dabbagh (Executive Director and Head, External R&D and Innovation Research Units, Pfizer Worldwide R&D) and Glen Gaulton (Professor, Pathology and Laboratory Medicine, Executive Vice Dean and Chief Scientific Officer, Perelman School of Medicine, University of Pennsylvania). The panel was expertly chaired by Rick Turner (PGCE Senior Scientific Director, Clinical Communications, Quintiles and Fellow, Society of Behavioral Medicine).
Our topic was, “Advancing Pharma R&D through Communications and Collaboration.” It was an exciting 90 minutes.
The issue of out-sourcing R&D isn’t new – but it’s mighty contentious. And it’s the new reality of drug development. But, if we are to learn any lesson from the CRO experience, it’s that while we say “partnership,” the danger is that it devolves into a vendor-like relationship. It’s the Golden Rule. He who has the gold makes the rules. Will that be acceptable to high-level, big ego Ivy Hall-ers?
And then there’s the issue of academic priorities, specifically tenure. Does industry funding carry the same weight as NIH grants when it comes to advancing a university career? Not at present. That will have to change.
Will university researchers pursue their programs with the same time-driven focus as their pharma counterparts? After all, there aren’t any quarterly analyst calls to worry about. And, as already mentioned, if the funding is viewed as secondary to “tenure-track” funding, how will the work be prioritized?
And then there’s the IP question. According to both Dabbagh and Gaulton, IP negotiations are generally smooth. That’s good news – but there’s a lot of road ahead on the R&D partnership superhighway.
CROs, at least know what needs to be done. Researchers (whether inside a company or a university) don’t. That’s why it’s called research. One issue that arose is the need for total sharing and absolute transparency between industry and academic partners on all levels of the engagement. Is this happening? Sometimes. The good news is that when it does occur – the relationship (and results) are more positive.
But talk with CROs and you’ll hear their frustration over being instructed what to do by their pharma masters. (In most cases their former employers.) Fortunately, that too is changing as the urgency of new and innovative clinical trial protocols and programs becomes self-evident.
Need drives change. Just as CROs are now really partnering with pharma to drive the development of personalized medicine, so too must academic researchers and their industry partners collaborate on the continued evolution of pharmaceutical innovation. It will take discipline and focus. It will be risky. And it will take will. There is a confluence of interest.
To borrow some FDA parlance, will industry/academic partnerships result in expedited pathways in drug development? It must – because if it is only viewed as a cost-saving mechanism it will fail.
“Change is not required. Survival is not mandatory.”
-- W. Edwards Deming
BioCentury reports that the FDA is seeking comments on approaches to make available to non-FDA experts and other interested parties de-identified and masked subject level clinical trial data derived from regulatory applications. According to the notice published in the Federal Register, the agency said the proposal is driven by its "desire to improve the product development process," noting that data may be useful "to address key hurdles in drug development" like validating new trial endpoints or clarifying how products work in different diseases. FDA also noted that pooled data can also be used to identify and analyze safety issues.
FDA is seeking input on strategies to minimize the ability to identify specific products, including making available certain data from a random sample of patients or pooling data for a number of products within a product class. The agency is also seeking input on how to mask data and limitations to making the masked data available, as well as input on de-identifying data. Additionally, FDA is seeking input on situations where disclosing masked data would be the most useful to advance public health. FDA said its proposal does not address making available unmasked safety and efficacy data that can be linked to a specific, identified application, including full study reports. The agency also said it "will not make available business-related confidential commercial information contained in product applications." Comments are due Aug. 3.
By the end of June, EMA is slated to publish a draft policy for public consultation providing more details on its plan to proactively release patient-level clinical data for approved drugs.
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.
