Latest Drugwonks' Blog
In case you missed the FDA’s Viewpoint commentary in JAMA, here it is:
Addressing Prescription Opioid Overdose: Data Support a Comprehensive Policy Approach
And it’s not about banning Zohydro:
“The risk in singling out a single drug like Zohydro ER in this complex, multidrug epidemic is that resulting policy is unlikely to have an effect on the underlying causes—the abuse of multiple opioids and other drugs and inappropriate prescribing.”
The first sentence of the concluding paragraph says it all, “The problem of opioid overdose demands well-informed policies.”
Not knee-jerk. Not political. Not bowing to threats. Well-informed policies.
Attention must be paid.
Leading medical innovation think tank claims CBS is ‘dead wrong’
New York, NY (PRWEB) October 08, 2014
The Center for Medicine in the Public Interest (CMPI.org– a non-profit research organization specializing in promoting medical innovation) criticized CBS’ “60 Minutes” segment on cancer treatment prices as not just distorted but ‘dead wrong.’ “‘60 Minutes’ ignores the contribution new medicines make in reducing health care costs, improving wellbeing and saving lives,” said Robert Goldberg, Vice President for Research of CMPI. “Featuring physicians that argue that treatment prices are too high does not change the fact that the cost of cancer drugs are about 12 percent of what health insurers spend on cancer or that new cancer drugs actually save health insurers money. Yet insurers are increasing oral therapy cost-sharing requirements to actively encourage patients to use infusible products.
While a Milliman study found that shifting to co-insurance would only add about $2 per member per month in private health plans, 60 Minutes never discusses this solution. Instead, 60 Minutes remains silent about the cost shifting and has never advocated for co-pay reforms that could relieve the burden on patients.
Spending on cancer treatments has climbed from $24 billion in 2004 to about $40 billion today. But such treatments represent only 0.6 percent of U.S. healthcare spending, and that proportion has been fairly consistent for the last several decades.
Such medical innovations were largely responsible for a 40 percent increase in cancer survivors (from 9.8 million to 13.6 million) since 2004. Since 2004 the use of new therapies saved $188 billion on hospitalizations, and avoided nearly $100 billion in lost productivity each year. Since 1990, new cancer drugs have helped generate 51 additional years of life, worth nearly $5 trillion.
A doubling in the use of new-targeted therapies will raise the amount spent on medicines as a percentage of total health care spending. But that increase must always be compared to what would be spent on healthcare, disability, unemployment and rehabilitation in the absence of medical innovation.
Peter Pitts, the President and Co-Founder of CMPI noted, “New medicines almost always reduce the cost of living longer and healthier lives and increase the value of such improvements. It is disappointing that “60 Minutes” failed to investigate why some health plans have deliberately made these innovations unaffordable by requiring patients to pay up to half their costs. And it is disheartening to know that doctors are more interested in attacking innovators than in defending patients from such cost shifting.”
Pitts also remarked, “We are happy to talk about the prices of cancer therapies, but only in the context of value and the rising cost and prices of other healthcare services that constitute a substantially bigger portion of total spending. In addition, the discussion should only be in the context of what can and should be done to cut the cost of developing new therapies through smarter, faster regulation.”
CMPI has a website—valueofmedicalinnovation.org—that provides a balanced view of the role new medicines play in our lives.
The Center for Medicine in the Public Interest is honored to join the US Pain Foundation and other patient-centered organizations in offering our support to the FDA in general and Commissioner Hamburg specifically for savvy and strategic management of pain medicine regulation.
Why now? Well for starters because of the unfair, unbalanced, ad hominem and just plain erroneous attacks on the agency’s efforts.
It’s all spelled out in this letter of support just sent to HHS Secretary Burwell. It’s designed for impact.
Have a look and please share. This issue is too important to allow the lunatic fringe to drive the agenda.
BioCentury reports that the EMA's management board unanimously adopted its clinical data transparency policy, to be introduced in two phases starting Jan. 1, 2015. In the first phase, EMA will publish clinical reports supporting MAA submissions while excluding individual patient level data. The policy will apply to any MAA submitted after Jan. 1, 2015.The agency noted that as a practical matter, this would mean the first data for newly approved medicines would become available in 18 months, allowing for review time and approval by the European Commission. For line extensions and extended indications of approved drugs, EMA will publish clinical data for applications submitted as of July 1, 2015. EMA said the second phase, disclosure of individual patient level data, will follow a consultation with stakeholders to determine how such data can be made available in compliance with privacy and data protection laws.
The agency said commercially confidential information will be redacted at its discretion with input from sponsors, noting "the starting point of the redaction principles is that clinical reports do not, in general, contain CCI." After reviewing stakeholder concerns that clinical data would only be accessible by viewing it on a screen, EMA amended the policy to allow identified users to download, save and print trial data for academic and non-commercial research purposes. The policy provides two permission routes for access, one for "general information purposes" by individuals and another for "academic and non-commercial research purposes." EMA also published a document addressing frequently asked questions relating to the policy.Speaking of opioids, here are three interesting items from today’s edition of DIA Daily. One thing, however, that remains absent in the debate over how to address the issue is the Hamburg Manifesto – enhanced physician education. Now it's time for the DEA to step up to the plate and mandate specific education as a must-have for prescribing rights.
So far, alas, only silence from the DEA on this topic. This must change.
Pharma Association CEO Offers Possible Reforms To Curb Prescription Drug Abuse.
Mark Merritt, president and CEO of the Pharmaceutical Care Management Association, writes in the The Hill (10/2, Merritt) about options in trying to abate prescription drug abuse issue in the US. Merritt offers “two basic reforms”: first, a “safe pharmacy” or “lock-in” policy in Medicare in which “the small segment of patients who are at risk of abusing opioids would choose – along with their health plan – a single, convenient pharmacy to fill their prescriptions for controlled substances.” This eliminates the possibility of “drugstore shopping,” or “the practice of filling prescriptions for controlled substances...at multiple drugstores to avoid detection.” A recent HHS OIG report called for such an approach. A second option would be to “close the loophole in Medicare that prevents Part D plans from suspending payments to pharmacies suspected of fraud or diversion.” A second HHS OIG report outlined the benefits of this potential reform.
Neurologists Urge Caution With Opioids For Non-Cancer Use.
The Los Angeles Times (10/2, Healy) reports that patients on opioid painkillers for chronic pain unrelated to cancer, such as headaches, may face a situation where the risks of taking the medications outweigh the benefits. The paper notes that patients “are more likely to risk overdose, addiction and a range of debilitating side effects than they are to improve their ability to function,” citing the American Academy of Neurologists. In a new position statement released Wednesday, the group warned that even for patients “who do appear to benefit from opioid narcotics,” doctors “who prescribe these drugs should be diligent in tracking a patient’s dose increases” and should insist as a condition of continued use “that opioids are improving a patient’s function.”
Abuse-Resistant Version Of Controversial Painkiller Unveiled.
The Wall Street Journal (10/2, Burton, Subscription Publication) reports, Zogenix Inc., the maker of the controversial opioid painkiller Zohydro ER (hydrocodone bitartrate), disclosed Wednesday it filed an application with the FDA for a modified version of the medication that it says would be harder to abuse. According to the firm, the new version will be a capsule containing a gel, making it more difficult to snort or inject. Still, some doctors noted that it could be abused by addicts, who could choose to orally ingest the contents.
To the editor:
Per Detailing Financial Links of Doctors and Drug Makers (NYT, October 1, 2014), collaborations between physicians and industry are fundamental to advancing medicine. Without in any way diminishing the governmental and non-profit agencies that support research, it is the partnership between physicians and industry that has created many, if not most, of the major medical breakthroughs that have reduced the rates of death and other serious outcomes in recent years – as any literature search of major medical journals will quickly confirm.
Conducting clinical research has become so rigorous and sophisticated that those who serve as consultants and investigators to industry recognize it as a major commitment or even a primary career path. In addition, as studies are completed, physician researchers add to their professional commitment by becoming the teachers of this new information.
Critics of these collaborations see compensation of physicians as evidence of undue influence on medical practice. But doctors, like all professionals, should be paid fairly for their time and work. The truth is that physicians who are busy with these activities are not as well rewarded as their fellow specialists in full-time clinical practice. Adding to this remunerative divergence is that a key part of research, writing articles for publication, takes weeks of work -- often unpaid so as to avoid any suggestion of bias.
The Sunshine Act will create troubling misconceptions for and about physicians. Payments reported for physicians by industry will likely include funding they didn’t personally receive nor will they take into account costs incurred by these physicians in paying their own staff and covering overhead expenses. Doctors involved in industry-supported research and education may easily get discouraged and frustrated explaining these complexities to an audience already biased and sated by sensationalistic media reports of physicians “on the take.”
And yet the Sunshine Act, paradoxically, could have a positive effect. Despite the near impossibility of reliably interpreting all the reported data, this information might well serve as a yardstick of cooperative achievement --identifying those physicians at the forefront of medical innovation.
Peter J. Pitts
Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest
I recently had the pleasure of meeting with Captain Valerie Jensen, USPHS, who has led the FDA’s efforts to tackle the problem of drug shortages in the US over the past several years. (She is officially the Associate Director of the FDA’s Drug Shortage program.)
Alas, as the tabloid press reminds us, “if it bleeds, it leads.” The media frenzy surrounding drug shortages has vanished. Why? Because, due in no small measure to efforts from the FDA, the problem is being successfully addressed and ameliorated. Unfortunately, that’s not news – but it should be. Here are some interesting points.
The FDA’s strategy in addressing the issue is to enlarge and empower a drug shortage ecosystem. Rather than seeking more Federal dollars (a fool’s errand), the FDA undertook to combine the resources of the constituent players, manufacturers, hospitals, and large-scale purchasers such as GPOs. Combined with appropriate and savvy use of enforcement discretion, the FDA has taken leadership of the drug shortage ecosystem and is driving a sustainable solution.
The drug shortage ecosystem relies on more than just FDASIA-mandated notification requirements –it’s built on trust. Specifically, that the FDA wants to work with manufacturers to solve the problem rather than simply whack them with 483s. The agency is showing more regulatory flexibility when resolving manufacturing and quality issues.
Curiously, a new cause of shortages has arisen – poor corporate planning. Shortages, for example, in Sodium Chloride IV kits have hit the agency’s radar screen. The problem isn’t due to manufacturing quality, but rather lack of inventory. Hello?
Smart business projections must also be a part of the drug shortage ecosystem.
Speaking of business practices, there remains the 800-pound gorilla of the drug shortage dilemma -- artificially low prices are a major causational problem. And that’s not something the FDA can help fix.
It’s time for our lawmakers to revisit the legislative solution proposed in Senator Orrin Hatch’s Patient Access to Drugs in Shortage Act. There are three key codicils:
1. Price Stability
The Hatch language would change the Medicare reimbursement rate for generic injectable products with 4 or fewer active manufacturers from ASP + 6% to Wholesale Acquisition Cost in order to achieve market price stability.
2. Medicaid/340B Rebate Exemption
Exempt generic injectable products with 4 or fewer active manufacturers from Medicaid rebates and 340B discounts in order to achieve market price stability.
3. Extended Exclusivity
Manufacturers who hold an approved application for a drug that would mitigate a shortage can extend by 5 years any period of exclusivity, even if the drug is eventually moved from drug shortage designation.
So, kudos to Captain Val and her team at the FDA.
Now it’s time to enlarge the eco-system via timely and targeted legislation.
The U.S. Pharmacopeial Convention (USP) endorsed a draft World Health Organization (WHO) proposal to create a unique suffix for biologic drugs, including biosimilars.
A WHO working group proposed a voluntary scheme to create unique identification codes, called "biological qualifiers," that would be distinct from international non-proprietary names (INN). The BQs would be random four-letter codes that would be linked in a WHO-maintained database to the product's INN, trade name, name and address of the manufacturer, and regulatory status. According to the WHO, the scheme could facilitate decision making about substitution and interchangeability and help regulators and physicians track patients' responses to products with the same INN.
In comments submitted to WHO, USP suggested that the BQ system be applied to all biologics, not just biosimilars. BQ codes could be assigned retrospectively.
The endorsement comes as FDA is finalizing its biosimilars naming policy in anticipation of regulatory decisions on at least two pending biosimilar applications. Sandoz, a unit of Novartis AG (NYSE:NVS; SIX:NOVN), has submitted an application for a biosimilar version of Amgen's Neupogen filgrastim and biosimilar version of Remicade infliximab, a mAb marketed by Johnson & Johnson (NYSE:JNJ) and Merck & Co. Inc. (NYSE:MRK)
Superb analysis of the recent NICE draft technology appraisal against the use of Abraxane from the folks at Context Matters. Well worth the read.
NICE: Draft Guidance in Context
In early September, the UK’s National Institute for Health and Care Excellence (NICE) released a draft technology appraisal recommending against the use of Celgene’s Abraxane (nab-paclitaxel) for Pancreatic Cancer. NICE often releases drafts prior to their final reports, giving pharmaceutical companies and other stakeholders the opportunity to offer further information, evidence, and comments. This preliminary decision inspired a number of news articles—PMLiVE, Fierce Pharma, The Telegraph, PharmaTimes, and other news sources all added to the headlines about the agency’s initial report.
Although each of these articles states at least once that the appraisal is draft guidance, not a final decision, it is important to emphasize that NICE’s draft decisions are subject to change. This past May, for example, another Celgene drug, Revlimid (lenalidomide), received a negative draft decision for the treatment of myelodysplastic syndromes. Months later, in August, NICE released a new draft with a positive decision based on additional information provided by Celgene. In March, NICE issued a draft guidance against Alexion’s Soliris (eculizumab), but requested more information justifying the cost. At the beginning of September, NICE, satisfied with Alexion’s addendum, released new positive draft guidance. These three instances alone occurred in 2014; there are more in NICE’s history.
Publishing draft guidance brings up an important point about NICE in particular and HTA agencies in general. NICE’s efforts toward transparency are admirable, but decisions can change until the final guidance is released. Each of the above news articles represents a single data point—a single update in an ongoing process toward a final decision. Only the final decision is binding. Although following the news is certainly valuable, these brief updates do not tell the entire story. Furthermore, though influential, NICE’s decisions do not determine how other countries will make their decisions. NICE is a high-profile agency, but there are others that provide insight into global HTA and reimbursement policies and decisions.
Draft Guidance from NICE: Not the Last and Final Word
Indeed, NICE issued negative draft guidance about Abraxane; but the story does not end there. Abraxane has also been reviewed for Breast Cancer and Ovarian Cancer, and it has been reviewed multiple times by other agencies: HAS in France, PBAC in Australia, CCO in Canada, and SMC in Scotland have all reviewed the drug for at least one indication.
The Snapshot, generated here by the Context Matters Reimbursement Risk Tracker (RRT) database platform, shows that Pancreatic Cancer reviews currently comprise only a small percentage of all HTA reviews for Abraxane. The news about a NICE draft provides manufacturers and stakeholders an opportunity to present additional data and commentary, but it does not include the necessary and complete context to understand the market access status of the drug around the world, or for other disease conditions. Informed action requires more context than that included in the drafts alone.
Furthermore, the draft does not represent all that NICE has decided about Abraxane in the past. NICE recommended Abraxane for the treatment of Ovarian Cancer in 2005; but in 2006, the agency issued a negative decision for its use in Breast Cancer.
The Scottish Medicines Consortium (SMC) has already reviewed Abraxane for Pancreatic Cancer. The agency issued a negative recommendation, finding it more efficacious/effective, but less cost effective than its comparators.
To be sure, draft guidance reviews from NICE provide insight and are valuable issuances to read once published in the news. However, they do not guarantee future and final outcome, nor do they tell the whole story.
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.
