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Always listen to the experts. They’ll tell you what can’t be done and why. Then they do it. – Robert Heinlein
On Tuesday the Center for Medicine in the Public Interest (www.cmpi.org ) hosted a Capitol Hill panel entitled, “PDUFA without the Politics.” I was the moderator and was honored to be joined by a panel of peerless nonpareils that included Vincent J. Ventimiglia, Jr (Former Assistant Secretary for Legislation at the US Department of Health and Human Services), Paul T. Kim (Former Deputy Staff Director for health policy for Senator Edward M. Kennedy), Michele J. Orza (Former Assistant Director of the Health Care Team at the Government Accountability Office), and Tim Franson (Former Vice President, Global Regulatory Affairs, Eli Lilly & Co., President, USP Convention). The Honorable Michael C. Burgess, M.D. (Vice-Chairman, Subcommittee on Health, U.S. House of Representatives) offered remarks as well.
The participants were pithy, poetic, and pragmatic.
We will shortly post videos of all the aforementioned presenters. In the meantime, here’s a short news story from Drug Information Daily:
Lawmakers Plan to Use PDUFA V Bill to Ease Conflict Rules for Advisory
Panels
Lawmakers plan to attach a bill easing financial conflict rules for FDA advisory panel members to legislation reauthorizing the Prescription Drug User Fee Act (PDUFA), a congressional leader says.
Rep. Michael Burgess (R-Texas) introduced H.R. 3206 last month and said Tuesday it would be attached to the measure authorizing PDUFA V, yet to be introduced to Congress. H.R. 3206 would eliminate the existing financial conflict rules for panelists, opening the door for committee members with greater expertise on the products they discuss.
Burgess, vice chair of the House Energy and Commerce Health Subcommittee, offered similar legislation during consideration of PDUFA IV in 2007, but it was voted down along party lines. With his Republican party now in control of the House, he may have an easier time gaining approval of the bill.
Speaking at a PDUFA V briefing for Capitol Hill staffers hosted by the Center for Medicine in the Public Interest (CMPI), Burgess said there are about 10 strong candidates for legislation to tack on to PDUFA V. One possibility is a bill addressing the issue of drug shortages, he said.
Another likely candidate is a bill to increase incentives for antibiotic makers, according to Foley Hoag lobbyist Paul Kim.
Proactive Lawmakers
Also at the briefing, Peter Pitts, former FDA associate commissioner and president of CMPI, urged Congress to be more proactive in changing the technical letter the FDA and drugmakers agreed to during PDUFA negotiations earlier this year. Specifically, Pitts called for more programs and agency action to address transparency and striking a better risk-benefit balance in product reviews.
Following these recommendations might mean altering the package the FDA and industry ratified, which has only happened once in the previous four PDUFA bills.
Recently, David Wheadon, PhRMA's senior vice president for scientific and regulatory affairs, said the package includes a "robust set of programs" to provide an objective measure of risk-benefit analysis at the agency.
http://www.cbsnews.com/8301-18560_162-57330802/hard-times-generation-families-living-in-cars/
EC proposes new research program
The European Commission proposed a new EU research and innovation program for 2014-20 called Horizon 2020, which would have a budget of EUR 80 billion ($107 billion). The proposal includes allocations of EUR 24.6 billion ($32.8 billion) for science research in Europe, including an increase in funding of 77% to EUR 13.2 billion ($17.6 billion) for the European Research Council (ERC); EUR $13.7 billion ($18.2 billion) for industrial technology development and small and medium-sized enterprises; and EUR 31.8 billion ($42.4 billion) to address societal concerns in areas such as climate change, energy and resource scarcity, and health and aging.
The EC said Horizon 2020 will have a simpler set of rules and procedures for obtaining funding than those of previous research programs, with the goal of reducing time to funding following a grant application by about 100 days. The European Parliament and EU Council will now review the proposal, with the goal of adopting it by year end 2013. The current research program, Framework Programme 7 (FP7), has a budget of EUR 54 billion and runs from 2007-13.
Here are a few key excerpts:
But above all, Mr. Lechleiter explains, "There's no better investment that we can make than in biomedical research and in our health. This is not something that we're trying to steal away from someone else. This is not a nascent industry." Pounding his desk on each word—"America leads the world, okay?"
"I believe this will be the biomedical century," he continues. "We'll look back a hundred years from now and say the 20th century was the century of chemistry and physics, and the 21st century was the century of biomedicine."
Mr. Lechleiter adds that "The challenge or the opportunity we have is that never before has the science and our knowledge base been riper for exploitation." For most of the pharmaceutical industry's existence—since Civil War veteran Col. Eli Lilly began to improve on the patent medicines of the day—"it was akin to feeling your way around a dark room and trying to make sense of what's what. Suddenly the lights are on and we can see, aha: In a cell, this pathway and that pathway both contribute to, say, tumor formation."
Not only is there an ongoing revolution in genomics and systems biology, Mr. Lechleiter continues, we increasingly have the tools to make use of this basic research and commercialize it. "A process that used to take years and years and rely too much on serendipity and conjecture can now be accomplished in a period of time that looks closer to months and months." Researchers are more "mission driven and deliberate" and, with a biological target, can "come up with a viable clinical candidate, something that we could hope to take into human testing" faster and with more confidence than ever before.
Read the full piece here.
To help shape thinking in this area, Beijing think tank the Cathay Institute for Public Affairs invited me to discuss with officials and experts how the system works in Britain at a symposium and workshop in Beijing, with additional comment from local policymakers and experts on general practice reform.
Britain presents a case study of how not to organise a health system. Since britain nationalised healthcare in 1947, subsequent governments have struggled to improve productivity, contain costs and improve the quality of care. Up until 1991, the system was mainly managed through central targets and control, with a chain of command going right up to the minister's office in Whitehall. By the late 80s, this system was beginning to look rather tired.
As part of the Thatcher government reforms, competition was introduced into the health service, in the form of an internal market. Providers and purchasers were split for the first time, and GPs were given the power and budget to buy care on behalf of their patients from any NHS provider. The aim was to use their market power to drive down costs and promote innovation. While this system achieved some success in reducing patient waiting times, Tony Blair’s government on election in 1997 decreed that this created a two tier system in which some received faster treatment than others.
This nascent market was therefore abolished and replaced with the old command and control system based on centrally-mandated targets. But by 2004, it was clear that this system was failing to manage an increasingly complex and massive health system, so the government reintroduced a diluted form of the internal market it had abolished, wherein GPs could form voluntary groups to commission care on behalf of patients. This time the budgets were notional instead of real, and any notional savings would be reimbursed by the ministry for spending on developing innovative services. Not surprisingly, few doctors found they had the time to engage with this new bureaucracy, so it has remained something of a sideshow to the main modus operandum of the NHS which is targets and central dictat.
Finally, the new government of David Cameron proposes reinvigorating the internal market by making it compulsory for GPs to commission care on behalf of their patients, and extending it to the majority of areas of care. This met with fierce opposition from opposing parliamentarians, as well as doctors unions and most of the medical profession, who fear that moves to a more market based system might undermine their not inconsiderable pay and generous employment conditions (as well as having ideological objections to the involvement of markets in healthcare). Pro reformers worry that the new bureaucratic apparatus required to manage this quasi market will work against physician autonomy, and could act as conduit for central government to reassert control - health civil servants are notorious jealous of their status as guardians of such a significant part of the body politic.
The key lesson for China, then, is that once they embark down a route in which central government is the primary provider and guarantor of care, it is very difficult to inject market force to tackle the problems that always arise from monopolies - interest groups, once entrenched, are very difficult to tackle. As a result of these failings, GP care in the UK is no way comparable to that in the US, as they have little incentive to develop the doctor patient relationship beyond responding to a narrow set of clinical and waiting time targets.
While things are bad in the UK, in China they are several orders worse. At our workshop Qin Jiangmei from the Ministry of Health outlined the major problems with the GP system, including the poor quality of doctors and service providers, and the fact that patients (especially in rural areas) normally have to pay out-of-pocket to poorly trained GPs. And as GPs derive most their income from sellling medicine or hosptial procedures, a visit to the doctor for even a mild ailment can end up with a huge bill as a result of over-prescribing. As a result, the majority of Chinese simply avoid the healthcare system altogether.
Professor Gu Yuan of the General Practictioner Association of Chinese Medical Association outllined her vision of the future system, which included training 300,000 extra doctors by 2020, and creating a new system of contract between patient and doctor that would inject some accountablity into the system. She also pointed out that General Practice is a very unpopular career choice for medical doctors, the majority of whom aim for a clinical specialism. This is hardly surprising when General Practitioners earn less than average civil servants. As a result, GPs are few and far between, particularly in rural areas, and they are often only in possession of a sketchy knowledge of medicine. In order to increase their wages to something reasonable, GPs routinely overprescribe, and make unnecessary use of expensive diagnostics (when for instance the patient may only have a cold). This is exacerbated by the fact that doctors receive a large proportion of their salary from profits they generate for the hospital, which leads to overprescribing, and not enough focus on preventative care and basic diagnostics (such as checking blood pressure).
Prof Wang Zhen on the Institute of Economics noted the problems inherent in expanding a system of coverage to a rapidly ageing population, in the face of rapidly changing technology and medical knowledge. The NHS experience shows that state-monopolies are not well-placed to foster innovation either in managerial techniques or the use of new medical technologies. China therefore needs to embrace choice and competition in its design of coverage systems if it is to avoid making the same mistakes as Britain. He noted that the government’s role should be restricted to supervision or the provision of additional services that cannot be provided by the market.
Excellent article in the always excellent RX Compliance Report on the Europe’s continuing debate over the dissemination of healthcare information.
European Commission proposal on patient information draws divergent views
The European Commission’s (EC) revised proposals on information to patients, released last month, drew divergent assessments across the Atlantic. The European Federation of Pharmaceutical Industries and Associations (EFPIA), which represents research-based pharma companies operating in Europe, cautiously welcomed the amendments, urging all member states to adopt a “pragmatic approach” to address what it calls “the existing inequality” of access to quality data. “The revised proposals put rights, interests and safety of patients first,” insisted John Dalli, European Commissioner for health and consumer policy.
However, former FDA Commissioner for External Relations, Peter Pitts, spoke for many on this side of the Atlantic when he said the proposals appear to slow what had been a movement on the part of the EC to liberalize the industry’s ability to provide information about prescription drugs to patients. “That effort was on the ‘slow track’ before last month’s announcement,” says Pitts, who heads the Center for Medicine in the Public Interest in New York. “Now it’s on the ‘super slow track.”
International compliance expert, Paul Woods, says he is hopeful that the revised proposal will eventually be enacted in a form that will enable citizens all across Europe to have equal access to high quality non-promotional information about their prescription medicines. “The outcome, however, remains uncertain,” he says.
The complete RX Compliance Report article can be found here.
And a happy Thanksgiving to all.
By now it should be clear to regulated healthcare communicators that delaying robust entry into the world of social media due to lack of FDA guidance is an empty excuse. And, as more and more people turn to social media as their first and primary portal for healthcare information, absence from the playing field isn’t only a bad business decision – it’s irresponsible.
Regulated healthcare industry must participate in social media –not because of its potency as a marketing vehicle – but because it’s the right thing to do. That being said, here are 11 principles that must serve as the basic substrate of regulated social media participation. (Why eleven? Because, in the immortal words of Spinal Tap’s lead guitarist, Nigel Tufnel, “It’s one louder.”)
1. We engage in social media to help improve the lives of patients and advance the public health of our nation.
2. We will thoughtfully engage in social media while remaining in compliance with both the letter and the spirit of FDA regulations.
3. Our social media engagements will have both strong public health themes and appropriate marketing communications.
4. All social media messages and partnerships must be accurate, appropriate and transparent.
5. We believe that social media presents multiple opportunities to learn more about how our products impact the lives of patients.
6. We believe that social media engagement allows us to correct errors and misperceptions about both our company and our products.
7. We believe in using social media discover adverse drug experiences, which will then be addressed off-line.
8. We will strive to interact in a timely manner, appropriate to the general expectations of social media.
9. We believe that social media must be regularly monitored and our programs measured in real time to gauge effectiveness.
10. We respect but are not responsible for user-generated content that resides on sites we do not control.
11. We believe the path to engagement is through useful and thoughtful content and commentary.
One principle that runs as a red thread throughout all of these 11 principles is transparency. Real, honest transparency – not the usual translucency that “in compliance” often brings.
It’s time for action. As Friedrich Engels said, “An ounce of action is worth a ton of theory.
In particular, people who should know better let the FDA's spin affect their judgment. For example, Avik Roy who writes for Forbes and is a fellow at The Manhattan Institute who in turn salutes another MI fellow for his support of the Avastin withdrawal:
"Paul Howard is the guy who gets it right:
If you think (as I do) that the FDA should be expanding the accelerated approval pathway and allow more drugs to get to market based on promising early studies. rather than waiting for large Phase III clinical trials that can take years to complete, you can argue that this outcome actually strengthens AA. Critics have charged that AA is sop to industry, and that companies never do the follow up studies to support AA. Avastin proves them wrong.
This is exactly the point. If you want the FDA to approve more innovative, new drugs based on promising but early clinical results, you have to give the FDA a way to revoke those approvals later on, should larger trials prove that those drugs aren't as safe or effective as they first seemed. This is why the FDA should be congratulated for the way it has handled the Avastin breast cancer saga, and why I hope we will see the FDA handle more cases like this one, not less."
http://www.medicalprogresstoday.com/2011/11/fda-did-the-right-thing-in-pulling-avastin-for-breast-cancer.php
Both of them have no clue.
In fact the decision is less about Avastin and says more about the deep resistance at the Office of Oncology Drug Products to accelerated approval. And those who support the reversal as some victory for medical innovation and accelerated approval do not understand either the scientific challenges of establishing clinical benefit in first line MBC care and, given how hard it is to demonstrate any clinical benefit in end stage patients, how significant the addition of Avastin to current therapies really is. I am disappointed that observers treat the decision with the same sort of seat of the pants analysis reserved for Yankee post-game shows.
Dr. Hamburg's sympathy and support clearly lies with patients and the accelerated approval process. However her explanation of the reason for the withdrawal of the approval reflects a combination of evasiveness and duplicity on the part of FDA staff in the Office of Cancer Drugs that has dogged the accelerated approval of Avastin from the start.
Dr. Hamburg seeks to refute the claim that the FDA never switched the outcome measure of additional studies Genentech conducted to support ongoing approval of Avastin for MBC. She claims (and I will guess this was written for her) that progression free survival (how long people lived without tumors growing) was always the standard and that the OODP did not demand to see an increase in overall survival (how long people with and without Avastin treated before they died of any cause) which is a harder goal to meet. In fact, her decision memo shows that the FDA never came straight out to say PFS of any given length would be the basis for approval. Rather, FDA used the words "probably" or "might" and talks about the "potential" of using PFS for approval as long as the "magnitude" of the benefit was significant given the risks of the drug. Did FDA ever define what "magnitude" was sufficient? Never. Rather, as Dr. Padzur who runs OODP, told Genentech he would determine what the right "magnitude" would be when he saw the results.
In otherwords FDA said that improvement in PFS is not adequate unless a large enough magnitude that it will decide after the fact and by the way if it doesn’t think it is big enough then a cancer drug must show a statistically significant improvement in OS to support approval. And Dr. Hamburg calls this objective evidence?
Additionally, Dr. Hamburg claims that the magnitude of clinical benefit has nothing to do with achieving a statistically significant benefit. (Those words might and should be used by companies going forward as they design clinical trials.) All well and good. Yet the FDA ultimately revoked approval because Avastin did not show a statistically significant increase in overall median survival. So does statistical significance matter or not? Apparently it does when you want to undercut approval for use of a product you opposed at the outset. Similarly, when Dr. Hamburg claims that Genentech could never identify a subset of patients that might be more likely to benefit from Avastin in MBC she fails to point out that the FDA never asked for such data, nor did it state that it would extend approval based on response in a small group of patients. In any event, the FDA decided that post-hoc analysis to identify super-response of a statistically significant amount is not evidence of magnitude.
Her opinion ignores the fact that several other first line treatments for MBC were approved using PFS as an endpoint with half as much clinical benefit. Let me put in terms that supporters of the decision can understand and let me put it in bold: Specifically in the first-line treatment of MBC setting, two other drugs have received full approval using progression free survival or time to progression (TTP) effects of a much low magnitude than was used to grant Avastin accelerated approval. Several second and third line drugs also were given full approval with well below the median gains in PFS of Avastin. In all but two cases, full approval was granted without a statistical significant improvement in OS.
Dr. Hamburg offers some hints about how to show clinical benefit going forward: patient outcome data will count and designing trials to show which people might gain most from medicines will as well. Her brief discussion about how groups that support continued use of Avastin in MBC could appear to biased because they receive unrestricted support from Genentech is a very low blow and hypocritical because the FDA is seeking to loosen its own conflict of interest rules to get more qualified experts on it's advisory committees.
But in general Dr. Hamburg justifies the creation of higher threshold for the use of surrogate endpoints, a threshold that is arbitrary and without any empirical justification, in order to force all drugs to demonstrate OS. All this suggests that allowing FDA to retain discretion over what is enough benefit will generate more uncertainty and ends accelerated approval as a real alternative. The Avastin decision says less about the science of the FDA and more about how regulatory uncertainty (as a result of bias or lack of knowledge) can undermine innovation. How can the so-called supporters of accelerated approval endorse Hamburg’s endorsement of this bait and switch?
I’ve just returned from the Third International Conference for Improving Use of Medicines (ICIUM). The meeting was held in Antalya, Turkey – not that there was any Thanksgiving-related iconography.
The first thing to remember is that in the world of NGO healthcare policy, “international” means “non-Western.”
There were quite a lot of government officials from Africa, Asia, the Middle East, and a dusting of officials from the US (mostly USAID) and the EU.
The third ICIUM, for the first time, included members of the innovator pharmaceutical industry. They were not asked to wear yellow stars. Noticeable by their absence were any generics manufacturers.
Not surprisingly, most of the ICIUM participants fall into the category of old familiar faces, although there were some noticeable by their absence – like Jamie Love. Well, absence makes the heart grow fonder.
It was an acronym-heavy event, with the most commonly used abbreviation being “RUM” for “Rational Use of Medicine.” (And remember, you can’t spell “rational” without R-A-T-I-O-N.) The other acronym of note was LMIC (“Lower/Middle Income Countries) also referred to as “indexed countries” (IC).
Every day brought a slew of interesting commentary. For those of you unable to make the voyage, some selected tidbits:
Kathleen Anne Holloway (WHO) raised the issue of the deleterious impact of competition among NGOs (as well as the WHO) for project funding. She was blunt about the negative consequences of “competition for prestige, attention, and priorities.” Those negative consequences, it turns out, result in less funding for some WHO projects. Not that the majority of the audience gives marketplace competition any credence – although it’s surprising such incredulity also seems to extend to the marketplace of ideas.
Klaus Leisinger (Novartis Foundation for Sustainable Development) spoke about the need for everyone engaged in the international debate over the improved use of medicines to stop stereotyping each other. (Amen. It’s about time we fought to put the “civil” back in “civil society.”) He then called for the creation of a “grand coalition” think tank to address the opportunities that working together might offer. Klaus – sign me up.
Richard Laing (WHO), after praising GSK CEO Andrew Witty for his company’s devotion to corporate social responsibility, offered that “There is often an unfortunate disconnect between pharma HQs and their marketing departments when it comes to the issue of access to medicines.” Is a word to the wise sufficient?
(Remember -- A Word to the Wise was a 1770 play by the Irish writer Hugh Kelly. His first work was the 1767 hit False Delicacy.)
Dr. Laing also acknowledged the absence of both national and global generics companies from the ICIUM event -- a rather gaping hole that needs to be filled next time around. This issue came up again and again as panelists pointed out (again and again) that in many markets the prices of both branded and non-branded generics are actually higher than (off-patent) innovator medicines.
The role of generics cannot be overlooked considering that something on the order of 98-99% of all medicines on the WHO’s Essential Drug List were either never patented of their patents have long since expired in the IC countries. That being the case, there must be other reasons (beyond the traditional whipping boy of intellectual property rights) to explain why millions of people do not enjoy regular and reliable access to life-saving medicines.
Eva Ombaka (identified only as “formerly of the Tanzanian Ecumenical Pharmaceutical Network”) suggested that pharmaceutical companies should redirect their spending on “inappropriate marketing” to the public health dissemination of “access to medicine information.”
Note to Big Pharma – please refocus all “inappropriate marketing” line items to this effort.
Batool Jaffer Suleiman (Oman Ministry of Health) said that, when it comes to promoting RUM programs, government should “be seen as a support rather than the police.” Indeed. But support for whom and for what? Price or patients? She also shared that her office publishes a regular RUM newsletter called “Pharmaco-Logical.” Who said Omanis don’t have a sense of humor?
Jing Sun (China National Health Development Research Center, Ministry of Health, China) offered an excellent presentation on her nation’s nascent healthcare reform efforts. Her complete presentation will shortly be available on the ICIUM website, http://www.inrud.org/ICIUM/ICIUM-2011.cfm.
She noted that China currently spends 5.14% of GDP on healthcare and (of that 5.14%) 44.5% is spent on pharmaceuticals (about 2.5% of total GDP). When you compare this to the roughly 17% of GDP spent on healthcare by most western nations (with roughly 12% directed towards medicines), there should be some questions asked. And the first one should be; is the higher Chinese percent spend on pharmaceuticals a good thing or a bad thing?
Time and again, speakers from the LMICs noted that their spending on pharmaceuticals was regularly in the 30-40 percent range of their annual healthcare expenditures (i.e., Jordan with 10% of GDP spent on healthcare and 34% of that amount on medicines). Considering that product costs for index nations are significantly lower than western prices, perhaps the higher LMIC spend on pharmaceuticals is because medicines are something these nations can offer their populations – making up for a dearth of spending on the medical professionals and hospitals they do not have – but which represent the lion’s share of western healthcare spending.
In this respect, the high percentage of spending on modern medicines is akin to the LMIC telecom leap-frogging that has created a much higher penetration of mobile phones per capita than in the West. Something to think about.
Another question to ponder is to what extent government interventions may account for higher medicine prices in LMICs. Many governments implement aggressive protectionist policies that allow local generics companies to charge higher prices for products that are off patent – paying too much for older medicines that should be available to patients at commodity prices.
Saul Walker (Department for International Development, United Kingdom) bemoaned the unfortunate and counterproductive tension between “sustainability and innovation.” He also pointed out, with great honesty, that “government is not a unified entity” when it comes to developing and implementing healthcare policy. (He was directing his comments at the LMICs – but it’s equally true across the board and around the globe.) He also mentioned “Big P and Small P” battles being fought both within governments and NGOs. And the P (in case you haven’t guessed) stands for “politics.” Perhaps a panel at the next ICIUM can focus on a new definition of “P value for public health.”
(And, while we’re on the subject of ICIUM IV, there should be a subject track on safety. There was almost no discussion of bioequivalence, GMPs, narrow therapeutic indexing, therapeutic switching, or biosimilars.)
Jonathan Quick (Management Sciences for Health, USA) asked if universal health care was “the golden ring or a Trojan horse?” His answer seemed to be “yes,” concluding that “failure to fully maximize needed expertise” is a major reason that universal healthcare systems are failing. One might also add “failure to understand the realities of market-based economies.” Socialism, as they say, works great until you run out of other people’s money.
Maryam Bigdeli (WHO) made the excellent point that, “Many of today’s problems are because of yesterday’s solutions.” Or as Abraham Lincoln said, “You cannot escape the responsibility of tomorrow by evading it today.”
Danya Qato (Brown University), offering one of the few US-centric presentations, made the excellent point that when it comes to medicines (specifically) and healthcare (more broadly) the barrier isn’t just cost. (Shocking, right?) Her US-based research among a largely Medicare-eligible population demonstrates that racial, social, and geographic disparities play a major role and that “access does not equate to use.” Reality. What a concept.
The other US-based presentation was by Elissa Ladd (MGH Institute of Health Professions). Her talk was on the detailing practices of Big Pharma towards the growing population of nurse-prescribers. (According to Ms. Ladd, there are 150,000 nurse-prescribers in the US, compared with only 100,000 physicians in general practice.) You’ve heard the argument before – pharmaceutical detailing is “bad” because it helps to “sell” products for profit!
She provided no evidence (anecdotal or otherwise) that the information pharmaceutical detailers provide to nurse-prescribers is in any way slanted or anything other than factual and 100% FDA-compliant. Her organization undertook some “educational” efforts that resulted in nurse-prescribers questioning the reliability of pharma-provided information. She positioned this as “success.” But – is having nurse-prescribers (or, for that matter, any prescriber) discount important medical information really a move in the right direction?
And then there’s the Access to Medicine Index (ATMi). The ATMi is an attempt to measure and compare the corporate social responsibility of both innovator (20) and generics (7) companies based on a number of different (and often quixotic) indicators. According the Access to Medicine Foundation, the index “aims to help poor people in developing countries gain access to medicine by encouraging the pharmaceutical industry to improve its commitments and practices related to this issue.” Since it’s a comparison, the theory is that competition amongst companies will drive desirous “socially responsible” behaviors.
A noble goal – but the devil is in the details. Consider subjectivity. For example (and most notably) the index’s four strategic pillars are “commitments, transparency, performance, and innovation.” And its “technical” benchmarks include such vague categories as General Access to Medicines Management, Public Policy and Market Influence, and Capability Advancement in Product Development and Distribution.
As Goran Tomson (Karolinska Institute) pointed out, the index’s methodology cannot be reproduced, hence it cannot be considered statistically valid -- unless you choose to abide the Marxist (Karl not Groucho) maxim that “a special environment creates a special class.”
There are also troubling issues relative to the ATMi’s metrics for success. As the index’s methodological designer, Afshin Mehrpouya (HEC, Paris), opined, the only current measurements are “web hits and media coverage.” Not very exciting, plausible, or helpful from a health policy analysis perspective.
Another ATMi metric is the opinion of patient groups. When asked why certain patient groups were chosen (they are not named in the ATMi), the answer was that groups were chosen based on their “credibility.” In NGO-land that’s code for groups who do not accept funding from the pharmaceutical industry or may not share the anti-private sector bias of the party line. At minimum, that’s a dubious selection bias.
Most damning was Dr, Mehrpouya’s admission that the index, “doesn’t take the patient viewpoint into perspective.
Dr. Tomson also pointed out that the ATMi’s “review committee” consisted almost entirely of “familiar faces,” thus creating an issue of normative bias.
These are all polite ways of saying that the design criterion stacks the deck. But, hey – doesn’t the end justify the means?
What the ATMi has succeeded in doing is getting the attention of innovator companies who want to strut their corporate social responsibility stuff index-wise. The result is that many LMICs are considering the index when making national formulary decisions, thus giving additional points to innovators over generics companies. Some observers at the ICIUM conference viewed this as an unintended negative consequence. But the truth hurts.
To paraphrase Adlai Stevenson, “If NGOs and generics companies will stop telling lies about pharmaceutical innovators, perhaps Big Pharma will stop telling the truth about them.”
One suggestion that came up during the panel debate on the ATMi is to create a parallel index that measures LMICs by whether or not their policies and political environment facilitate or hinder their citizens’ access to healthcare. One such measure, as bravely noted by Jeffrey Kemprecos (Merck), is to measure and address the 800-pound gorilla in the room – the lack of transparency in the public sector and – yes, he dared utter the word -- corruption.
Goran Tomson put the discussion about the ATMi -- as well as the entire ICIUM enterprise -- into perspective when he said the index lacked for “higher ambitions.”
Any maybe that’s the best go-forward message from and for ICIUM – let us strive for “higher ambitions” as colleagues who can (in the words of Klaus Leisinger) “agree to disagree.” Easier said than done. But it’s worth a try.
In other words – let’s talk turkey.
During my tenure at the FDA I served on the agency’s first Counterfeit Drugs Taskforce. Alas, one of the items in our draft report – that criminal penalties for counterfeiting be significantly enhanced – didn’t make it into the final recommendations since it was seen as exceeding the agency’s mandate (and stepping on toes at the Department of Justice).
Well – better late than never. A new bipartisan (and bicameral) effort has been introduced to stiffen the penalties counterfeiters will face for putting illegal and unsafe medicines into the medicine chest of unsuspecting Americans.
The Counterfeit Drug Penalty Enhancement Act will increase penalties for the trafficking of counterfeit drugs to reflect the severity of the crime and the harm to the public. While it is currently illegal to introduce counterfeit drugs into interstate commerce, the penalties are no different than those for the trafficking of other products, such as electronics or clothing. The Counterfeit Drug Penalty Enhancement Act will target violators that knowingly manufacture, sell or traffic counterfeit medicines to the USA.
On the Senate side the bill is sponsored by, Patrick Leahy (D, VT), Chuck Grassley (R, IA), Michael Bennet (D, CO), and Richard Blumenthal (D, CT) and on the House side by Patrick Meehan (R, PA) and Linda Sanchez (D, CA).
Senator Leahy: “While the manufacture and sale of any counterfeit product is a serious crime, counterfeit medication poses a grave danger to public health that warrants a harsher punishment. This legislation will raise those penalties to a level that meets the severity of the offense. Deterring this epidemic problem is a bipartisan effort.”
Senator Grassley: “Counterfeit medicines are some of the most profitable commodities for criminal organizations. Purchases of counterfeit drugs by unsuspecting customers are growing at alarming rates, especially over the internet.” These drugs present a serious threat to the health and safety of people around the world. It’s important we address this threat by imposing harsher penalties on criminals who counterfeit these medicines.”
It’s about time.
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.
