Latest Drugwonks' Blog
Big Hairy Audacious Goals set doers apart from talkers.
Last week, “under the patronage of the Lebanese Ministry of Public Health,” I was honored to participate in the two-day “Drug Quality Forum” held at the American University of Beirut.
The focus of the event was on the impact of drug quality and safety with focused presentations on (among other topics) API quality, GMP requirements (as well as the often forgotten but hugely important GSDPs – Good Storage and Distribution Practices), data requirements and regulatory pathways. In attendance were senior health ministry officials from Lebanon, Syria, Jordan, and Iraq as well as representatives from the WHO and France’s Agence nationale de sécurité du médicament et des produits de santé (ANSM) – née AFSSAPS.
Some brief verbatims:
David Holt (St. George’s University, London): “There is nothing more expensive than clinical failure.” He also cited the following from a recent paper in the British Journal of Pharmacology (2011;72:727-30)
“There are few prospective studies assessing potential additional risks associated with substitution and there are no established protocols by which switching is monitored or assessed.
This may make it difficult to know whether the money saved on the initial drug will still be saved as treatment outcomes on the substituted drug become apparent.”
Gilberto Lopes (The John’s Hopkins Singapore International Medical Center) pointed out that it’s not about the cost of pharmaceuticals but rather spending on pharmaceuticals – and that having more new therapies is a good thing.
Mark McGrath (Novartis) made the very important point that, “Quality cannot be implied.” This followed on my earlier comment that “Quality cannot be tested into a product. Quality is by design.”
At the end of the first day, Conference chairman, Dr. Joseph Simaan (Professor and Chairman, Department of Pharmacology and Toxicology, Faculty of Medicine, American University of Beirut), commented that public health officials are faced with “a moral dilemma” of wanting (and being under pressure) to approve locally manufactured generics – but cannot, in any way, allow sub-standard products onto the market.
As Khahil Gibran wrote, “Perplexity is the beginning of knowledge."
Day Two focused on topics ranging from bioavailability to bioequivalence and biowavers – highly technical presentations, but not without some humor. One example:
Q: Why were the two generic chemists so sad?
A: They were dissolutioned.
(Who said drug regulators don’t have fun?)
On that same topic, Soula Kyriacos (Pharmaline) gave a very informative talk on the correlation of product dissolution and bioavailability (in vitro vs. in vivo). And take my word for it – anything sounds riveting when presented by a smart woman speaking English with a Lebanese/French accent.
The last session of the meeting was devoted to developing a series of consensus recommendations for the attendees to take back to their respective nations within the GCC (Gulf Countries Cooperative). I was pleased to help moderate this effort along with Dr. Simaan, Dr. Ghassan Hamadeh (Associate Dean for CME, Faculty of Medicine, American University of Beirut) and Dr. Ziad Nassour (President of the Lebanese Order of Pharmacists).
Here are those recommendations:
1. Ministries of Health (MOH) to develop criteria for approval of bioequivalence centers (CROs) in compliance with International standards (GCP, GLP) in line with FDA, EMA, WHO or ANSM mechanisms for inspection and approval.
2. MOH to ensure that complete, detailed, and well-documented files are submitted for registration of all drugs according to ICH guidelines.
3. Any biosimilar application for an imported product must include a prior EMA or FDA approval
4. MOH must insist on emphasis of the quality of the active ingredient with full information on sourcing, materials and manufacturer.
5. Information leaflets of generic and innovator drugs should be up to date and made accessible and include all safety information.
6. No marketing approval for drugs without stability, and bioequivalence studies according to ICH guidelines.
7. No marketing approval for drugs not approved or marketed in their country of origin.
8. MOH to initiate product specific pharmacovigilance systems.
9. MOH to set up an efficient communication system to be used by Health Care Professionals and the Public to report safety issues with drug products
10. Drugs should not be sold in dispensaries. (Note: In Lebanon, “dispensaries” are supposed to provide generic medicines for those who cannot afford to buy them at no charge.)
11. Create a Department for Quality Control within the Ministry of health to review, authenticate submissions and inspect dossiers as well as track batches and perform random testing of marketed products.
12. Ensure all drugs are available through controlled supply chain.
13. Build capabilities in the ministry of health through enhanced training in
Big Hairy Audacious Goals? Absolutely.
That’s precisely what makes them so exciting.
In a pithy commentary in Nature, Sir Alasdair Breckenridge, Chairman of the MHRA, (et al.) writes:
A life cycle approach to pharmaceutical regulation, in which the benefit–risk balance of new drugs continues to be robustly assessed following market approval, is emerging in both the United States and Europe.
Certainly.
But …
Whether the pharmaceutical industry joins in this consensus and whether companies will serve as willing or reluctant partners in an invigorated post-marketing drug regulation scheme remains unclear. Additional regulatory requirements are never welcome in the short term, and the post-marketing studies that may be required under the life cycle approach may impose substantial costs on manufacturers. However, industry has learned the hard way that inadequate responses to safety signals can have a serious detrimental impact not only on public health but also in economic and reputational terms. The long-term interests of manufacturers and regulators in well-characterized benefit–risk profiles are essentially the same.
The complete article can be found here.
What’s in a name? That which we call a rose by any other name would smell as sweet.
No doubt. But a name also is an aspiration.
It is with that preface that I am pleased to share a bit of news that arrived with my July Drug Information Journal – the publication (the official journal of the DIA) is changing its name.
Transparency Alert: I am an Associate Editor of this journal.
The new title, Therapeutic Innovation & Regulatory Science, is a stake in the ground. First, it’s not just about “drugs.” That’s a great leap forward. Second, it’s about innovation. That’s where the action is. And third, it’s not about regulation, but rather “regulatory science.” That’s a global topic -- from the FDA’s Critical Path initiative to the EMA’s IMI and beyond. And, dare we hope – one that might some day lead to greater harmonization.
Like I said – a name is also aspirational.
Bon Courage, Therapeutic Innovation & Regulatory Science!
Our aspirations are our possibilities.
-- Samuel Johnson
According to information presented at the American Society of Health-System Pharmacists Summer Meeting, health systems that rely primarily on voluntary reporting of adverse drug events identify as few as 6% of events." James M. Hoffman, PharmD, MS, BCPS, Medication Outcomes & Safety Officer, and Director, Medication-Use Safety Residency Program, and Associate Member in pharmaceutical sciences at St. Jude Children's Research Hospital in Memphis, TN, argued for using several different methods of detecting adverse events in order "to get a more complete understanding of opportunities to improve the medication use process … Underreporting is a key limitation of error reporting systems.” Hoffman proposed the use of four common methods: "incident reporting; direct observation; medical record review; and trigger tool." He also said that electronic health records can be "a key resource for event detection."
“Apple could hang on for years, gamely trying to slow the decline, but few expect it to make such a mistake. Instead it seems to have two options. The first is to break itself up, selling the hardware side. The second is to sell the company outright.”
That was in 1995.
I will not predict what the future holds for healthcare reform. If history is any teacher Obamacare will not be overturned or replaced anytime soon. No major health care expansion or program has. I could be wrong and I hope I am.
In the meantime, one thing might be more certain: Those who supported and voted for Obamacare now own it.
In my opinion, that's not a good thing.
Roberts got Obamacare off on a technicality and turned it into a tax collection/IRS issue. All the happy talk about pre-existing condition coverage will fade. Now the question will be: Who voted to raise taxes on the middle class? On medical device companies? On the dividends of retirees? All for something that Congress does not have power to require people to buy? Are you serious?
When premiums rise, costs explode, government hands out Solyndra like contracts on the one hand while cutting Medicare benefits on the other, when new treatments are denied and things go wrong it's the Democrats that people will blame.
In the short term, Democrats will seek to shift the discussion by claiming Romney is the intellectual godfather of Obamacare.
Not even close.
True, Romney had a (small) penalty tied to a real mandate (not a mandate posing as a tax pretending to be penalty). He didn't raise taxes. He took Medicaid money and state funds to extend Medicaid to cover low income people that did not have or could not afford insurance and required them to pay part of the cost of premiums and health care. That's something forbidden under Obamacare. There were no boards to ration or control what technologies or treatments doctors used or patients could have.
In any event, when premiums rise, costs explode, government hands out Solyndra like contracts on the one hand while cutting Medicare benefits on the other, when new treatments are denied and things go wrong it's the Democrats that people will blame.
Regardless of whether the president is re-elected or not or whether Republicans gain control of the Senate and hold on to the House, the Democrats and the interest groups that pushed so hard for government expansion of healthcare will and should be held responsible for the taxes, the premiums and the implementation.
As a great man once said, nothing is over till we (the people) decide it is.
http://www.youtube.com/watch?v=MsmybQKpmTw
Healthcare Evolution and the Survival of the Fittest
It doesn’t stop with the Supreme Court. Now we actually have to do something.
One of the big mistakes of the Affordable Care Act (“Obamacare”) was to try to solve all of our national healthcare problems at the same time and on a national scale. The Individual Mandate was just the poster child for many of the law’s fantasy solutions.
Now’s the time to stop talking about healthcare “reform” and start focusing on the need for healthcare evolution.
But the Supreme Court ruling notwithstanding – a grandiose national solution is never going to work. While it sounds good politically to say, “we solved the healthcare problem” – it’s just not feasible.
Post the Supreme Court ruling many see problems, but there’s also opportunity.
The opportunity is to realize that the way we can evolve healthcare is by recognizing that it must be done locally – on a state-by-state level. When it comes to reform, states are the laboratories of invention. (Welfare reform and the “Wisconsin Works” success comes to mind as a stellar example.)
But, just as a “one size fits all” national model is a naïve chimera, so too is the hope that one state’s success in healthcare will be equally workable in any other member of the Union. The many positive achievements of “Healthy Indiana” (which requires enrollees to contribute up to 5% of their gross income to an account used to pay for medical expenses – with the state picking up the rest) may not translate to larger states such as New York, Texas or California. Needless to say, there are many lessons to be learned from the failure of “Commonwealth Care” in Massachusetts (such as the danger of providing insurance without ensuring access to a physician).
If a key goal of healthcare evolution is broader coverage at lower costs, one national program that does offer valuable lessons for the path forward is Medicare Part D (the Medicare prescription drug benefit). Part D applies free-enterprise principles to the nation’s health-care system (letting competition drive down prices and increase choice and quality) rather than operating like a government-managed utility.
Part D is a resounding success among seniors (as measured by participant satisfaction pushing 90%), below budget costs (the price of Part D over the next decade is expected to be nearly $120 billion less than originally estimated) and lower than expected premiums (in August 2011, HHS announced that premiums would be slightly lower in the drug program in 2012).
Smart partnership between government and the free market works.
It works at keeping costs low and – most importantly – improving care. As JAMA reported, “Implementation of Medicare Part D was followed by increased use of prescription medications, reduced out-of-pocket costs, and improved medication adherence.” And this, in no small measure, significantly reduces more drastic medical interventions -- which in turn reduces our overall national health care spending.
The Supreme Court’s ruling notwithstanding, the President should grant waivers to all 50 states to opt out of the ACA’s dictates and restrictions and allow them to develop their own strategies for healthcare evolution.
It is not the strongest of the species that survives, nor the most intelligent, but the one most responsive to change.
-- Charles Darwin
CDER’s Office of Prescription Drug Promotion (OPDP) has decided to issue five separate guidance documents that will address “social media” issues that have arisen in advertising and promotion areas. “We decided not to issue just one guidance because it would be burdensome and changing all the time,” OPDP director Tom Abrams told a Drug Industry Association conference in Philadelphia 6/25. ”Instead, guidance will be developed on policy-specific issues and not on any specific technology platform. We don't know what the platforms will be in two years. We know Facebook, Twitter, and Youtube are very popular now, but we don't know how they will evolve or what new platforms will be available next year...So to try to have a guidance based on one platform, such as Facebook, would be a mistake and quickly outdated. And we are developing guidance applicable to whatever platform companies my use for prescription drug promotion.”
A guidance under development is on Fulfilling Regulatory Requirements When Using Tools Associated with Space Limitations, which will address how to include risk information when there is a character limitation such as Twitter, Abrams said.
A guidance on Fulfilling Post-marketing Submission Reporting Requirements is under development to address how to submit promotional material at the time of initial use when engaging patients and others in chat rooms, Abrams told the conference. Other guidances will touch on the appropriate use of hyperlinks, and the best practices for correcting misinformation on third-party Web sites.
Without providing any specific timeline for issuance, Abrams said OPDP has assigned substantial resources to the guidance development and other areas, like the Office of Chief Counsel, are also helping with their drafts.
By BRETT NORMAN | 6/25/12 10:36 PM EDT
The bipartisan treatment the FDA user fee bill has enjoyed did not fall like manna from heaven. It came at the insistence of the pharmaceutical and medical device industries, the FDA and legislators on both sides of the aisle who agreed early on they would get a deal done. And then actually did it.
The Senate is expected to send the Food and Drug Administration Safety and Innovation Act — a major piece of election-year health care legislation — to President Barack Obama late Monday or Tuesday. The bill, which would provide the FDA with more than $6 billion in industry user fees over five years to fund a share of its medical products reviews, passed the full House last week with scarcely a complaint.
Read more: http://www.politico.com/news/stories/0612/77815.html#ixzz1z0fEUZiA
From USA TODAY:
Doctors urged to be 'gateway' for obesity tests, treatments
Physicians should screen all adult patients for obesity during office visits and either refer obese patients to comprehensive weight-management programs or offer them one, says the U.S. Preventive Services Task Forcein new recommendations announced Monday.
From today’s edition of the Wall Street Journal:
LONDON—Europe's drugs industry is urging EU leaders give it two major concessions to help keep medicine supplies flowing into crisis-hit countries such as Greece and Spain.
Drug makers in the region have been hard hit by payment arrears and steep price cuts caused by the economic crisis.
They are now urging EU leaders at their coming summit to grant the concessions to prevent drug price discounts granted to troubled southern European countries from distorting supply and demand in the bloc.
In a letter issued Monday, Andrew Witty, chief executive of GlaxoSmithKline GSK.LN +0.14% PLC and the current head of Europe's pharmaceuticals association identified two huge problems for the industry: drug reference pricing, or referring to other countries when setting prices, and parallel trade, or the re-exportation of pharmaceutical products from lower-priced to higher-priced countries. He said they represent a threat to orderly supply of medicines in the region.
"The practice of referral to other countries when setting prices for medicines results in inefficiencies and sometimes in limited supplies. Where the industry has agreed to temporary price cuts to bridge funding gaps, such as in Greece or Portugal, other countries not subject to the same financial pressure automatically lower their prices," Mr. Witty said.
The other major impact comes from parallel trade, which is legal in the 27-member European Union. Low prices in Greece, Spain and other southern European countries have drained medicines from the region to wealthier countries where the prices are higher.
"Recent months have seen a significant increase in this arbitrage trade, which is the result of market distortions caused by pricing policies," Mr. Witty said.
"One immediate impact is a shortage of medicines for patients in countries such as Greece and Romania. There is a genuine risk of supply disruption in several countries," he said.
Mr. Witty's letter has been sent to European Union leaders, Commission President José Manuel Barroso and Council President Herman Van Rompuy ahead of the June 28-29 summit. Mr. Witty sent the letter in his capacity as president of the European Federation of Pharmaceutical Industries and Associations, or EFPIA.
In the letter, EU leaders are urged to exclude countries that are undergoing fiscal restructuring programs from the basket of countries to which they refer in setting medicine prices.
Illustrating the impact of reference pricing, Mr. Witty said a 10% price cut in Greece cost the drug industry €299 million ($375.8 million) in Greece, but €799 million in Europe and €2.15 billion world-wide if all countries were included, re-referencing Greek prices through formal and informal links.
"The impact of a price cut in Greece therefore resonates across the E.U. and globally—the implications for the R&D-based industry are clear," Mr. Witty said.
EFPIA also wants a temporary ban on re-export of medicines to higher-priced countries, to prevent supply shortages.
"The Commission should accept this temporary response to an emergency situation," Mr. Witty said in his letter.
"These measures would deliver tangible relief and give a very important signal about Europe's support for pharmaceutical innovation, while ensuring fair patient access to innovative medicines. They could also provide a platform for further discussions at national level on a more strategic approach to supporting innovation and managing cost-containment."
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.
