Latest Drugwonks' Blog

Yesterday, Manhattan District Judge Paul Engelmayer asked some very probing – and some very naive questions regarding Amarin’s request for a preliminary injunction allowing it to tell doctors about the unapproved use of its Vascepa (icosapent) fish oil pill.

Judge Engelmayer engaged Assistant US Attorney for the Southern District of New York Ellen London, why a truthful, non-misleading statement by itself could be considered “actus reus” – the wrongful act of a crime – to support misbranding. London asserted that statements serve as evidence of intended use and require other elements to be prosecuted as misbranding. Good answer – but not good enough for Judge Engelmayer.

The judge kept asking why the FDA allows a heart disease claim for dietary supplements with EPA but does not permit it for Vascepa. His Honor needs a primer on DSHEA – and the FDA had better be prepared to offer it, otherwise this misunderstanding will persist – to the detriment of the agency’s case.

The Judge also questioned London about when the agency would be issuing further guidance on off-label communication, asking if it would be in 2015 or afterwards, or before Labor Day. Ms. London said she has “no idea” when the agency would act or if more speech will be permitted when it does. That looks like a signal that the court wants to grant the agency chevron deference – but not minus a written guidance.

According to Judge Engelmayer, there were “terrific arguments on both sides,” And closed the hearing without making a decision.


Note to FDA, “An ounce of action is worth a ton of theory.” (Friedrich Engels)

From BioPharma Reporter:

Top of Form

Bottom of Form

WHO further clarifies ‘biological qualifier’ system for naming biological active substances

The fight over how to name biosimilars has taken a turn toward further clarification, with the WHO (World Health Organization) offering another, updated draft  of its intended policy on "biological qualifiers" (BQ).

Nearly identical to the previous draft , which the USP endorsed , the WHO says the BQ system would not just be for biosimilars but “all biological active substances.” The scheme would assign four random consonants to an active substance, but it would not replace the INN (international nonproprietary name) scheme. Instead it would support INNs to “better harmonise international pharmacovigilance efforts,” as well as to avoid the “proliferation of separate and distinct national qualifier systems.”

As far as what products will use the BQ, the WHO calls on the names to be issued for biologics, biosimilars, as well as non-glycosylated and glycosylated proteins. “Only exceptions will be vaccines, impure mixtures and complex biologically-extracted products like heparin or pancreatin to which INNs are not assigned,” the WHO clarifies.

Companies applying for a BQ for global use will be allowed to use it “for substances made in all manufacturing sites demonstrated to be comparable and by all manufacturing authorisation holders (MAH) distributing products which contain the substance,” the WHO says in the latest draft. “Should a regulatory authority find that a manufacturing site does not produce a comparable product, they may require application for a different BQ for that manufacturing site, but the two BQ’s would be hyperlinked in the INN BQ database.”

The WHO notes that some regulatory authorities have already decided that the use of a trade name and INN are adequate for prescription and dispensing, and that trade name, INN, MAH name and batch number are adequate for pharmacovigilance in conjunction with other tracking systems, such as 2D barcoding.

 But the WHO looks to persuade undecided regulators, such as the US FDA, with the idea that the use of the BQ offers a means to not only uniquely identify the drug substance even if used alone, but to help with crosschecking other information supplied in a prescription/dispensing or pharmacovigilance setting, in the absence of other sophisticated tracking systems.

 “While the FDA has not yet issued draft guidance on how current and future biological products marketed in the United States should be named, the agency intends to do so in the near future,” FDA spokesman Kristofer Baumgartner told us. 

Potential Issues

The WHO also points out in the latest draft scheme a possible area where problems could arise with the BQ system, noting that it is intended that drug substances would have the same BQ as long as it has the same amino acid sequence and is marketed with the same INN, but if “a change is made in which glycosylation is found to be not comparable, then a new Greek letter and BQ would be assigned.

This lack of comparability would need to be determined by the regulatory authority and the WHO INN should then be informed... This may mean that the same drug substance may have different BQs in different jurisdictions if different assessments of the comparability are made by the regulatory authorities, but this is likely to be rare and hyperlinks between the two database entries would be introduced.”

In addition, if a regulator finds that a manufacturing site does not produce a comparable product, they may require application for a different BQ for that manufacturing site, but the two BQ’s, again, would be hyperlinked in the INN BQ database.

From today's edition of Morning Consult:

PDUFA VI: Do Ask. Do Tell.

By: Peter J. Pitts

Something that bothered me a lot about the Fifth Prescription Drug User Free Act (PDUFA V) was that when you asked people in industry, what does success look like to you, almost unanimously insiders said, “success means getting it done early and having it done clean.” That’s just punting on an amazing opportunity. If success means fast and clean, you miss the rare chance to bargain hard when the agency is really listening.

As we move towards PDUFA VI, people will look hard at what PDUFA V delivered which was, in many respects, a lot of meetings. Today people are saying, “now that we’ve had the meetings we want action, we want movement forward, we want greater predictability in new, more complicated areas.” It’s going to be interesting to see if industry is really willing to step forward and hold the FDA’s feet to the fire, not necessarily hold them hostage but be tenacious negotiators rather than roll-over puppy dogs.

That which gets measured gets done and the FDA will get high marks for, among other things, Patient-Focused Drug Development. There were many disease-specific meetings and they were very worthwhile and respectfully contentious. There was creative tension.

Now, per PDUFA VI, what is going to happen next? How is the FDA going to work with all of the groups they met with to help them help the agency help patients? How can the agency help these organizations draft guidances? What’s the mandated requirement? How quickly can it happen?

Communication is another key issue. PDUFA V spoke to more and more regular communications post-filing but also during the early stages of the process — and that happened. But what hasn’t changed is who is doing the communicating. Has there been more communications at higher levels when there is scientific dissonance between sponsor and agency? I don’t know whether you can write that into PDUFA, but it’s a significant issue. I don’t know how FDA would facilitate since there are only so many senior folks, but it’s an issue that has to move forward – and PDUFA VI presents that opportunity.

What’s interesting, although not strictly speaking a PDUFA issue, is how is the FDA is dealing with the regulation of opioids. One of former Commissioner Hamburg’s big victories was putting her foot down and saying, “Listen, all therapies have risks, and a broader pharmacopeia within any therapeutic categories is important. We have to enhance education.” So where in PDUFA is the FDA’s mandate to do better physician, pharmacist, patient, and caregiver education in a lot of these areas, especially in areas where the medications are of higher risk – and politically sensitive?

Alas, you can’t hold PDUFA negotiations at Yankee Stadium. PDUFA V was the first time patient advocates actually had a seat at the table. But what about smaller biopharmaceutical companies that aren’t dealing in tens of billions of dollars of issues, who have one or no products and aren’t members of PhRMA or BIO? How do you empower companies who aren’t major players by volume to be a valuable part of the regulatory policy conversation? That’s a tough question. You have to bring more senior minds to the table from companies currently at the table as well as those who are on the periphery.

It’ll be curious to see who attends and participates at the July 15th kickoff meeting for PDUFA VI. I’d like to see more patient groups there. I’d like to see more representation of smaller biotech companies too so they can see first-hand about what’s going on. It needn’t be a secret club.

Should there be a discussion within PDUFA VI of the FDA actually putting on paper new, more segmented rules for bioequivalence? It’s been almost three years since the recall of generic bupropion and the FDA is still dealing with bioequivalence on an ad hoc basis for seizure meds, antipsychotics, long-acting release medicines and ADHD products. Industry has said “enough!” Mallinckrodt is suing the FDA for asking them to take their generic methylphenidate off the market because of bioequivalence issues. A judge looking at this case might say, “You know what FDA? I generally want to give you chevron deference on the science issues, but where’s the guidance?” The FDA should drive the issue rather than litigate the process, and maybe PDUFA VI is a way to move the conversation forward in a collegial rather than a confrontational manner.

The issue of what data can you use in approvals, which is within the current draft of 21st-Century Cures, is fascinating and a potential game-changer — but shouldn’t that more appropriately be a PDUFA VI conversation? Similarly the issue of off-label communications. Better to have it dealt with in PDUFA VI than in the courts. A potential legal decision could be a sledgehammer solution that will do nothing other than empower people with ill intentions. The issue of communications isn’t only about who’s presenting it and in what context, it’s also about the intent. And when you begin to try to litigate intent, it’s a blunt instrument. And what about finally getting real in PDUFA VI about FDA’s role – and resources – in accelerating biomarker qualification?

Another thing, relative to PDUFA VI, is recognizing what was done and didn’t work in its previous iterations. Let’s specifically call out early safety signal communications. The theory was the agency was going to, on a quarterly basis, publish a list of products for which it had enough information to require further investigation. All that resulted in were sensational media stories and too many patients going off their medicines. Communications issues notwithstanding, did this program result in one significant label change or product recall? It’s time to examine the risk/benefit ratio.

Should PDUFA VI change the FDA commissionership from a political appointment to a six-year term, like the FBI director? Take it out of the political cycle. It’s an opportunity to have that conversation. The FDA commissioner counts when the commissioner has an aggressive public health agenda. If the commissioner chooses to just be the public face of the agency, it’s a wasted opportunity.

What’s the difference between having a confirmed commissioner and having an acting commissioner? How does it impact the way the agency operates on a day-to-day level? The answer is that it doesn’t really have any impact at all. The agency continues to run. Interesting fact, the FDA has around 16,000 employees. Within that whole 16,000, there are (give-or-take) about ten Schedule Cs (political appointments). That means the director and every employee, every single one, top to bottom of every center without exception is a career public servant. The value of having a confirmed commissioner is having a long-term leader who is engaged AND who doesn’t have a strong learning curve — a guy like Rob Califf, for example, who can actually say, “This is my mission.” A successful commissioner sells his mission to the senior staff and then it becomes organic.

If you think about PDUFA VI, go back to First Principles. Why user fees? Why was the industry willing to pay for something that it previously got for free? The answer is that industry wanted predictability, whether it’s six months, a year, 18 months, two years, getting a PDUFA date is crucial for drug developers for a variety of reasons. Take the concept of predictability one step further and say, “We want not just an action date but predictability on a whole variety of issues — off-label communications, expedited pathways, postmarketing surveillance, biomarker development, over a broad spectrum of regulatory activities”   — that will lay the groundwork for a more creative and fruitful conversation.

By Robert Goldberg
Special to Roll Call

Thanks to President Barack Obama’s landmark healthcare law, insurance companies are no longer allowed to turn away patients with pre-existing health problems. For millions of sick Americans, this “guaranteed issue” mandate has been transformative, ensuring they can secure the coverage they need to afford vital medications.

Unfortunately, insurers have discovered a sneaky way to undermine this requirement. They’re structuring plans to heap huge costs and bureaucratic burdens onto high-risk patients. Technically, such patients are insured. But they don’t actually have access to medical care.

Federal officials must halt such discriminatory practices. Fortunately, Reps. David McKinley, R-W. Va.,  and Lois Capps, D-Calif., recently introduced the Patients’ Access to Treatments Act (PATA) (HR-1600) to do just that. Lawmakers should pass it immediately.

Insurers strap sick patients with big bills by putting expensive medications into the highest “tier.” Insurers typically divide their drug benefits into different tiers, with the lowest providing the most financial support and the highest providing the least. The higher the tier, the higher the patient’s out-of-pocket expenses.

For example, in the state-level insurance exchanges established by Obamacare, more than half of the popular “Silver” plans place all multiple sclerosis drugs in the top tier. Patients suffering from this devastating conditions are getting hit with huge costs. Many are forced to forego needed treatments.

Making matters worse, the slice of Silver plans requiring enrollees to pay 30 percent or more of the cost for top-tier drugs has jumped dramatically since last year, from 27 percent to over 40 percent.

PATA ensures that patients can afford these life-saving medications. The bill prohibits insurers from grouping specialty drugs in higher cost sharing tiers than the ones used for regular medicines.

Many health insurers also have a “fail first” policy. Patients must first take drugs that are less effective and often less safe — and only when these fail can they receive needed medicines.

In other words, insurers force people to get sicker before offering them lifesaving treatments.

Consider the plight of Shima Andre, a hepatitis C patient in Los Angeles. Shima’s insurance company refused to cover Harvoni — a costly yet highly effective hepatitis drug — because her liver wasn’t damaged enough. Even after repeated pleas from her doctor, the insurer wouldn’t budge.

Technically, Shima is insured. But that coverage means little, since she can’t access the drugs she needs. Rather, she must take older medicines that the FDA warns have a high risk of serious and even fatal side effects.

A new Harvard study suggests insurers are deliberately offering thin coverage for high-cost therapies to dissuade chronically ill patients from signing up in the first place. When people who need costly drugs see treatments’ price tags, they look elsewhere for coverage — which is what insurers want.

Insurers justify such discrimination by claiming it helps contain healthcare costs, keeping premiums affordable. After all, those with serious illnesses disproportionately rely on expensive, specialty medicines. They’re the 1 percent of patients — often referred to as “super spenders” — that account for more than a fifth of the nation’s annual health expenditures.

But this argument is misleading. Expanding access to prescription drugs actually helps bring down long-term healthcare costs — and denying sick patients needed medications drives costs up.

Cancer drugs, for instance, help keep patients healthy and out of the hospital. As a proportion of total cancer treatment costs, drug spending nearly tripled from 2001 to 2011. Over that same time, the share of total costs spent on hospital stays dropped by 25 percent. Spending more on better medications has improved cancer patient health and saved money.

Restricting drug access to trim healthcare expenses usually backfires. The average patient will skip prescribed medications if her monthly out-of-pocket costs exceed $200. Such “non-adherence” typically causes a patient’s condition to worsen to the point where she requires much more expensive medical interventions.

America already spends over a $100 billion a year on avoidable emergency room visits and hospital stays because patients abandon their medication regimens. This will drive up non-adherence even higher and exacerbate these problems.

One of Obamacare’s central promises was that sick patients would no longer suffer from discrimination. Insurers are breaking that promise by strapping vulnerable patients with huge costs and forcing them to fail on less effective treatments. Lawmakers must stop these abuses — and the Patients’ Access to Treatments Act is a good place to start.

Robert Goldberg is vice president of the Center for Medicine in the Public Interest.

From FDA News:

The FDA is taking steps toward getting more patient input during the drug development process, a move that puts the agency more in line with its EU counterparts, agency officials said at the DIA annual meeting.

The FDA has met with patients from over 20 different disease areas to hear their perspectives on how their treatments are working and how their diseases impact their lives as part of the PDUFA reauthorization process. The agency wants to glean information they don’t get from clinical trials, said Robert Temple, deputy director for clinical science in CDER, during a session at the DIA annual meeting in Washington, D.C.

Temple said patients’ chief complaints aren’t being factored into drug development or clinical trials, and they should be. Trial designs for new drugs should aim to identify the three most irritating symptoms patients with a particular disease have, so that drugmakers can focus on solving those issues. “Any endpoint that affects a patient is an endpoint that needs to be included in a trial,” he said.

Theresa Mullin, director of CDER’s Office of Strategic Programs, echoed that statement, saying the motivator for the FDA to include more patient input is the need to develop better endpoints. The agency can’t properly assess medicines without fully understanding the value, benefit and effect on patients, she said.

In the EU, patient participation is grounded in legislation, with patients having input at all levels of a clinical trial, said Enrica Alteria, head of human medicines evaluation at the European Medicines Agency. The EMA holds meetings with patient advisory and advocacy groups four times a year, and patients view labeling information and leaflets, she added.

As part of that patient discussion, Alteria said the agency consults with health technology assessment bodies early in the drug development process to gather additional patient evidence. In this respect, the EMA takes a broader lifespan approach than the FDA, she said.

The FDA is precluded by law from taking cost into account when it decides to approve or disapprove a drug. Temple said the agency wants people to have choices. “We try hard not to think about the economics.”

This week the American Society of Clinical Oncology released a tool that it claims will help oncologists measure the value of drugs and discuss their costs with patients.   The Task Force that developed this so-called value framework proclaims that the perspective of the patient is of central importance in defining value.  It notes that “patient perception of value is highly individualized, can be subjective, may change over time and include convenience and quality of life as well as cost. “

Yet value framework – which will be used to develop an app -- does not take into account any measure of the value of individual responses to treatments.  It admits that it doesn’t measure the costs saved by taking newer medicines or the productivity gains generated by treatments that require quitting a job and spending months in the hospital.

The app only measures value by more or less estimating how much would be spent on cancer drugs for the additional survival the medicine has shown in clinical trials.  (“Bonus” points are added if studies show the drug to generate fewer side effects than a placebo).  In addition, the app uses the out of pocket cost to the patient as the benchmark.

There are several defects with this approach that will likely reduce the number of lives saved from cancer.

First, the app uses average response obtained in clinical trials to measure value.  For example, most news accounts have made a big deal about how treating people with advanced non small cell lung cancer (NSCLS) with Avastin with two older drugs costs $10000 to add 2 months of survival compared to using the older drugs alone for $800.

But average response is virtually meaningless in treating cancer.  Yet the comparison does not take into account lung cancer patients with specific genetic mutations who are likely to live much beyond that average.  It can't measure the relative value of "regimens..not directly compared in clinical trials. "That’s just about 90 percent of cancer treatment combinations approved for use.   Combining Tarceva with Avastin in lung cancer patients with specific mutations adds 4 months to people who would otherwise die. 

Multiply the number of treatments by the number of genetic mutations shaping each and every tumor and then combine these targeted medicines with therapies that teach the immune system to shut down tumor growth.   Waiting for randomized clinical trials that compare each and every of these combinations would deny thousands of people potentially life saving and life enhancing care. 

Further, the Task Force claims there is no clinical benefit to increasing overall survival by less than 20 percent. That would eliminate the use of a whole host of treatments for pancreatic, brain, lung, stomach and cancers in use or being studied today.  By way of comparison, the increase in average life expectancy for people with AIDS was less than 20 percent a year between 1987 and 2000.  The ASCO app would have completely rejected these increases, which in turn would have denied life to thousands of people alive today.

Indeed, the Task Force asserts – without evidence – that the use of new drugs is being driven by “sometimes unrealistic patient and family expectations that lead clinicians to offer or recommend some of these services, despite the lack of supporting evidence of utility or benefit.”  For all it’s lofty rhetoric about the primacy of patient value, the Task Force sneers that cancer patients “overestimate the benefits of treatments that sometimes extend life by only weeks or months or not at all. “  In fact the evidence suggests just the opposite. 

As a result, the app does not calculate the value of hope as measured by the significant progress made in reducing the death and cost associated with cancer. Since 2000, new cancer drugs have been responsible for nearly 90 percent of the decline in cancer death rates.  The number of cancer survivors has increased from about 9.8 million to nearly 14 million today.  Over that time we have added 26 million additional life-years worth over $2 trillion in better, healthier life.

As the number (and price) of targeted treatments have increased the percent of health care dollars devoted to cancer spending has remained at 4.6 percent.  How? New cancer drugs reduce spending on more expensive medical services.   In 2001, 64 percent of cancer care went to hospitals and only 3.6 percent to drugs.  By 2012 drug spending ‘skyrocketed’ to 11.3 percent of cancer costs but hospitalizations dropped to 38 percent of care.  That’s why a government study concluded, “The net value of (cancer) treatment has grown substantially, consistent with medical technology improving over time and leading to better health outcomes at a lower cost per patient.” 

All these benefits have been generated by new medicines that are only .7 percent of health spending.   The Task Force acknowledges that new medicines are a small fraction of total costs but then asserts that use of new medicines is driving up premiums, reducing wages, etc., when the evidence clearly concludes the opposite is the case. 

If the app completely disregards the evidence of patient preferences, cumulative life years saved from small gains in survival, the reduction in treatment costs and increase in the economic value of health, what it’s good for?

It turns out that the app will be very helpful to health insurers who want to
“evaluate the relative value of new treatments” as they develop “benefit structures, adjustment of insurance premiums, and implementation of clinical pathways and administrative controls.” 

The task force acknowledges that one important effect of these benefit structures is “patients will find themselves increasingly responsible for a greater proportion of the cost of their health care. Cost shifting or sharing can occur through the increased use of high-deductible policies and larger copayments. “  Which appears to be acceptable to ASCO since it does not take into account evidence of how such practices, by reducing the cost of drugs, drives up the number of deaths and the cost of care.

Health plans are forcing cancer patients to pay thousands of dollars out of pocket for new medicine.  And they are requiring them to ‘fail first’ on less costly treatments that insurers want to claim are as, on average, effective as new therapies.  Conveniently insurers are already using the ASCO calculation to restrict access. 

Meanwhile, the Department of Health & Human Services has warned “placing most or all drugs that treat a specific condition on the highest cost tiers discourages enrollment by individuals based on age or based on health conditions, in effect (is) making those plan designs discriminatory.”

If the app is used by health plans as ASCO hopes – to support the development of pathways and administrative controls – it will be powerful tool for trampling on the civil rights of cancer patients.

ASCO has asserted that oncologists should be aware of the value of an intervention in terms of societal cost when making treatment decisions. It assures patients that this does not conflict with the Hippocratic oath.   It doesn’t just conflict; it out right violates that solemn pledge.

Bass Hole

  • 06.22.2015

The Patent Trial and Appeal Board of the U.S. Patent and Trademark Office has accepted a request from Celgene to consider sanctions against the Coalition for Affordable Drugs, an entity controlled by hedge fund manager Kyle Bass.

Celgene filed a motion seeking sanctions as part of its response to an inter partes review (IPR) petition the Coalition filed that attempts to invalidate some of Celgene’s patents on Thalomid thalidomide. 
In a document filed with the PTAB, Celgene accused the Coalition of using the IPR process to affect the value of public companies. "This is not the purpose for which the IPR process was designed,” the company wrote. Celgene also alleged that before the IPRs were filed, someone associated with the Coalition “threatened to file IPRs against the challenged patents unless Celgene met their demands.”

Celgene wrote that when it did not pay, the IPRs were filed. 
Celgene described the alleged attempt to obtain payment to forestall IPR challenges as an “abuse of process and misuse of the IPR proceedings.” 
In granting Celgene’s request to file a sanctions motion to dismiss the petitions, the PTAB said its decision “is not a decision on the merits of Patent Owner’s allegation of abuse of process.” The board set a July 30 deadline for Celgene to submit a brief supporting its request.

Per a report in BioCentury, the board’s decision is likely to be announced three months after the Celgene brief is submitted, according to Matthew Kreeger, a partner at Morrison & Foerster who is an expert in patent law.
The Celgene request puts the PTAB and the IPR process “in uncharted waters,” Kreeger told BioCentury. He noted that if the decision hinges on a demand for payment made prior to filing the IPR, then it might not affect other IPRs filed by Bass or other investors. 

Did somebody say “PDUFA VI?”

With the upcoming PDUFA renewal, 21st Century Cures uncertainty, and a leadership transition at FDA, it is a dynamic and uncertain time in the biopharma industry. The outcomes of these key policy debates, along with others, will impact how industry pursues drug development and works with patients and regulators to improve public health.

To advance the conversation and propose practical ways forward, a panel of regulatory experts, including former industry and FDA leaders, got together in Boston on May 20, 2015 to share their best thinking on pressing regulatory policy issues. And there was also a fair degree of  appropriate (and useful) venting.

The panel was chaired by Tim Franson, MD (Chief Medical Officer for YourEncore, Board Member for the Critical Path Institute, and current President of the USP Convention. Tim was a key contributor to PDUFA V, particularly as it relates to rare disease incentives, and is generally considered one of the  “founding fathers” of the PDUFA concept.

Panelist #2 was Joe Lamendola, Ph.D., the former VP of U.S. Regulatory Sciences for Bristol-Myers Squibb, responsible for over 20 global approvals across 10+ therapeutic areas. Over a 25+ year career, Joe also led the Regulatory Policy and Intelligence Organization for BMS, which was responsible for assessing and influencing regulatory policies, including PDUFA V and multiple therapeutic guidances.

And rounding out the panel was yours truly – the junior associate.

It’s called a regulatory RANT (Relevant Assessment and New Trends) and is a great read to get ready for PDUFA season. The full document can be found here. It's an entertaining and informative read with some relevant assessments and aggressive suggestions

Get ready. Get set. Go!

At the recent BIO confab in Philly I was honored to moderate the panel on “Public Sector Biotech Initiatives in Middle East and North Africa.” I was joined with public officials and regional experts (Marwan Abdulaziz Janahi, Executive Director of Dubai Biotechnology and Research Park, DuBiotech, Samir Khalil, Executive Director of Middle East & Africa, PhRMA, Tarek Salman, Assistant Minister of Health and Population for Pharmaceutical Affairs in Egypt, David Torstensson, Senior Consultant, Pugatch Consilium, and Jeffrey Kemprecos, Executive Director, Emerging Markets Public Policy, Merck).

A key take-away was that one of the key drivers of biotech investment in the region (and, indeed, any region) is sound regulatory policy. This was most directly addressed by Vice Minister Salman who discussed how the Egyptian regulatory authority had upgraded both its processes and procedures to reward innovation with a review pathway that is more predictable and timely.

As the panel’s moderator, I had the opportunity to present opening remarks.  Here’s what I had to say:

When it comes to biotech incentives in the MENA region, there are many languages, priorities, pressures, and impediments (social, political, cultural) to consider.

In April 2015 I spent three fascinating days in Sharm El Sheikh, Egypt at the Second Arab Conference on Food & Drugs.

Delegates from the Levant to Morocco had a lot to say and share. The fundamental take-away was that the Arab world is serious about coordinating their efforts in healthcare in general and in regulatory affairs specifically. “Convergence” and “harmonization” were the two key words of the event.

(The Middle East/North Africa Region – MENA – consists of 22 nations – but just 2% of global pharmaceutical sales.)

Biotech initiatives are a global opportunity, but they take many local forms --because public health is a global fraternity with national priority, local impact and global implications.

Biotech Initiatives take many forms:

  • Investment programs
  • Clinical trial incentives
  • Reinvention of medicine and device regulation
  • A high regard for Intellectual Property Rights
  • An embrace of the concept of both price and value
  • Transparency of laws and regulations
  • And the rule of law

Most importantly, national biotech initiatives rest on the foundation of the importance and urgency of healthcare innovation.

But Biotech Initiatives mustn’t falter under the false banner of Biotech Imperialism. Initiatives must benefit all parties, transnational, national – and local.

That means “doing the right thing.” It’s about “Nazaha” – Integrity.

There's been proverbial flood of articles on the high cost of drugs.  

This happens everytime a bunch of new medicines hit the market.   But how about putting the amount into context or discussing value.   As any baseball fan (or FBI agent investigating the St. Louis Cardinals hack of the Houston Astros scouting database) knows context is everything.   

So for instance, the Pharmalot blog posted:  How Much?! Global Prescription Drug Sales Forecast to Reach $987B by 2020

But the headline obscures and deflects attention from the fact that between 2015-2020 Rx spending will actually decline as a total share of global health spending. 

The same designed neglect applies to the breathless discussion of "skyrocketing drug prices or drug costs".   

You wouldn't know that drug spending as a percent of total health spending in the US will remain FLAT between now and 2025 even as specialty drug spending becomes half of total Rx expenditures.

Is it too much to ask for some context.  It took me, a C minus math student, five minutes to figure out percentages.  Why can't journalists and policy types who are a lot better at this stuff than I am run the same numbers?  


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.

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