Health Business Blog

Generic drugs have been an effective cost containment solution for traditional, small molecule pharmaceuticals. As large molecule biologics proliferate and take up a growing share of medical spending, we also increasingly need cost containment.

The path we’re on now in the US and Europe is to ape the experience with small molecule products by introducing generic versions as patents expire. As I’ve discussed in the past, this is a bad idea. Development costs are high, manufacturing is notoriously difficult, and the products won’t be identical anyway. That’s why the products are called “biosimilars.” As a result the products are going to be expensive –we won’t see nearly the costs savings as we do with small molecule products, and FDA will be stretched too thin monitoring the manufacturing facilities. Instead I propose to allow branded products to maintain their monopoly after patent expiration, but to regulate pricing.

Recently I’ve been thinking this through a little bit more and have become even more troubled by the idea of biosimilars. In particular I’m concerned about the ethical and practical issues of conducting clinical trials for these products.

Patient recruitment is a challenge for most clinical trials, and as a result studies are frequently delayed. A key problem is that few development-stage therapies offer significant improvements over what’s already on the market, so there is limited enthusiasm to participate in a study that has little reward but also includes risks. Doctors don’t feel comfortable recommending that patients enroll, and patients are understandably hesitant, too.

The challenge for bio-similar trials will be even harder. It’s hard for me to understand why a patient would want to join a trial just to help prove that a new therapy is very similar to an existing therapy. I suppose it’s possible that a biosimilar product could be a little better in specific instances, but mostly these trials will just attempt to prove a new drug is the same as the old.

The ethical issue is related to the practical one. If there’s no upside to joining a trial, is it reasonable to ask a patient to take on any extra risk, such as the risk that the product doesn’t work or makes them sicker? I kind of doubt it.

At the end of the day, I feel more strongly than ever that biosimilars and biogenerics  are a foolish and pricey prospect.

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By David E. Williams of the Health Business Group.

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Medication mix-up’s are a well-known source of errors and harm in the hospital. So we shouldn’t be surprised that similar errors occur in other settings, including the home. Medication Errors in the Home: A Multisite Study of Children With Cancer in the journal Pediatrics documents the high rate of errors in at-home administration of medications for pediatric cancer.

Error types include administering at the wrong dose or frequency, incorrect label, missed doses, using expired medication, and using the wrong administration technique. Many of the errors have the potential for harm, some caused actual harm.

From the article:

“In our study, parent administration errors were often caused by miscommunication between parents and clinicians or between in-home caregivers regarding changes in oral chemotherapy dose. Frequent changes in dose, which caused the bottle label to be outdated, were often a root cause of parent errors.”

I’ve seen similar things happen outside of oncology. Sometimes a patient is taking 10 or more medications and supplements, so it’s very hard for a parent to remember what to do even when they are well educated, organized, and have the best of intentions.

I’d like to see someone come up with a comprehensive solution to managing multiple, frequently changing pediatric medications in the home setting. I don’t have a specific solution in mind, but would be very appreciative if one came on the market.

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By David E. Williams of the Health Business Group.

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It’s hard for policy wonks, politicians or health plans to be viewed credibly when promoting health care cost containment. Discussion quickly turns to “rationing,” and “death panels,” which no one wants to be associated with, and as a result the federal government has done almost everything possible to make sure cost effectiveness and overall costs are ignored in policy making.

Those closer to the action know better. In particular:

• Many costly treatments aren’t worth the money
• New treatments with tiny or no benefits often cost a multiple of existing therapies
• Despite their reputation for penny-pinching, health plans are often not aggressive in negotiating price
• Patients are already suffering mightily from high costs –and it impacts quality of life and survival as well as financial health
• Society as a whole can not afford to pay the high prices charged for so many of the new therapies

So it’s encouraging to see a perspective in the journal Blood endorsed by more than 100 experts. The piece, The Price of Drugs for Chronic Myeloid Leukemia (CML); A Reflection of the Unsustainable Prices of Cancer Drugs: From the Perspective of a Large Group of CML Experts,  is very useful because it comes from people who know what they’re talking about and who have traditionally been sympathetic to drug makers and unperturbed about costs.

Here are some excerpts that are noteworthy for their candor and clarity:

“If drug price reflects value, then it should be proportional to the benefit to patients in objective measures, such as survival prolongation, degree of  tumor shrinkage, or improved quality of life. For many tumors, drug prices do not reflect these endpoints, since most anti-cancer drugs provide minor survival benefits, if at all.”

…

“In the US, prices represent the extreme end of high prices, a reflection of a “free market economy” and the notion that “one cannot put a price on a human life”, as well as a failure of government and insurers to more actively negotiate pricing for anti-cancer and other pharmaceuticals, in contrast to practices in other parts of the world.”

…

“In Europe and many developed countries, universal health coverage shields patients from the direct economic anxieties of illness. Not so in the United States (US) where patients may pay an average of 20% of drug prices out-of-pocket(about $20-30,000 per year, a quarter to a third of an average household budget), and where medical illnesses and drug prices are the single most frequent cause of personal bankruptcies. High drug prices may be the single most common reason for poor compliance and drug discontinuation, and the reason behind different treatment recommendations in different countries.”

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By David E. Williams of the Health Business Group.

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USA Today has a full page article on the rise of heroin addiction in the suburbs, but adds absolutely nothing to what’s already widely known. (See, for example, my post on the topic from early 2012.) Teens and adults start by abusing the painkiller oxycontin, which is available by prescription, then turn to shooting heroin once they figure out how pricey it is to acquire oxycontin on the street.

The article presents no real ideas on what to do about the problem. If anything the article implies that it would be better to make oxycontin more widely available in order to stem the use of heroin. That’s a nonsensical approach as far as I’m concerned.

There are alternative approaches that might be more promising.

One idea is to establish better guidelines on the prescribing of painkillers after surgery. Many patients –maybe you’ve been one of them– receive an overly generous supply of oxycontin or vicodin after a minor surgical or dental procedure. Sometimes the patient gets addicted from that initial supply, other times the extras end up in the family medicine cabinet where teens might find them and try them out. It’s not always obvious how to dispose of these medications, which contributes to them hanging around.

One hurdle to overcome is that follow-up visits are inconvenient and also not very profitable for doctors. Perhaps if there were quality measures associated with good practices that would change the equation and tighten the initial supply.

Another issue relates to so-called “drug seekers.” We’ve all heard about drug seeking patients who come to the emergency room to get drugs. There are IT systems coming online that can at least identify such drug seekers and alert doctors, but this only works if the systems are consulted, which may not happen when middle-class patients are involved. It’s easy to label patients as “drug seekers,” which makes them sound like bad people. Some are. But many others are patients who are somewhere down the path toward dependency. They’re not trying to become oxycontin addicts and certainly aren’t looking to move to heroin. Rather than turning people away it would be better to have a path to refer these patients into treatment and then to track their progress.

There are great opportunities for physicians, payers, employers, consumers and pain management experts to work together to develop a more comprehensive view of the problem, to develop a strategy to address it, create new quality and safety measures related to achieving the strategy, and align incentives so that physicians are rewarded for doing the right thing.

We won’t solve the problem of painkiller abuse in one shot. But it’s reasonable to start by tightening up on the relatively easy places, such as cutting down on the distribution of unneeded post-surgical pain meds and figuring out how to better direct “drug seekers.”

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Pharmaceutical Benefits Manager (PBM) Express Scripts released a report claiming that more than $400B in annual pharmacy expenditures are wasted and that the greatest waste occurs in the poorest states, i.e., the South. The map is pretty striking with the North the best, middle next, and South the worst.

Express Scripts breaks waste into 3 categories:

• ~$60B wasted on high priced meds when cheaper equivalents, (e.g., generics) were available.
• ~$95B from not using “the most cost-effective and clinically appropriate pharmacies, including home delivery and specialty.” This is further broken into savings from lower drug costs (~$35B ) and savings from higher adherence through those channels (~$60B)
• ~$270B from avoidable pharmacy and medical expenses from low adherence, independent of the waste included in the second bullet point

All three bullet points are self-serving, especially the second one, since it describes the line of business that is most profitable for Express Scripts. (On a side note, I’ve noticed that PBMs have replaced the term “mail order” with the friendlier sounding “home delivery.”)

Nonetheless these numbers are probably supportable and directionally correct.

There’s another big category, too, which is drugs that should not have been prescribed in the first place. I don’t know how big that category is but I bet it rivals point number 3 in magnitude. Express Scripts benefits financially from the perpetuation of that kind of waste, however, which could explain why it’s not included in the study.

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A reader made an interesting comment on an old post, so I’m highlighting it here in the hopes that more might see it.

Anyone heard about this?

“Has Genenetch looked into the practice of retinal specialists multi-dosing Lucentis, i.e., administering up to 4 doses, from a single use vial? Some providers argue that, since each vial contains 5 doses, they can do this by drawing out the doses into multiple injection needles (other the one injection needle that Genetech supplies in the package). It seems that there is a danger here for microbial infection, and also some concern about the provider lowering there actual costs of purchase by adminstering multiple doses from a single use vial. Comments?”

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This is the transcript of yesterday’s podcast with Castlight Health President John Driscoll.

David E. Williams:  This is David Williams from the Health Business Group.  I’m speaking today with John Driscoll, president of Castlight Health.  John, nice to speak with you today.

John Driscoll:  Great to be with you, David.

Williams:  John, we’re going to talk about two topics today.  One is the new Castlight Pharmacy product and another is Castlight’s emergence in my home market of Massachusetts with its first health plan deal with Harvard Pilgrim.

Let’s talk about pharmacy first.  What is this Castlight pharmacy product?  What is the need that you’re addressing?

Driscoll:  As you know, David, we are absolutely aggressive about making sure that consumers have real time information that allows them to make the best choice.  And we are focused on the lack of cost and quality information in marketplace.  That’s an entirely new area for consumers and patients.

The one area where consumers are ready and able to make smart purchasing decisions is pharmacy.  So we thought it was important to invest in a new capability to allow consumers to make decisions right after they’ve left the doctor’s office with their mobile device or when they’re searching on the Internet.

Consumers are very focused on how much drugs cost.  And many patients are trading off drug costs against their weekly budget.  So we thought it was important to invest in this capability and roll it out quickly because we knew that patients and consumers needed it now.

Williams:  Pharmacy, as you said, is an area that consumers are really ready and able to engage in it.  It seems like that’s partly because it’s a discrete product that they identify as opposed to going to a hospital and not knowing what set of services they’re receiving.  But it also is maybe because it’s an area where there’s been some focus both from health plans and from PBMs.  I know you spent some time in that industry.  So can you distinguish what Castlight is doing from what’ already out there?

Driscoll:  It’s clear in other categories where we are providing cost information for the first time to patients.  But even in the pharmacy world, even with all the information and tools that health plans provide, they’re not focused on providing real time information at the point of decision in a way that impacts consumers immediately.

That’s probably a function of the fact that at Castlight we have a major focus on consumer behavior. Frankly, this is all we do.  We provide the best tools with the best information. So much of how you get a consumer engaged and keep them engaged is providing simple consumer-oriented tools that are as sophisticated as a consumer needs.

It could be as simple as making sure they know the difference between branded and generics and home delivery versus a local pharmacy.  Those basic choices –and providing them in a way that consumers can act on– extends the value of their benefit at the point where they are given a script.

Those tools really don’t exist in the marketplace in a way that really engage the consumer.  There’s an art to putting together the consumer interface and the product and then making sure that we’ve got real time information that is directly relevant to where the people are in their benefit.

Williams:  Unlike with medical benefits, there seems to be a fair amount of competition at the retail level on drug prices. So you have Wal-mart, which kicked things off with the $4 generic program and you see it extended into some pharmacies that offering low priced Lipitor or free antibiotics.  Can that information be integrated into Castlight pharmacy or does that stand outside of the system?

Driscoll:  What we are focused on is what’s covered under the insured benefit. Any part of the benefit that employers are paying for is integrated into the tool.  And it’s integrated in a simple way to enable the consumer to make a decision at the point of shopping.

Williams:  Is there a connection between what you offer on Castlight Pharmacy and the medical part of the benefit or are they standalone tools that are used separately?

Driscoll:  They’re really standalone tools, but the great thing about pharmacy is the richness of the data and the fact that the consumers are ready to comparison shop. What’s hard is integrating in a simple way that enables the consumer with limited time to make a decision.  And that’s really what this component of the product is.  It sits on top of the traditional Castlight transparency product.

Williams:  Does Castlight Pharmacy serve the Medicare Part D market or is this more for commercially insured patients?

Driscoll:  Today, our pharmacy product is focused exclusively on the commercially insured, but we absolutely are going to be looking at Medicare and Medicaid, because there are opportunities for consumers to save in every market.

Williams:  My understanding is that with Castlight, a consumer would need to have their employer be a customer of Castlight in order to access the tools.  Is that the case with the pharmacy product as well or is it available to individual consumers?

Driscoll:  Yes.  The employer has to buy it. It’s through the employer purchasing that enables us to gain access to information that powers the tool and allows the employee to understand exactly how much is covered and exactly how much things cost.

Williams:  Let me turn to another topic: health plans. I think about Castlight as somewhat of a competitor or threat to health plans. You may be revealing information to their customers that they perhaps should have revealed themselves.  And yet it seems you’re starting to work with health plans, beginning with Harvard Pilgrim, which is often ranked as the number one health plan in the U.S.

Can you talk about what you’re thinking with health plans and then what specifically is going on with Harvard Pilgrim?

Driscoll:  First of all, I would say that health reform is a team sport. We look at all of the health plans that work with us as partners. We’re all driving better outcomes and lower prices; the vast majority of people in the health insurance industry want to do that.  We feel like we’re all playing for the same cause.

We will partner with health plans in small ways or large to deliver value for their covered lives.  And we’re very excited to be able to adapt the cost estimator, our technology and our approach and to work with such a great partner as Harvard.

We look at the health plan marketplace as a real opportunity for Castlight.  In some cases, we’ll be working with employers. In other cases, we’ll be working directly for the health plan and with employers in that marketplace.

For us, it’s just a variation on what we think is a huge need in the market place – for better information and an empowered consumer.  As it happens, there’s a complete values match between what Harvard wants to achieve in the market place and what we’d like to achieve in the market place.  They are very focused on transparency and may want to empower their approach to have Harvard Pilgrim.

Williams: John, it’s interesting to see that you signed your first health plan in Massachusetts, which is a market that has led the way with health reform. The Affordable Care Act was modeled on the Massachusetts plan.  We’ve also been quite active in transparency.  Did the legislation and the move in the policy sector toward transparency have anything to do with why Massachusetts is the first market you’re entering with health plans?

Driscoll:  There is no question that the health plans in Massachusetts are particularly progressive. And obviously, Harvard Pilgrim is one of the best and the highest-ranked plans in the country.  I think it is no surprise that we are finding great partners in the most progressive markets in the country.  What’s interesting about the Massachusetts marketplace is its employers, its legislators, as well as health plan leadership that are driving a much more transparent system.  And I think where you’ll see transparency not just on cost but also on quality and outcomes.  It’s also a place where there’s a lot of very interesting and enlightened thinking around how to compensate for value and how to measure value.  It’s really a laboratory for the rest of the country in health reform innovation.

Williams:  Yes, it’s interesting.  We also had one of the earlier all-payer claims databases in Massachusetts.  My impression is there’s been a lot of information put into that database. The plans have submitted that information, but not all that much has come out of it.  Does Castlight complement the all-payer claims database?  Is it a substitute for it?

Driscoll:  I think it complements the all-payer database.  There’s still a fair amount of data gaps in every public database that’s been put up, but every time another database is developed and is improved, it’s another step towards having a more transparent system. That means a fairer system, a more accurate system and a system where not just software companies are selling services, but the patients and doctors and health plans and everyone else will gain from having a system where you can measure and then drive better results.

Ultimately, Castlight is more interested in getting access to information that historically folks haven’t had access to, making it actionable for employers and employees in a way that helps them drive better outcomes and lower costs.  And as of today, we need to be able to get more information and provide it in a particularly elegant way and constantly improve it. The bigger Castlight gets, the more we know about the consumers and we can create tools that have meaningful impacts on cost and quality.

Williams:  You talked about health reform being a team sport. Clearly Castlight and health plans and employers are on the team.  Are providers part of that team as well and if so, how do they play?

Driscoll:  They are.  We’re only in the first few innings of transparency for employers and employees, but I’m not even sure it’s the first inning of transparency for providers.  The majority of providers want to do the right thing but don’t have actionable information.  They don’t have tools they can use.  They don’t have tools that are meaningful, that fit their workflow and that can furnish providers with better information on cost and quality.

One of the concerns I have about some of the more progressive markets like Massachusetts is making sure that providers have access to that same information that Castlight currently provides to employers. Without that, to compensate doctors and hospitals on performance metrics seems unfair.  We will only have a fair system that can function and create better performance if providers have access to the same kinds of information that Castlight is currently providing to employees.

Williams:  Obviously, physicians are under a lot of pressure.  They’re being asked to do things that they weren’t asked to do in the past and didn’t learn about in medical school. They’ve got a lot of requirements to adopt health information technology, a lot of compliance requirements, and an imperative to do more with less.  And as I look at a lot of information systems that are out there, the providers really don’t have such great access to the kind of transparency data that you’re describing.

What’s the solution there?  And how long does it take?  And are there ways to see milestones along the way that may indicate longer term success?

Driscoll: Providers are only going to use tools that are meaningful.  They want to save money for their covered lives and the patients they serve and they want to create better outcomes.  But they don’t have information right now and certainly nothing’s been built into workflow. I think the next step is working, these companies life task is working with health plans to provide their network participants better information and to make certain that it is meaningful, simple and doesn’t slow down the hard work that doctors are doing.

The promise of companies like us or the promise of technology is to create this frictionless change where people can actually do more with less hassle. If you can integrate software tools into physician and hospital practices with the kind of information that the health plans have, doctors will make wiser choices.  There’s a real opportunity right now but I’m concerned that without those kinds of tools, that putting doctors on new kinds of performance metrics like bundling, or bonusing them more on value is unfair unless they have the information to make wise choices.

Williams:  I’m a member of a health plan in Massachusetts, a different one than the one you described, and my primary care physician is part of a contract that does pay based on value. I’m wondering what kind of information would it be useful for her to have that she probably doesn’t now that could help me and could help her?

Driscoll:  I don’t know what information your doctor has access to, but certainly every doctor needs to have better feedback for the covered member that they’re taking care of, what’s covered and what’s not.  For the doctors they refer to, what are their historic outcomes, how frequently have they done certain procedures, how satisfied are patient, and how do they fit into an episode of care? In a fee-for-service world there are informal referral networks.  We want to create a system leveraging information that creates an informed referral.

Williams:  I’ve been talking today with John Driscoll, President of Castlight Health.  We’ve been talking about the new Castlight Pharmacy product and also talking about Castlight’s first health plan customer, Harvard Pilgrim, in Massachusetts.

John, thanks so much for your time.

Driscoll:  Thank you.

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Health care transparency leader Castlight Health has launched a tool to manage pharmacy costs and signed its first deal with a health plan: Harvard Pilgrim. In this podcast interview, Castlight president John Driscoll and I discuss:

• What the new pharmacy tool adds to the offerings already on the market from health plans and PBMs
• Why the first health plan customer is in a market (Massachusetts) that’s already a leader in transparency
• The role of health care providers in the transparency movement

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David Williams interviews Castlight's John Driscoll [ 17:17 ] Play Now | Play in Popup | Download

In Europe, reimbursement decisions for drugs often include explicit consideration of cost effectiveness and a comparison of the efficacy of the new drug with products that are already available. In the US, such considerations are excluded, at least for Medicare, which is the biggest payer. In the latest Health Affairs, Joshua Cohen, Ashley Malins and Zainab Shahpurwala conclude that the European approach leads to lower costs, better access to therapy for patients, and better outcomes –at least in some cases.

I asked Cohen –a senior research fellow at the Tufts Center for the Study of Drug Development– to comment on some of the findings.

Why did you base your research on patient access rather than market availability?

I’ve been studying patient access for over 10 years. I try to distinguish between key dimensions of patient access. Broadly, patient access is a function of: i. market availability (off-label uses are an exception to the rule); ii. coverage by payers; iii. patient out-of-pocket costs. Market availability captures one element of access. It is a necessary, but insufficient condition of access given that the vast majority of cancer drugs are paid for by third party payers.

From the patient standpoint, what are the advantages and disadvantages of the US v. European approaches?

The biggest advantage in the U.S. versus Europe with respect to cancer drugs is faster market availability of a greater number of drugs. Two rather stunning facts stood out: 1. None of the common subset of 29 drugs were approved in Europe before the U.S. And in most instances the lag was at least 4 months. 2. At the same time, for drugs licensed by the EMA and approved for reimbursement by the national health authorities there were hardly any out-of-pocket costs for patients in Europe. Contrast this with co-insurance percentages of as high as 40% for some drugs in the US. There are medications with annual price tags of over $20,000 –and 40 percent of  $20,000 is a lot of money to shell out, especially for those on fixed incomes.

The comparative outcomes information you cite in the article is very old and excludes drugs approved since 2002. Why is this the case? Is there any way to look at more recent information?

The articles themselves are not old. They are recent publications (2009, 2010, 2011). However, if one looks carefully at the time period during which survival data were being measured it becomes clear that the newer vintage drugs were not included in the studies. Hence, one cannot conclude that better survival statistics for a number of cancers in the U.S. are due to better access to newer cancer drugs. Until we have data showing survival that can indeed be attributed to better access to newer drugs, we are left to speculate. My hunch is that better access in the U.S. to newer cancer drugs (i.e., faster and greater numbers of approvals, as well as fewer coverage restrictions) has been beneficial to some patients, as has improved screening and earlier diagnostic work-up.

In the timeframe you considered, 41 oncology drugs were introduced in the US but only 31 in Europe. Are there clinically significant products that make it to market in the US but not elsewhere? Can you provide an example?

Provenge (sipuleucel-T) comes to mind as a drug with a lot of fanfare in the U.S. It was approved in 2010 by the FDA, yet still not approved in Europe. At the same time, it should be said that there are certain differences in regulatory mechanisms that have benefited market uptake of a number of drugs in Europe, including Iressa (gefinitib). Iressa has led practically a moribund existence in the U.S., while in Europe, as a result of EMA approved of a companion diagnostic in 2009 – an EGFR mutation test kit – sales have increased steadily.

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By David E. Williams of the Health Business Group 

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Sanofi is testing a new drug for myelofibrosis and is presenting details of a phase II study at the American Society of Hematology’s annual meeting this week. I asked Tal Zaks, M.D., Ph.D., VP, Oncology Clinical Development, Sanofi Oncology to provide further insights regarding myelofibrosis and the company’s JAK2 inhibitor, code-named SAR302503.

In this interview (conducted by email) Dr. Zaks describes the disease, existing treatment strategies and treatment gaps. He also comments on the delicate topics of head-to-head comparisons with ruxolitinib (a competing JAK2 inhibitor) and affordability.

1. How many people are affected?

Myelofibrosis (MF) is a rare, debilitating and life-threatening progressive malignant blood disease characterized by abnormal blood cell production and fibrosis (scarring) within the bone marrow.  Scarring in the bone marrow interferes with blood cell production; the spleen and liver try to produce and store extra blood cells which can cause these organs to become enlarged.

Most patients with MF have greatly enlarged spleens that can result in a range of symptoms with dramatic impact on quality of life.  These include fatigue, abdominal pain, night sweats, feeling full without eating, cough or shortness of breath and decreased physical activity.  Other signs and symptoms of MF include anemia, thrombocytopenia, weight loss and severe itching.

Primary MF is one of the three main types of slow growing malignant hematologic diseases called myeloproliferative neoplasms that cause blood cells to grow abnormally in the bone marrow.  The other two are polycythemia vera (PV) and essential thrombocythemia (ET) and can precede the development of secondary myelofibrosis. PV and ET are characterized by too much production of red blood cells and platelets, whereas in MF there is not enough blood being produced by the bone marrow. All three types are characterized by genetic mutations in the JAK2 enzyme leading to abnormally activated JAK2 signaling in the cells that produce blood components.

The exact prevalence of MF, PV and ET is not known. The latest research, presented at the 2012 American Society of Hematology Annual Meeting, estimates that the prevalence of MF ranges from 4.2 to 5.6 per 100,000 people in the U.S., or approximately 15,000 patients, suggesting that the prevalence is higher than has been reported to date. Prevalence estimates in Europe are less clear.

2. What are the existing treatment strategies?

There is an unmet need for new MF treatments.  Most available therapies are palliative, providing some relief of symptoms, but do not impact the underlying disease. The primary goal of the treatment is to control symptoms, including low blood counts and the size of the spleen. Treatment may involve blood transfusions, radiation and chemotherapy, recombinant erythropoietin to help stimulate red blood cell production, and spleen removal. Bone marrow transplants are the only treatment that can cure MF, but have significant risks.

3.  What are the treatment gaps that Sanofi’s development-stage JAK2 inhibitor is meant to address?

Currently, there are limited treatment options for patients with MF.  Most available therapies are used to manage symptoms, and none have been shown to reduce or reverse the underlying disease.   Many patients do not respond to currently available therapies, and even among those who initially respond, many discontinue treatment due to intolerance or resistance.

SAR302503 is a selective JAK2 inhibitor  that has shown activity in patients with myelofibrosis from the initial phase I study. New Phase II data presented at the 2012 American Society of Hematology Annual Meeting help define the relationship between the dose ofSAR302503, its ability to inhibit molecular signaling of JAK2 in the blood of the patients, and its ability to ameliorate the spleen size and alleviate symptoms.  These data also confirm a very good pharmacological profile that allows once-a-day dosing. The pivotal Phase III study, JAKARTA, is currently underway; enrollment of 289 patients has been completed and results are expected in the second quarter of 2013.

4. What is the best-case expectation for the drug and how many patients will it be relevant for?

Sanofi does not publicly speculate about potential sales.  There remains a substantial unmet need for effective and safe treatments for people with primary and secondary MF.  We believe SAR302503 may provide a benefit for patients with these difficult-to-treat diseases.

5. Does Sanofi intend to compare this new drug head-to-head with Ruxolitinib? If so, when? If not, how will doctors and patients be able to decide which one to use?

The Phase III pivotal study has been designed with similar endpoints to those that led to the approval of ruxolitinib and will be able to clearly demonstrate the safety and efficacy of SAR302503. There are no plans for a head-to-head trial at this time; however, a Phase II trial is underway evaluating SAR302503 in patients who were previously treated with ruxolitinib but who either could no longer tolerate ruxolitinib or are resistant to it, although results won’t be available for a few years.

6. Hematology is a fairly small specialty in terms of number of physicians and patients, yet it seems to be attracting a lot of interest lately from pharma companies and from Wall Street in particular.  Why is that?

Hematology is an area where we believe we can bring a substantial benefit to patients. Unlike other cancers, there is a close correlation between the clonal origin of the cancer cell, the molecular aberration unique to the cancer, and the clinical sequelae of the disease.  For example, hyperactivation of JAK2 in blood progenitor cells that produce red blood cells and platelets is associated with conditions of both marrow overproduction (MF and ET) and underproduction of these blood components, with the expected clinical outcomes (i.e., excessive tendency to clots in the former and excessive tendency to fatigue and bleeding in the latter). Consequently, the ability to therapeutically inhibit JAK2 signaling in the appropriate disease and patient population enables more rational and rapid drug development. This is exemplified by the development of SAR302503, which has progressed quite rapidly from Phase I to Phase III.

This correlation is not unique to JAK2; as can be seen from our hematology pipeline, Sanofi is developing a broad range of treatments for various hematologic malignancies including myelofibrosis (MF), acute myeloid leukemia (AML), non-Hodgkin’s lymphoma (NHL) and acute lymphoblastic leukemia (ALL).

7. This product seems like it has the potential to be helpful to patients, but also a drug that would need to be used for a long period of time and that would be expensive. What are your thoughts about the affordability of this kind of therapy in an era of health reform in the US and economic challenges in Europe?

Myeloproliferative neoplasms are chronic, progressive diseases and thus require long-term treatment.  New research to be presented by Wang et al., at this year’s ASH annual meeting shows that MF is associated with a significant burden of illness, incurring about five times the health care expenditures than similarly matched patients without MF.  MF patients had higher overall comorbidities (mean CCI of 2.1 vs. 0.9), were hospitalized more often (34% vs. 11%), had higher number of average hospital days (7 vs. 1 day), and had more outpatient office visits (58 vs. 22).

MF-associated medical resource utilization and the corresponding expenditures for those services are substantive.  Today, treatment for MF is largely palliative using a variety of medicines and other medical interventions.   New and more effective treatments, especially those that may modify the course of the disease, have the potential to significantly reduce the burden of illness associated with MF.

One of our top priorities at Sanofi is to provide rapid and broad access to our medicines by demonstrating their value to both patients and payors; consequently, we integrate direct input from payors into our clinical development plans. Once a drug is approved and launched, Sanofi has programs to provide assistance to patients having difficulty accessing their medications.  These include a comprehensive patient access program called Patient Connection, which provides product-support services for indigent and functionally uninsured patients for future dates of service, reimbursement-support services to practice managers, a Drug Replacement Program (DRP) for insured patients who have been denied coverage and alternative services to assist patients and caregivers.

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