UPDATE 2-Merck says hepatitis C drugs to lose breakthrough status
By Ransdell Pierson
Feb 4 (Reuters) - Merck & Co on Wednesday said the U.S. Food and Drug Administration intends to rescind its "breakthrough therapy" designation for the company's experimental combination treatment for hepatitis C because of other recently approved treatments.
Merck, in its fourth-quarter earnings report, said it plans to discuss the matter with the FDA, and still expects to seek U.S. approval for the treatment in the first half of 2015. It consists of a protease inhibitor called MK-5172 and a so-called NS5A inhibitor called MK-8742 that together had received the "breakthrough therapy" designation from the FDA.
Shares of the second-biggest U.S. drugmaker slumped 2.3 percent in premarket trading.
The setback for Merck's treatment follows recent approvals of costly oral treatments for the liver disease from Gilead Sciences Inc and AbbVie that have wiped out all signs of the virus in more than 90 percent of patients after eight or 12 weeks.
In the face of such potent rival medicines, Merck in June announced it would ramp up its involvement in the lucrative hepatitis C market by buying Idenix Pharmaceuticals Inc for $3.85 billion and combining the two companies' most promising drugs to produce a faster, more effective cure for hepatitis C. It agreed to pay $24.50 per share, more than three times Idenix's share price before the deal was made public.
Idenix had three drugs in development to treat hepatitis C, most notably a pill in early-stage trials called IDX21437, which Merck has renamed MK-3682. Like Gilead's Sovaldi, it is a nucleotide inhibitor - or "nuc" - that blocks a protein needed by the hepatitis C virus to replicate.
Merck is conducting a mid-stage trial to study combinations of MK-3682 and MK-5172 (grazoprevir) with either MK-8742 (elbasivir) or another drug called MK-8408. It hopes to begin larger late-stage trials later this year.
"We do not see this affecting the development of the combo," said JPMorgan analyst Chris Schott, referring to the potential loss of the special "breakthrough" designation, which provides a gloss of specialness to drugs and can speed up an FDA review.
Gilead introduced Sovaldi in late 2013 at a cost of $1,000 a pill, and reported fourth-quarter sales of $1.73 billion for the drug. Gilead later introduced Harvoni, a new pill combining Sovaldi with another treatment, and it captured sales of $2.11 billion in the quarter.
After AbbVie launched its Viekira Pak treatment late last year, it and Gilead have fought for market share by offering rebates to group payers.
Gilead estimated that 141,000 Americans have been started on one of its hepatitis C products so far, and it expects 250,000 to be treated this year. As many as 3.2 million people in the United States are estimated to be infected with the hepatitis C virus, which can lead to severe liver disease.
STRONG DOLLAR HURTS
Merck on Wednesday reported slightly disappointing fourth-quarter sales and predicted 2015 earnings below analyst forecasts, citing the negative impact of the stronger dollar, as most of its U.S. rivals have done in making their own cautious forecasts.
Merck said it expects full-year 2015 earnings of $3.32 to $3.47 per share, excluding special items, with foreign exchange factors crimping earnings by 27 cents per share. Wall Street had predicted $3.49 per share.
The company said it earned $7.32 billion, or $2.54 per share, in the quarter. That compared with $781 million, or 26 cents per share, in the year-earlier period.
Excluding special items, the company earned 87 cents per share. Analysts, on average, expected 85 cents per share, according to Thomson Reuters I/B/E/S.
Company revenue fell 7 percent to $10.48 billion, trailing Wall Street expectations of $10.5 billion, held back by lower sales of its Remicade arthritis drug, now facing competition abroad from cheaper generic drugs. Sales would have fallen 4 percent if not for the stronger dollar, which lowers the value of sales abroad, when converted back into U.S. currency. (Reporting by Ransdell Pierson; Editing by W Simon and Paul Simao)
Wednesday, February 4, 2015
Tuesday, February 3, 2015
Catamaran Selects Gilead Sciences as its Exclusive Hepatitis C Treatment Provider
Catamaran (CTRX) Selects Gilead Sciences (GILD) as its Exclusive Hepatitis C Treatment Provider
Catamaran Corp. (NASDAQ: CTRX) announced that it is introducing a new clinical outcomes-based program for hepatitis C treatment through its specialty pharmacy, BriovaRx.
This new program will provide clients of Catamaran and BriovaRx an innovative new approach to hepatitis C treatment. Centered on producing positive health outcomes, the program will allow for overall treatment costs to take into account clinical results. Patients will be monitored during their entire treatment regimen through their participation in BriovaRx's Hepatitis C Patient Management Program in order to support full adherence.
"When looking for a hepatitis C partner, our goal was not only to ensure the best possible value for the treatment cost alone, but also to promote the best possible health outcomes for our patients," said Albert Thigpen, SVP, Industry Relations & Supply Chain Management, Catamaran. "This innovative strategy supports Catamaran's view that a smarter approach to medicine has the power to lower overall healthcare costs and promotes optimal outcomes over the long-term."
Gilead Sciences, Inc.'s (NASDAQ: GILD) hepatitis C treatments, Harvoni and Sovaldi, are the exclusive options on Catamaran's National and Value Formularies. Catamaran's commercial clients will be eligible to participate in this clinical outcomes-based program for hepatitis C if they utilize one of these formularies or have a custom formulary and opt-in to the program.
"Catamaran and BriovaRx are rooted in delivering a more integrated healthcare experience for patients through a holistic and personalized approach," said, Sumit Dutta, M.D., M.B.A., Senior Vice President and Chief Medical Officer, Catamaran. "Our clinical outcomes-based program for hepatitis C supports this mission, empowering our nurses and pharmacists to partner with patients in a way that truly drives better health outcomes and ensures these high-cost therapies accomplish what they were designed to do."
To offer patients personalized care and achieve higher persistency and adherence rates, BriovaRx employs a variety of programs to aid patients. BriovaRx's Hepatitis C Patient Management Program features a nursing team that creates an individualized care plan for each patient based on the complexity of their condition and co-morbidities. Offerings like this have helped increase adherence and persistency for patients, contributing to overall cost savings and improved clinical outcomes.
For more information, please visit CatamaranRx.com, and for industry news and information, follow Catamaran on Twitter, @CatamaranCorp.
This new program will provide clients of Catamaran and BriovaRx an innovative new approach to hepatitis C treatment. Centered on producing positive health outcomes, the program will allow for overall treatment costs to take into account clinical results. Patients will be monitored during their entire treatment regimen through their participation in BriovaRx's Hepatitis C Patient Management Program in order to support full adherence.
"When looking for a hepatitis C partner, our goal was not only to ensure the best possible value for the treatment cost alone, but also to promote the best possible health outcomes for our patients," said Albert Thigpen, SVP, Industry Relations & Supply Chain Management, Catamaran. "This innovative strategy supports Catamaran's view that a smarter approach to medicine has the power to lower overall healthcare costs and promotes optimal outcomes over the long-term."
Gilead Sciences, Inc.'s (NASDAQ: GILD) hepatitis C treatments, Harvoni and Sovaldi, are the exclusive options on Catamaran's National and Value Formularies. Catamaran's commercial clients will be eligible to participate in this clinical outcomes-based program for hepatitis C if they utilize one of these formularies or have a custom formulary and opt-in to the program.
"Catamaran and BriovaRx are rooted in delivering a more integrated healthcare experience for patients through a holistic and personalized approach," said, Sumit Dutta, M.D., M.B.A., Senior Vice President and Chief Medical Officer, Catamaran. "Our clinical outcomes-based program for hepatitis C supports this mission, empowering our nurses and pharmacists to partner with patients in a way that truly drives better health outcomes and ensures these high-cost therapies accomplish what they were designed to do."
To offer patients personalized care and achieve higher persistency and adherence rates, BriovaRx employs a variety of programs to aid patients. BriovaRx's Hepatitis C Patient Management Program features a nursing team that creates an individualized care plan for each patient based on the complexity of their condition and co-morbidities. Offerings like this have helped increase adherence and persistency for patients, contributing to overall cost savings and improved clinical outcomes.
For more information, please visit CatamaranRx.com, and for industry news and information, follow Catamaran on Twitter, @CatamaranCorp.
Engineering New Tissues and Organs
How can you mend a broken heart? Or repair a damaged liver, kidney, or knee? NIH-funded scientists are exploring innovative ways to fix faulty organs and tissues or even grow new ones. This type of research is called tissue engineering. Exciting advances continue to emerge in this fast-moving field.
Tissue engineering could allow doctors to repair or replace worn-out tissues and organs with living, working parts. Most important, tissue engineering might help some of the 120,000 people on the waitlist to receive donated kidneys, livers, or other organs.
Doctors have long used tissue-engineered skin to heal severe burns or other injuries. But most tissue engineering methods are still experimental. They’ve been tested only in laboratory dishes and sometimes in animals, but only a few new approaches have been tested in people. Several clinical studies (involving human volunteers) are in the early stages of testing newly developed tissues.
“With this approach, scientists are combining engineering and biology to restore a damaged organ or tissue, whether it’s been damaged by disease or injury or something else,” says Dr. Martha Lundberg, an NIH expert in heart-related tissue engineering.
Some scientists are creating special net-like structures, or scaffolds, in desired shapes and then coaxing cells to grow within them. Some use a mixture of natural substances called growth factors, which direct cells to grow and develop in certain ways.
“Other scientists are using different 3-D bioprinting technologies—some are like fancy inkjet printers—to create new tissues or organs,” Lundberg says. They’ve printed 3-D kidneys and other organs that look like the real thing. But while most of these printed body parts have the right shape, they’re not fully functional.
“Scientists haven’t yet figured out how to print an organ that includes the correct blood vessel patterns, nerve connections, and other components that come together in a mature organ,” Lundberg says. “When creating a new organ, if it can perform the right job and functions, it may not need to look like the real thing.”
Many tissue engineering methods use stem cells, which can be nudged to turn into different cell types. One research team guided human stem cells to become a 3-D structure that can respond to light. The method might one day lead to new therapies for eye disorders. Other stem cell approaches may lead to improved treatment for spinal cord injuries, diabetes, and more.
Another approach, called decellularization, involves removing all the cells from an organ. What’s left behind is a thin, pale framework that contains the organ’s natural structural proteins, including the pathways for tiny blood vessels and nerves. By infusing new cells into this mesh-like matrix, some researchers have successfully created working animal kidneys, livers, hearts, lungs, and other organs.
The decellularization technique was used by Dr. Martin Yarmush and his colleagues to create a functional rat liver that included a network of working blood vessels. Yarmush is a biomedical engineer at Rutgers University and the Massachusetts General Hospital.
The engineered livers his team created were kept alive in the laboratory for days and functioned for several hours after transplantation into rats. The researchers are now working to help those transplanted livers survive even longer. They’re also scaling up the methods to create a decellularized human liver that can be repopulated with functional cells.
“A parallel effort we are pursuing involves taking a donated organ that is not considered transplantable for a particular reason, and then using a reconditioning solution and perhaps even stem cells to revitalize the organ so it becomes transplantable,” Yarmush says.
Other researchers are working to repair damaged body parts that are still in the body. At the University of Washington in Seattle, Dr. Charles Murry and colleagues are searching for ways to fix injured hearts. One of their latest studies used human stem cells to repair damaged hearts in monkeys. The stem cells were coaxed to become early-stage heart cells, which were then infused near the heart injury.
The new cells made their way into the damaged heart muscle and organized into muscle fibers in all of the treated monkeys. The infused stem cells replaced nearly half of the damaged heart tissue and began beating in sync with the heart. Still, the scientists note they need years of research before this type of therapy might be tried in people.
Some methods are already being tested in humans. Dr. Martha Murray, a surgeon at Boston Children’s Hospital, is exploring new ways to heal a common knee injury known as a torn ACL (anterior cruciate ligament). Athletes who do a lot of twisting and turning, as in basketball or soccer, are at risk for damaging the ACL.
“Typical treatment today, called ACL reconstruction, works well, and it gets patients back to the playing field at a relatively high rate,” Murray says. But the surgery involves removing a piece of tendon from elsewhere in the body and using that to replace the ACL. “So it involves making 2 injuries that the body has to heal from. And even with this treatment, patients still develop arthritis in the knee 15 to 20 years later,” Murray adds. “We wanted to find a better therapy—something less invasive.”
After testing several biomaterials, Murray’s team found that stitching a bioengineered sponge between the torn ends of an injured ACL allows blood to clot and collect around the damaged ligament. Because blood naturally contains stem cells and growth factors, the blood-soaked sponge acts as a “bridge” that encourages ACL healing. The sponge is made of some of the same proteins normally found in ligaments, and it dissolves after a few weeks.
Studies in large animals showed that the bioengineered sponge was much less likely to lead to arthritis, and it healed ACL injuries as well as standard reconstruction surgery. The U.S. Food and Drug Administration recently approved human safety testing of the sponge in 10 people with ACL injuries.
Metal, plastic, and other non-biological devices can also replace or enhance malfunctioning body parts. One promising possibility still in development is an artificial kidney that could be implanted in the body and used in place of dialysis to treat end-stage kidney disease. Scientists are also studying a synthetic glue modeled after a natural adhesive that might help to repair tissues in the body. You can learn more about these and other cutting-edge studies at www.nibib.nih.gov/science-education/bionic-man.
References:
Generation of three-dimensional retinal tissue with functional photoreceptors from human iPSCs. Zhong X, Gutierrez C, Xue T, Hampton C, et al. Nat Commun. 2014 Jun 10;5:4047. doi: 10.1038/ncomms5047. PMID: 24915161.
Regeneration and experimental orthotopic transplantation of a bioengineered kidney. Song JJ, Guyette JP, Gilpin SE, Gonzalez G, Vacanti JP, Ott HC. Nat Med. 2013 May;19(5):646-51. doi: 10.1038/nm.3154. Epub 2013 Apr 14. PMID: 23584091.
Human embryonic-stem-cell-derived cardiomyocytes regenerate non-human primate hearts. Chong JJ, Yang X, Don CW, Minami E, et al. Nature. 2014 Jun 12;510(7504):273-7. doi: 10.1038/nature13233. Epub 2014 Apr 30. PMID: 24776797.
Use of a bioactive scaffold to stimulate anterior cruciate ligament healing also minimizes posttraumatic osteoarthritis after surgery. Murray MM, Fleming BC. Am J Sports Med. 2013 Aug;41(8):1762-70. doi: 10.1177/0363546513483446. Epub 2013 Jul 15. PMID: 23857883.
NIH News in Health, February 2015
Galled by the Gallbladder?
Galled by the Gallbladder?
Your Tiny, Hard-Working Digestive Organ
Most of us give little thought to the gallbladder, a pear-sized organ that sits just under the liver and next to the pancreas. The gallbladder may not seem to do all that much. But if this small organ malfunctions, it can cause serious problems. Gallbladder disorders rank among the most common and costly of all digestive system diseases. By some estimates, up to 20 million Americans may have gallstones, the most common type of gallbladder disorder.
The gallbladder stores bile, a thick liquid that’s produced by the liver to help us digest fat. When we eat, the gallbladder’s thin, muscular lining squeezes bile into the small intestine through the main bile duct. The more fat we eat, the more bile the gallbladder injects into the digestive tract.
Bile has a delicate chemical balance. It’s full of soluble cholesterol produced by the liver. This is a different type of cholesterol than the kind related to cardiovascular disease. If the chemical balance of bile gets slightly off, the cholesterol can crystalize and stick to the wall of the gallbladder. Over time, these crystals can combine and form gallstones.
Gallstones can range from the size of a grain of sand to that of a golf ball. When the gallbladder injects bile into the small intestine, the main bile duct can become blocked by these crystalline stones. That may cause pressure, pain, and nausea, especially after meals. Gallstones can cause sudden pain in the upper right abdomen, called a gallbladder attack (or biliary colic). In most cases, though, people with gallstones don’t realize they have them.
The causes of gallstones are unclear, but you’re more likely to have gallstone problems if you have too much body fat, especially around your waist, or if you’re losing weight very quickly. Women, people over age 40, people with a family history of gallstones, American Indians, and Mexican Americans are also at increased risk for gallstones.
“For the average person with an average case, the simplest way to diagnose a gallstone is by an ultrasound,” says Dr. Dana Andersen, an NIH expert in digestive diseases.
If left untreated, a blocked main bile duct and gallbladder can become infected and lead to a life-threatening situation. Gallbladder removal, called a cholecystectomy, is the most common way to treat gallstones. The gallbladder isn’t an essential organ, which means you can live normally without it.
Gallbladder removal can be done with a laparoscope, a thin, lighted tube that shows what’s inside your abdomen. The surgeon makes small cuts in your abdomen to insert the surgical tools and take out the gallbladder. The surgery is done while you are under general anesthesia, asleep and pain-free. Most people go home on the same day or the next.
Researchers have long investigated medications that can prevent gallstones from forming, but these therapies are currently used only in special situations.
It’s uncommon for the gallbladder to cause problems other than gallstones. Gallbladder cancer is often difficult to treat, as it’s usually diagnosed at an advanced stage. But such cancers are relatively rare.
While the gallbladder may not be the star of the digestive system, it still plays an important role. Treat it well by maintaining a healthy diet and getting regular exercise, and the little bag of bile should do its job. Don’t ignore pain or symptoms, and see your doctor if you’re in discomfort, especially after eating.
Your Tiny, Hard-Working Digestive Organ
Most of us give little thought to the gallbladder, a pear-sized organ that sits just under the liver and next to the pancreas. The gallbladder may not seem to do all that much. But if this small organ malfunctions, it can cause serious problems. Gallbladder disorders rank among the most common and costly of all digestive system diseases. By some estimates, up to 20 million Americans may have gallstones, the most common type of gallbladder disorder.
The gallbladder stores bile, a thick liquid that’s produced by the liver to help us digest fat. When we eat, the gallbladder’s thin, muscular lining squeezes bile into the small intestine through the main bile duct. The more fat we eat, the more bile the gallbladder injects into the digestive tract.
Bile has a delicate chemical balance. It’s full of soluble cholesterol produced by the liver. This is a different type of cholesterol than the kind related to cardiovascular disease. If the chemical balance of bile gets slightly off, the cholesterol can crystalize and stick to the wall of the gallbladder. Over time, these crystals can combine and form gallstones.
Gallstones can range from the size of a grain of sand to that of a golf ball. When the gallbladder injects bile into the small intestine, the main bile duct can become blocked by these crystalline stones. That may cause pressure, pain, and nausea, especially after meals. Gallstones can cause sudden pain in the upper right abdomen, called a gallbladder attack (or biliary colic). In most cases, though, people with gallstones don’t realize they have them.
The causes of gallstones are unclear, but you’re more likely to have gallstone problems if you have too much body fat, especially around your waist, or if you’re losing weight very quickly. Women, people over age 40, people with a family history of gallstones, American Indians, and Mexican Americans are also at increased risk for gallstones.
“For the average person with an average case, the simplest way to diagnose a gallstone is by an ultrasound,” says Dr. Dana Andersen, an NIH expert in digestive diseases.
If left untreated, a blocked main bile duct and gallbladder can become infected and lead to a life-threatening situation. Gallbladder removal, called a cholecystectomy, is the most common way to treat gallstones. The gallbladder isn’t an essential organ, which means you can live normally without it.
Gallbladder removal can be done with a laparoscope, a thin, lighted tube that shows what’s inside your abdomen. The surgeon makes small cuts in your abdomen to insert the surgical tools and take out the gallbladder. The surgery is done while you are under general anesthesia, asleep and pain-free. Most people go home on the same day or the next.
Researchers have long investigated medications that can prevent gallstones from forming, but these therapies are currently used only in special situations.
It’s uncommon for the gallbladder to cause problems other than gallstones. Gallbladder cancer is often difficult to treat, as it’s usually diagnosed at an advanced stage. But such cancers are relatively rare.
While the gallbladder may not be the star of the digestive system, it still plays an important role. Treat it well by maintaining a healthy diet and getting regular exercise, and the little bag of bile should do its job. Don’t ignore pain or symptoms, and see your doctor if you’re in discomfort, especially after eating.
How Hepatitis C Is Shining A Light On Critical Gaps In Payment Reform
How Hepatitis C Is Shining A Light On Critical Gaps In Payment Reform
by Kavita Patel, Morgan Romine, and Gregory Daniel
Since December 2013, regulatory approval of new treatments for hepatitis C have brought long simmering debates on drug pricing and value to full boil. The drugs—Gilead’s Sovaldi and successor combination treatment Harvoni, AbbVie’s Viekira Pak—represent significant steps forward for treatment in hepatitis C, demonstrating cure rates well above 90 percent in the clinical trial setting as well as greater tolerability for patients.
This unprecedented effectiveness, however, has come at a high cost, with treatment ranging from $63,000 for an eight-week course of Harvoni on the low end to above $150,000 for Sovaldi in combination with other products on the high end. This is likely to be representative of a wave of similar products coming through the drug development pipeline: highly targeted, highly effective, and highly priced.
Also indicative of things to come are the steps some groups are taking to blunt the impact of increased spending on hepatitis C treatments, such as formulary adjustments or prioritized coverage for particular subsets of the hepatitis C patient community. Days after the U.S. Food and Drug Administration (FDA) approved AbbVie’s Viekira Pak in December 2014, for example, the largest pharmacy benefit management company in the United States, Express Scripts, announced that the drug regimen would be the only hepatitis C treatment on its preferred list of covered drugs.
Express Scripts negotiated an undisclosed discount on the drug from AbbVie and agreed to expand access to the drug to all patients within their covered population—knocking Gilead’s Sovaldi and Harvoni off of their preferred coverage list in the process. A similar agreement between Gilead and CVS has since been announced. These types of arrangements—and the impacts they have on patient access and health spending—could become more common as high-cost drugs and medical products enter the market.
Still, there are additional means for addressing the challenges presented by high-cost, highly effective treatments. In virtually all other aspects of health care, payment and delivery reforms are achieving a palpable shift away from volume and intensity toward patient-centered, higher value care. The ongoing debate surrounding hepatitis C treatments—and efforts like those of Express Scripts and CVS—shine a light on the relative absence of novel drug payment models in such overarching reforms.
In this blog post, we consider alternative payment policies that could help to ensure that—despite their potentially high cost—breakthrough treatments are consistently delivering value to patients and that the health care system is addressing inefficiencies in the delivery and total costs of care.
Paying for Value and Outcomes: Alternative Provider Payment Models
Over the last several decades, payers and providers have taken significant steps to move coverage and reimbursement from fee-for-service (FFS) payments that incentivize higher volume and intensity to a focus on achieving better results and lower overall costs for patients. Such payment reforms have important implications for breakthrough treatments, as they allow treatment access while also ensuring care coordination and more efficient total spending.
Bundled payments, for example, have been piloted in areas like oncology. Proposed models create a single, episode-based payment for certain components of cancer care, such as chemotherapy administration and office visits. Additionally, a care coordination fee in the form of a monthly paymentcould help oncologists receive reimbursements for services which are undervalued in FFS payments while also helping begin to delink the delivery of care from pharmaceutical pricing.
Expanded bundles or episodic payments could cover a broader range of services, including chemotherapy and supportive-care drugs themselves, providing even stronger incentives to reduce drug costs. Given the existing complex benefit design structures, these models are likely to only work for physician-administered drugs that are covered by medical benefits and are traditionally purchased by providers who are then reimbursed by payers after administration.
The payments are also tied to quality measures such as the use of guideline-based therapies and clinical pathways, to provide more transparency about quality and to enhance incentives to avoid undertreatment. Physicians who provide care with a total cost below the established bundle price, while still performing well on the quality metrics, would be paid more.
While these models do not directly act on the price of a novel treatment, they are designed to decrease overall costs by promoting greater care coordination, improved efficiency in supportive care and related costs, and less use of ineffective or low-value treatments through financial rewards tied to outcomes and overall cost. They are directed at provider behavior and are highly contingent on the care team’s efforts; indeed, many of these models are intended to promote more effective team-based care. While they have the potential to promote more effective use of breakthrough drugs, bundled payments may not adjust rapidly to changes in the cost of treatment.
Paying for Value and Outcomes: Outcomes-based Reimbursement
A further extension of payment reforms that shift from volume and intensity to value is tying payments for the treatment itself to outcomes or other measures of performance. Outcomes-based contracting, in which manufacturers share the risk of a treatment outcome, could help alleviate concerns that high-cost treatments actually lead to patient improvement or cures in practice. At their most basic, outcomes-based reimbursement agreements establish defined payment for defined outcomes: a manufacturer shares in the cost of failure in practice through larger rebates, discounts, or refunds if a product does not achieve the performance or outcomes goals agreed to by the payer.
These goals could include patient medication adherence, evidence-based prescribing, or clinical outcomes. The payer monitors drug performance using medical and pharmacy claims, potentially augmented by other tests or modes of data collection (such as patient-collected data or additional laboratory results). These arrangements are particularly attractive when there is lingering uncertainty about the effectiveness profile or value of a treatment in real-world settings; as the potential impact and cost of a treatment rise, so does the importance of addressing such gaps between premarket studies and actual practice.
Though not without precedent in the last decade, outcomes-based reimbursement mechanisms have been difficult to design and implement. The outcomes or performance goals established may not be easily characterized or measured, or may be influenced by factors completely outside the scope of a drug’s performance. The administrative burdens for data collection, contracting, and establishing an infrastructure capable of exchanging the clinical information necessary for payment have been high, as have the costs of setting up such a system. The incentives for providers to take part have sometimes been misaligned, driving prescribing patterns toward other products not included in the outcomes-based mechanism. Government price calculations (e.g., best price) can further complicate the financials of the arrangement.
Still, the idea is not without merit, especially when a breakthrough, potentially-curative product gains FDA approval. Hepatitis C drugs, for instance, could be prime examples of treatments that would make great use of these types of payment. With a relatively high price tag, short treatment duration (8 or 12 weeks), and a clearly measurable outcome standard in sustained virologic response (SVR), the products would seem to avoid some obstacles to an outcomes-based payment model. Leveraging the growing integration and use of routinely collected electronic health data for real-world evidence development could further support efficient outcomes monitoring.
Further, while these types of contracts have been primarily driven by payers, new care delivery and risk-sharing models such as those seen in Accountable Care Organizations (ACOs) create stronger incentives for providers to work with manufacturers to increase effective use of a treatment, not just volume of treatment. Just as value-based payments may create “win-win” opportunities for payers and providers, they may also encourage win-win improvements in care management and adherence activities undertaken by payers and manufacturers.
Significant scientific and medical advancements over the last decade have led to truly game changing treatments and cures for some of our most life-threatening diseases — often with dramatically higher prices for innovator drugs that few can argue are sustainable in the long term. With many payment and delivery reforms already underway to substantially increase health care access and shift focus to achieving better outcomes at lower total costs, the question is no longer one of whether or not we should reform how biopharmaceutical products are financed and reimbursed, but rather one of how to design and implement these reforms in order to maximize impact for patients, increase value within the system, and safeguard further biomedical innovation.
Given the diversity of diseases and innovative treatments, no single approach will be appropriate for all settings. Individual patient or disease characteristics, the manner in which the treatment is administered, and the size of the population impacted are examples of factors that may influence which particular policy reforms may help. The options highlighted here are therefore not standalone solutions; providing breakthrough treatments will require a constellation of payment and policy efforts and a concerted collaboration on the part of all stakeholders to address the unique economic and innovation challenges within a diverse range of therapeutic areas.
by Kavita Patel, Morgan Romine, and Gregory Daniel
Since December 2013, regulatory approval of new treatments for hepatitis C have brought long simmering debates on drug pricing and value to full boil. The drugs—Gilead’s Sovaldi and successor combination treatment Harvoni, AbbVie’s Viekira Pak—represent significant steps forward for treatment in hepatitis C, demonstrating cure rates well above 90 percent in the clinical trial setting as well as greater tolerability for patients.
This unprecedented effectiveness, however, has come at a high cost, with treatment ranging from $63,000 for an eight-week course of Harvoni on the low end to above $150,000 for Sovaldi in combination with other products on the high end. This is likely to be representative of a wave of similar products coming through the drug development pipeline: highly targeted, highly effective, and highly priced.
Also indicative of things to come are the steps some groups are taking to blunt the impact of increased spending on hepatitis C treatments, such as formulary adjustments or prioritized coverage for particular subsets of the hepatitis C patient community. Days after the U.S. Food and Drug Administration (FDA) approved AbbVie’s Viekira Pak in December 2014, for example, the largest pharmacy benefit management company in the United States, Express Scripts, announced that the drug regimen would be the only hepatitis C treatment on its preferred list of covered drugs.
Express Scripts negotiated an undisclosed discount on the drug from AbbVie and agreed to expand access to the drug to all patients within their covered population—knocking Gilead’s Sovaldi and Harvoni off of their preferred coverage list in the process. A similar agreement between Gilead and CVS has since been announced. These types of arrangements—and the impacts they have on patient access and health spending—could become more common as high-cost drugs and medical products enter the market.
Still, there are additional means for addressing the challenges presented by high-cost, highly effective treatments. In virtually all other aspects of health care, payment and delivery reforms are achieving a palpable shift away from volume and intensity toward patient-centered, higher value care. The ongoing debate surrounding hepatitis C treatments—and efforts like those of Express Scripts and CVS—shine a light on the relative absence of novel drug payment models in such overarching reforms.
In this blog post, we consider alternative payment policies that could help to ensure that—despite their potentially high cost—breakthrough treatments are consistently delivering value to patients and that the health care system is addressing inefficiencies in the delivery and total costs of care.
Paying for Value and Outcomes: Alternative Provider Payment Models
Over the last several decades, payers and providers have taken significant steps to move coverage and reimbursement from fee-for-service (FFS) payments that incentivize higher volume and intensity to a focus on achieving better results and lower overall costs for patients. Such payment reforms have important implications for breakthrough treatments, as they allow treatment access while also ensuring care coordination and more efficient total spending.
Bundled payments, for example, have been piloted in areas like oncology. Proposed models create a single, episode-based payment for certain components of cancer care, such as chemotherapy administration and office visits. Additionally, a care coordination fee in the form of a monthly paymentcould help oncologists receive reimbursements for services which are undervalued in FFS payments while also helping begin to delink the delivery of care from pharmaceutical pricing.
Expanded bundles or episodic payments could cover a broader range of services, including chemotherapy and supportive-care drugs themselves, providing even stronger incentives to reduce drug costs. Given the existing complex benefit design structures, these models are likely to only work for physician-administered drugs that are covered by medical benefits and are traditionally purchased by providers who are then reimbursed by payers after administration.
The payments are also tied to quality measures such as the use of guideline-based therapies and clinical pathways, to provide more transparency about quality and to enhance incentives to avoid undertreatment. Physicians who provide care with a total cost below the established bundle price, while still performing well on the quality metrics, would be paid more.
While these models do not directly act on the price of a novel treatment, they are designed to decrease overall costs by promoting greater care coordination, improved efficiency in supportive care and related costs, and less use of ineffective or low-value treatments through financial rewards tied to outcomes and overall cost. They are directed at provider behavior and are highly contingent on the care team’s efforts; indeed, many of these models are intended to promote more effective team-based care. While they have the potential to promote more effective use of breakthrough drugs, bundled payments may not adjust rapidly to changes in the cost of treatment.
Paying for Value and Outcomes: Outcomes-based Reimbursement
A further extension of payment reforms that shift from volume and intensity to value is tying payments for the treatment itself to outcomes or other measures of performance. Outcomes-based contracting, in which manufacturers share the risk of a treatment outcome, could help alleviate concerns that high-cost treatments actually lead to patient improvement or cures in practice. At their most basic, outcomes-based reimbursement agreements establish defined payment for defined outcomes: a manufacturer shares in the cost of failure in practice through larger rebates, discounts, or refunds if a product does not achieve the performance or outcomes goals agreed to by the payer.
These goals could include patient medication adherence, evidence-based prescribing, or clinical outcomes. The payer monitors drug performance using medical and pharmacy claims, potentially augmented by other tests or modes of data collection (such as patient-collected data or additional laboratory results). These arrangements are particularly attractive when there is lingering uncertainty about the effectiveness profile or value of a treatment in real-world settings; as the potential impact and cost of a treatment rise, so does the importance of addressing such gaps between premarket studies and actual practice.
Though not without precedent in the last decade, outcomes-based reimbursement mechanisms have been difficult to design and implement. The outcomes or performance goals established may not be easily characterized or measured, or may be influenced by factors completely outside the scope of a drug’s performance. The administrative burdens for data collection, contracting, and establishing an infrastructure capable of exchanging the clinical information necessary for payment have been high, as have the costs of setting up such a system. The incentives for providers to take part have sometimes been misaligned, driving prescribing patterns toward other products not included in the outcomes-based mechanism. Government price calculations (e.g., best price) can further complicate the financials of the arrangement.
Still, the idea is not without merit, especially when a breakthrough, potentially-curative product gains FDA approval. Hepatitis C drugs, for instance, could be prime examples of treatments that would make great use of these types of payment. With a relatively high price tag, short treatment duration (8 or 12 weeks), and a clearly measurable outcome standard in sustained virologic response (SVR), the products would seem to avoid some obstacles to an outcomes-based payment model. Leveraging the growing integration and use of routinely collected electronic health data for real-world evidence development could further support efficient outcomes monitoring.
Further, while these types of contracts have been primarily driven by payers, new care delivery and risk-sharing models such as those seen in Accountable Care Organizations (ACOs) create stronger incentives for providers to work with manufacturers to increase effective use of a treatment, not just volume of treatment. Just as value-based payments may create “win-win” opportunities for payers and providers, they may also encourage win-win improvements in care management and adherence activities undertaken by payers and manufacturers.
Significant scientific and medical advancements over the last decade have led to truly game changing treatments and cures for some of our most life-threatening diseases — often with dramatically higher prices for innovator drugs that few can argue are sustainable in the long term. With many payment and delivery reforms already underway to substantially increase health care access and shift focus to achieving better outcomes at lower total costs, the question is no longer one of whether or not we should reform how biopharmaceutical products are financed and reimbursed, but rather one of how to design and implement these reforms in order to maximize impact for patients, increase value within the system, and safeguard further biomedical innovation.
Given the diversity of diseases and innovative treatments, no single approach will be appropriate for all settings. Individual patient or disease characteristics, the manner in which the treatment is administered, and the size of the population impacted are examples of factors that may influence which particular policy reforms may help. The options highlighted here are therefore not standalone solutions; providing breakthrough treatments will require a constellation of payment and policy efforts and a concerted collaboration on the part of all stakeholders to address the unique economic and innovation challenges within a diverse range of therapeutic areas.
State Medicaid Programs Seek Discounts on Pricey Hepatitis C Drugs
State Medicaid Programs Seek Discounts on Pricey Hepatitis C Drugs
By Amy Orciari Herman
Edited by Susan Sadoughi, MD, and André Sofair, MD, MPH
Several state Medicaid programs are offering to make certain hepatitis C treatments the preferred therapies for their beneficiaries in return for large rebates from the drugs' makers, the Wall Street Journal reports.
The high cost of the newer HCV treatments, including Harvoni and Viekira Pak, have come under much scrutiny in recent months.This week, Missouri cut a deal with Viekira Pak's manufacturer to make the treatment the state's preferred option (instead of Sovaldi) "in exchange for undisclosed rebates" — a move that may save the state $4.2 million in the next fiscal year. Up to 25 states could end up making similar arrangements with the company.
The WSJ notes that large private insurers have been striking similar deals with drug makers. One such instance: UnitedHealth Group has agreed to make Harvoni the preferred treatment for its beneficiaries in exchange for, again, "undisclosed" discounts.
Link(s): Wall Street Journal story (Subscription required)
Background: Physician's First Watch coverage of Viekira Pak approval (Free)
Source
Related:
Reducing the cost of new hepatitis C drugs
Daclatasvir, Harvoni (ledipasvir/sofosbuvir)/Sovaldi/Viekira Pak.
An index of articles pointing the reader to the current controversy over the high price of Sovaldi, Harvoni (ledipasvir/sofosbuvir) and AbbVie Viekira Pak.
By Amy Orciari Herman
Edited by Susan Sadoughi, MD, and André Sofair, MD, MPH
Several state Medicaid programs are offering to make certain hepatitis C treatments the preferred therapies for their beneficiaries in return for large rebates from the drugs' makers, the Wall Street Journal reports.
The high cost of the newer HCV treatments, including Harvoni and Viekira Pak, have come under much scrutiny in recent months.This week, Missouri cut a deal with Viekira Pak's manufacturer to make the treatment the state's preferred option (instead of Sovaldi) "in exchange for undisclosed rebates" — a move that may save the state $4.2 million in the next fiscal year. Up to 25 states could end up making similar arrangements with the company.
The WSJ notes that large private insurers have been striking similar deals with drug makers. One such instance: UnitedHealth Group has agreed to make Harvoni the preferred treatment for its beneficiaries in exchange for, again, "undisclosed" discounts.
Link(s): Wall Street Journal story (Subscription required)
Background: Physician's First Watch coverage of Viekira Pak approval (Free)
Source
Related:
Reducing the cost of new hepatitis C drugs
Daclatasvir, Harvoni (ledipasvir/sofosbuvir)/Sovaldi/Viekira Pak.
An index of articles pointing the reader to the current controversy over the high price of Sovaldi, Harvoni (ledipasvir/sofosbuvir) and AbbVie Viekira Pak.
Monday, February 2, 2015
More patent-opposition on Gilead’s hepatitis C drug, sofosbuvir
More patent-opposition on Gilead’s hepatitis C drug, sofosbuvir
Mumbai, Feb 2:
A fresh bout of opposition has been filed against Gilead’s patent application on hepatitis c drug sofosbuvir. This comes close on the heels of a spate of developments involving the drug, last month.
Non-government organisation Sankalp Rehabilitation Trust, represented by the Lawyers Collective has filed a pre-grant opposition on the drug in the Delhi patent office. (A pre-grant opposition allows interested parties to oppose a patent application before the Patent office decides on whether or not to grant the drug a patent.)
Earlier last month, the Patent office had rejected California-based Gilead’s patent application on the drug, on the grounds that it did not exhibit greater efficacy. Gilead had subsequently approached the Delhi High Court on this order. And later in the month, the Delhi High Court set aside the patent office’s order that rejected the application, in effect returning proceedings for a fresh round of hearings on the sofosbuvir patent application, a lawyer familiar with the case said.
Sankalp’s opposition is the fourth opposition against Gilead’s base patent application. Others include oppositions by I-Mak together with the Delhi Network of Positive People, Natco Pharma and BDR Pharmaceuticals International Limited.
Gilead was not immediately reachable for comment on the latest development. In fact, it has another patent application on the drug that is now pending at the Kolkata patent office.
“We have studied the patent application closely and are convinced that it has no merit” Anand Grover, Director, Lawyers Collective, said in a statement. “It fails to satisfy any grounds required to meet the threshold of what is patentable under India’s patent law. Not only is it not new or inventive, it also does not comply with the requirements of Section 3 (d)” he added.
The latest development comes even as Gilead received regulatory approval in India to market its version of the drug. It is expected to be launched around April.
Last year, Gilead also inked alliances with eight India-based drug companies, allowing them to make generic, less expensive versions of the drug for certain markets.
Gilead has been facing the heat on its otherwise breakthrough drug for its pricing. The drug is priced at $ 84,000 for 12 weeks and is expected to come to India at about $ 900 for the same period.
Oral pill
Sofosbuvir is a once-a-day oral pill that can change the treatment landscape for patients. It is reported to have cure rates of upto 95 per cent compared to 65-70 per cent witnessed with weekly-injections of Pegylated Interferon, the standard current treatment, a note said.
Globally, there are around 180 million people living with hepatitis C of whom 12 million live in India, it added.
Further, the pro-health group opposing Gilead’s patent on sofosbuvir pointed out, under the licenses generic sofosbuvir could cost anywhere between $300 and $900 per treatment.
“We know that even $300 is too expensive for the average person in India, let alone the poor. Open, unrestricted competition by Indian generic companies was responsible for drastic price reductions in ARV (anti-retroviral or anti-AIDSdrugs) prices which allowed scaling-up of treatment to millions,” Eldred Tellis, Director, Sankalp Rehabilitation Trust, said in a statement.
“It is unfortunate that generic companies in India, who once played David to MNC’s Goliath have now chosen to go down this road. As civil society, we will continue to use pro-public health provisions to further access to medicines” added Grover, referring to earlier groundbreaking patent-battles in the country.
jyothi.datta@thehindu.co.in
http://www.thehindubusinessline.com/companies/more-patentopposition-on-gileads-hepatitis-c-drug-sofosbuvir/article6847904.ece
(This article was published on February 2, 2015)
Mumbai, Feb 2:
A fresh bout of opposition has been filed against Gilead’s patent application on hepatitis c drug sofosbuvir. This comes close on the heels of a spate of developments involving the drug, last month.
Non-government organisation Sankalp Rehabilitation Trust, represented by the Lawyers Collective has filed a pre-grant opposition on the drug in the Delhi patent office. (A pre-grant opposition allows interested parties to oppose a patent application before the Patent office decides on whether or not to grant the drug a patent.)
Earlier last month, the Patent office had rejected California-based Gilead’s patent application on the drug, on the grounds that it did not exhibit greater efficacy. Gilead had subsequently approached the Delhi High Court on this order. And later in the month, the Delhi High Court set aside the patent office’s order that rejected the application, in effect returning proceedings for a fresh round of hearings on the sofosbuvir patent application, a lawyer familiar with the case said.
Sankalp’s opposition is the fourth opposition against Gilead’s base patent application. Others include oppositions by I-Mak together with the Delhi Network of Positive People, Natco Pharma and BDR Pharmaceuticals International Limited.
Gilead was not immediately reachable for comment on the latest development. In fact, it has another patent application on the drug that is now pending at the Kolkata patent office.
“We have studied the patent application closely and are convinced that it has no merit” Anand Grover, Director, Lawyers Collective, said in a statement. “It fails to satisfy any grounds required to meet the threshold of what is patentable under India’s patent law. Not only is it not new or inventive, it also does not comply with the requirements of Section 3 (d)” he added.
The latest development comes even as Gilead received regulatory approval in India to market its version of the drug. It is expected to be launched around April.
Last year, Gilead also inked alliances with eight India-based drug companies, allowing them to make generic, less expensive versions of the drug for certain markets.
Gilead has been facing the heat on its otherwise breakthrough drug for its pricing. The drug is priced at $ 84,000 for 12 weeks and is expected to come to India at about $ 900 for the same period.
Oral pill
Sofosbuvir is a once-a-day oral pill that can change the treatment landscape for patients. It is reported to have cure rates of upto 95 per cent compared to 65-70 per cent witnessed with weekly-injections of Pegylated Interferon, the standard current treatment, a note said.
Globally, there are around 180 million people living with hepatitis C of whom 12 million live in India, it added.
Further, the pro-health group opposing Gilead’s patent on sofosbuvir pointed out, under the licenses generic sofosbuvir could cost anywhere between $300 and $900 per treatment.
“We know that even $300 is too expensive for the average person in India, let alone the poor. Open, unrestricted competition by Indian generic companies was responsible for drastic price reductions in ARV (anti-retroviral or anti-AIDSdrugs) prices which allowed scaling-up of treatment to millions,” Eldred Tellis, Director, Sankalp Rehabilitation Trust, said in a statement.
“It is unfortunate that generic companies in India, who once played David to MNC’s Goliath have now chosen to go down this road. As civil society, we will continue to use pro-public health provisions to further access to medicines” added Grover, referring to earlier groundbreaking patent-battles in the country.
jyothi.datta@thehindu.co.in
http://www.thehindubusinessline.com/companies/more-patentopposition-on-gileads-hepatitis-c-drug-sofosbuvir/article6847904.ece
(This article was published on February 2, 2015)
Sunday, February 1, 2015
Video Weekend-Dr. Galati On New HCV Treatments: Sovaldi, Harvoni, Olysio, and Viekira Pak
On this lovely Super Bowl Sunday (I really only care about the halftime show with Katy Perry) we have two videos to share, both published by Dr. Galati over the weekend.
The first video is an overview of newly approved treatments; Sovaldi, Harvoni, Olysio, and Viekira Pak. The second video is a look at the importance of maintaining a diet low in sodium for people living with liver disease and cirrhosis.
Stay connected by visiting Dr. Galati's Blog.
Joe Galati, M.D.
For those suffering from hepatitis C, there are new hepatitis C treatments with a very high cure rate. The new drugs include Sovaldi, Harvoni, Olysio, and Viekira Pak. These new HCV drugs have over a 90% cure rate. Treatment with these new hepatitis C therapies will vary based on the genotype, presence or absence of cirrhosis, and transplant status. Patients with HIV co-infection might also be treated.
Low Salt Diet in Cirrhosis and Liver Disease: Advise to Patients and Family
by Dr. Joe Galati on 02/01/2015
In those patients with liver disease and cirrhosis, maintaining a diet low in salt (also knows as sodium) can be difficult at times. With all the eating out that America does, this processed food is overloaded with salt and sodium. Thus, it is nearly impossible to maintain a diet low in sodium. Usually, the target goal is to consume less than 2,000 mg per day.
***Common Food Sodium Content
For those suffering from hepatitis C, there are new hepatitis C treatments with a very high cure rate. The new drugs include Sovaldi, Harvoni, Olysio, and Viekira Pak. These new HCV drugs have over a 90% cure rate. Treatment with these new hepatitis C therapies will vary based on the genotype, presence or absence of cirrhosis, and transplant status. Patients with HIV co-infection might also be treated.
Low Salt Diet in Cirrhosis and Liver Disease: Advise to Patients and Family
by Dr. Joe Galati on 02/01/2015
In those patients with liver disease and cirrhosis, maintaining a diet low in salt (also knows as sodium) can be difficult at times. With all the eating out that America does, this processed food is overloaded with salt and sodium. Thus, it is nearly impossible to maintain a diet low in sodium. Usually, the target goal is to consume less than 2,000 mg per day.
***Common Food Sodium Content
Links
Dr. Galati is an expert in liver disease, evaluating patients will all facets of liver disease, including hepatitis C, hepatitis B, cirrhosis, liver cancer, fatty liver disease, and liver transplantation.
Located in the Texas Medical Center, Dr. Galati and his team is seeing new patients for evaluation. for more information, visit www.texasliver.com.
Tune in every weekend for Your Health First, Dr. Galati's weekly radio program on Clear Channel's 740 KTRH. Program information can be found at www.yourhealthfirst.com. Each week, Dr. Galati and his experts discuss a wide range of health topics.
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