Watch list 2016: Top therapeutic areas

This year, many new medications and treatments emerged to improve the management and prognoses of patients suffering from serious illnesses. But while these developments represent promise, they also raise complex decisions related to cost and quality. In 2016, as these developments play a larger role in health management and treatment, managed healthcare executives must be informed. Here's a quick look at some of the latest developments in top therapeutic areas that should be on your radar.

1. Oncology

As new, more tailored and effective oral treatments emerge, the oncology drug category will be one to watch. “These [new treatments] will replace traditional IV therapies that took a more ‘shotgun approach,’ by treating general cancer types,” says Steve Liles, PharmD, senior director, pharmacy services at Goold Health Systems, an Emdeon Company.

“This year, at least 10 to 15 new medications targeting specific cancers have been approved, and I expect that trend to continue,” he says. “This growth is led largely by the immuno-oncologics-effective, well-tolerated agents for which there is and will continue to be increasing use, often in combination regimens, for solid tumors.” 

Goldberg

Robert Goldberg, PhD, vice president and cofounder, Center for Medicine in the Public Interest, agrees. Managed care executives should look to the new immunotherapy treatments such as Opdivo and Keytruda, both for advanced melanoma, Goldberg says. “There are dozens of such therapies that all target specific immune factors that can be taught to stop tumors from spreading. They most often will be used in combination with other treatments customized to individual patients.” 

Heather Morel, vice president and general manager of Health Informatics, Reimbursement, Access and Safety Services for McKesson Specialty Health, says this therapeutic category of drugs, which is now being applied to treat metastatic melanoma and non-small cell lung cancer, is very promising.

Morel

“In some patients we are seeing durable benefits and long-term survival in diseases where the survival rates have been abysmal,” Morel says. “The hope and promise of these drugs is that we will learn the best ways to use them, whether that is in combination with other checkpoint inhibitors, with targeted therapy, chemotherapy, or as stand-alone therapy. There are a number of significant drug development programs targeting many of the major tumor types, and over the next few years we should see new agents enter the market and literature grow.”Liles says new oncology therapies will impact managed care organizations in several ways, including:

Helping them find the right treatment-faster. “The more targeted therapy will allow facilities to pinpoint the exact type of medication that will work for patients, cutting down on the waste associated with broader medication therapies,” he says. “Treatments can be identified more quickly, saving time and money for everyone involved. Moreover, it’s a lot easier for the physician to justify putting a patient on this type of therapy as it is better tolerated than traditional cancer treatment.”

Turning cancer treatment into chronic disease management. “Patients are receiving these new types of therapies over longer courses of time, in part because they are much more effective,” Liles explains. This will be a paradigm shift for managed care organizations. As patients respond better, oncology will shift from short-term and acute treatment to longer-term chronic disease management, he says. “In many ways, this shift will mirror the evolution of HIV treatments. In the early years, patients were given handfuls of drugs that blanket-treated the disease, had severe side effects and didn’t effectively work. Now, patients with HIV receive more specific drugs over a longer period of time; many are living without side effects and have good prognoses.”

Raising difficult cost discussions. These new oncology drugs are expensive, so everyone-including MCOs, employers, providers and patients-will have to weigh the cost of these therapies against the long-term benefits, says Liles.

“Having more choices and better access to data around therapies is great, but this means MCOs will have some tough decisions when selecting the medications that work best for their formularies,” he says. “Drug costs will always be increasing. So, MCOs will have to continue assessing medication spend against the overall percentage of medical costs and the opportunity cost of preventing possible downstream cost increases. At the end of the day, many will have to make an economic analysis of their options, which could be difficult to do.”

 The cost of new medicines, used in combination, could be as much as $100,000 or more for a course of treatment “but the value will be immense,” says Goldberg. “We are seeing significant gains in survival and a reduction in hospitalizations. To really support formulary managers, those benefits and savings should be factored into the determination of what these medicines are worth to the healthcare system, beyond just the cost of the drugs.”  

Next: Hepatitis C

Gleevec’s introduction in 2001 represented a leap in targeted cancer therapies, especially as these medicines use genetic markers in the course of diagnosis and treatment.   

Ambres

Tyrosine kinase blockers represent some of the first targeted therapies in oncology and have shown remarkable results in treating chronic myeloid leukemia (CML), to eliminate the blood cells containing the abnormal BCR-ABL gene, says Cynthia Ambres, MD, a strategy partner and member of the KPMG Global Healthcare Center of Excellence.

“Before these treatments, patients had the options of bone marrow transplantation or daily interferon infusions,” she explains. “Both had serious side effects and the five-year survival rate was only 30% before this category emerged ..." Now, she says, tyrosine kinase blockers have improved survival to  greater than 90% after five years.

2. Hepatitis C

Hepatitis C will be a growing therapy, with new drugs such as ombitasvir/paritaprevir/ritonavir (Viekira Pak), sofosbuvir (Sovaldi), and ledipasvir/sofosbuvir (Harvoni).

Wong

“These high-impact pipeline drugs are on our radar as they target large populations and we are forecasting that they will likely carry high price tags,” says Farrah Wong, PharmD, manager, pipeline and drug safety, OptumRx. “While the armamentarium for hepatitis C immensely grew the past few years, oral combination regimens only treat certain genotypes."

Next: New oral drugs

Sofosbuvir/velpatasvir and odalasvir are being studied in all hepatitis C genotypes and preliminary data look promising, says Wong.

These new oral drugs for hepatitis C promise higher cure rates due to improved compliance in taking the drug for less time and with fewer side effects, says Ambres.

Managed care organizations should closely consider member safety and drug efficacy when evaluating this drug category, says Wong, noting that many of these drugs will be approved based on short-term clinical trial data, so long-term efficacy and safety effects will be unknown. “Because many of these high-impact drugs target large populations, one of the main challenges we face in managing these drugs is ensuring that the appropriate population(s) have access to them and we are able to develop clinical programs to ensure appropriate use and support.”

O'ConnorMarc O’Connor, chief operating officer at Curant Health, says that while these drugs come with a high cost, MCOs should carefully consider the long-term effects. “After all of the hubbub about pricing for Sovaldi and Harvoni, they haven't crippled the system and if prescribed and adhered to properly, they can be cost effective in terms of value,” he says. “The $94,500 price for Harvoni, which brings cure rates for hepatitis C patients versus a $577,000 liver transplant is easy to understand."

As Wong monitors the high costs of new drugs entering the market and builds them into OptumRx’s utilization management plans, she says that she’s pleased to see competition, particularly in the increasingly high-cost category of next-generation hepatitis C drugs.“While they will still likely carry high-price tags, having multiple options for hepatitis C will help drive affordability,” she says. “As additional data are released for these high-impact drugs, we will be able to better evaluate their place in therapy. In addition, upcoming new drug approvals in various categories give us an opportunity to re-evaluate our formulary decisions to help drive affordability for our members and clients.”   

Engagement of companies with medication management protocols proven to improve adherence and outcomes for chronically ill patients should be a high priority across all therapeutic areas for managed healthcare executives in 2016, O’Connor adds.

Next: Diabetes

3. Diabetes

New diabetes medications are represent another drug category that’s increasingly costly that plan managers should be on the lookout for, according to National Center for Policy Analysis Senior Fellow Devon Herrick, PhD. These include empagliflozin (Jardiance), canagliflozin (Invokana), dapagliflozin (Farxiga), and albiglutide (Tanzeum).

Invokana, Farxiga and Jardiance are from a new class of medications known as sodium-glucose co-transporter-2 (SGLT2) inhibitors. “These drugs treat type 2 diabetes by inhibiting renal glucose reabsorption in the kidneys,” Herrick says. “They are an advance over older therapies. But they are also expensive. A regimen taking these drugs would cost around $5,000 per year, not including lab tests and physician care. Diabetes is a condition that can be costly to treat; and costly not to treat.”

The problem is the cost of therapy comes early, while the benefits of treatment often accrues long after a patient has left private health plans and is on Medicare, he says.

4. High cholesterol

PCSK9 inhibitors are a new class of drugs to control cholesterol in statin-intolerant patients. These include Praluent (alirocumab) and Repatha (evolocumab).

“These are highly effective at controlling low-density lipoprotein cholesterol [LDL], but at a significant cost,” Herrick says. The estimated cost of therapy with alirocumab is $15,000 per year, while evolocumab costs about as much.  Most drug plans will get a discount off that price, but it will be high nonetheless.”

O’Connor agrees. “Given the current price tags … for an indefinite period of time, provision of access, adherence to, and results, in order to measure their value, will be of keen interest for managed care executives,” he says.

Patients currently treated with these costly drugs have excessively high cholesterol, and do not respond to statins, says Herrick. "Some experts worry that over time the threshold for treatment will be lowered to include those who could have been treated using the cheaper drug therapy,” he says.

Next: HIV

5. HIV

HIV is another area to watch, as promising therapies and treatments continue to evolve. Death rates due to HIV have plummeted with the introduction of anti-retroviral therapies, says Ambres. In fact, it is estimated that the number of people who died from AIDS topped 40,000 annually between 1993 and 1996, according to the Centers for Disease Control and Prevention.

 “The industry has been churning out combination therapies and new medicines to stay ahead of the disease, keeping opportunistic infections at bay in hundreds of thousands of patients living with HIV,” Ambres says.

The 1917 Clinic, a Ryan White grantee at the University of Alabama at Birmingham (UAB), serves a large patient population that struggles with adherence. Earlier this year, UAB clinicians completed analysis of 652 HIV patients over a 38-month period who engaged in medication management protocols provided by Curant Health as part of their treatment plans. Of 157 patients who did not have suppressed viral loads at enrollment, 103 achieved viral suppression during follow-up tests, which ranged from six weeks to one year after enrollment. That’s a statistically significant increase in the proportion of patients achieving viral suppression, says O'Connor.

A 2012 study of patients on highly active antiretroviral therapy (HAART), by Patrick Dunham (CEO of Curant Health) and Jeffrey Karkula (RPh) also showed that improvements in adherence to HAART via effective medication management protocols are capable of generating overall healthcare cost savings for HIV patients of $3,000 per patient per year.

Next: Rheumatoid arthrits

6. Rheumatoid arthritis

Before tumor necrosis factor (TNF)-Inhibitors arrived on the market, patients with rheumatoid arthritis depended upon a regimen of gold salts and methotrexate to reduce inflammation and corticosteroids for acute inflammation, says Ambres.

Now, TNF inhibitors are improving patient outcomes when they are added to methotrexate, according to a study, “Tumor necrosis factor alpha inhibitors, methotrexate or both? An inquiry into the formal evidence for when they are to be used in rheumatoid arthritis,” published in Clinical and Experimental Rheumatology. 

The combination disrupts the inflammatory process and slows disease progression for millions of patients, according to the study. Additionally, Rheumatology published a compilation of studies that didn’t see improvement of monotherapy versus add-on methotrexate, but found that the combination prolongs TNF inhibitor drug survival. 

“These drugs represent another treatment option for an array of autoimmune/inflammatory diseases, including plaque psoriasis, Crohn’s disease and ankylosing spondylitis, when the vertebrae of the spine fuse together,” Ambres says.