Revenue opportunities for health systems and hospitals include manufacturing gene therapies on their own or in partnership with biotechnology companies.

After biotechnology companies or healthcare organizations receive approval for gene therapies, there are three key capabilities to bring the therapies to market, a new PwC Health Research Institute report says.

Gene therapies modify patient genes or cells to treat or cure disease. Over the next decade, the number of patients who will have received gene therapies is expected to reach 500,000.

Health systems and hospitals are playing a crucial role in the development of gene therapies, including autologous therapies that take cells from patients that are manipulated then reintroduced to the body, the PwC Health Research Institute report says.

"In contrast to traditional manufacturing, clinicians and health systems are often an essential part of gene therapy production. For, autologous therapies they participate in the first step of the production process—collecting the cells—and the last one—administering the altered gene therapy product. They are, in essence, extensions of the biopharmaceutical company."

Treatment facilities are pivotal in ensuring patient safety and maintaining quality, the report says. "Companies may need to expand their views of the 'production facility' to include treatment centers, since improper collection, handling or administration during the process could put patient safety at risk and undermine quality. Already, some organizations like the Foundation for the Accreditation of Cellular Therapy are working to set standards for these organizations to ensure consistency of quality standards."

After a gene therapy has received regulatory approval, the three crucial capabilities for biotechnology companies and their healthcare organization partners are advanced manufacturing, responsive supply chains, and tailored commercialization and reimbursement models.

1. Advanced manufacturing
 

Unlike traditional medications, most gene therapies are personalized treatments designed for an individual patient or manufactured in small batches, the PwC report says.

"Traditional medical products are made for many patients to take, with differences in doses, release mechanisms, or coatings allowing a regimen to be more personalized to the patients' needs. Manufacturers of these products have long relied on post-approval scale-up activities to quickly meet market demand by producing millions, and even billions, of doses of product per year."

For gene therapies, the inability to manufacture at large scale has three implications for biotechnology companies and their healthcare organization partners:

• Competition for manufacturing capacity is expected to make acquisitions and partnerships attractive options.
   
• Training staff will be essential to avoid production bottlenecks. "Due to the novel techniques and technologies used in manufacturing gene therapy products—and the small number of approved gene therapy products—few prospective employees have ready-to-hire experience in gene therapy," the PwC report says.
   
• Manufacturers will have to focus on time-to-patient (TTP), which is the amount of time between when a treatment is prescribed and when it is received by a patient. Unlike traditional treatments, which often have TTP measured in hours, TTP timelines for gene therapies can be lengthy, the report says. "TTP can be weeks after accounting for doctor's visits, insurance approvals, manufacturing, and the treatment's time in transit. Decreasing this time will help increase patient and provider satisfaction, and potentially lead to better outcomes." Solutions include having multiple manufacturing sites across the country or manufacturing at the site of care.

2. Responsive supply chain
 

"Gene therapy companies often must rely on a robust supply chain with advanced capabilities, from collection of the cells from a patient to administration of the treatment. Key among these capabilities is a 'cold chain' ensuring products are stored at the right temperature and handled properly from manufacturer to patient or vein-to-vein. A single temperature failure in the supply chain could render the product useless, even dangerous," the PwC report says.

There are two implications from the need for a responsive supply chain:

• Gene therapy manufacturers should consider personalized engagement of patients similar to apps offered by companies such as Domino's Pizza. "Gene therapy companies could borrow this approach, showing patients where their cells are, how far along they are in the production process, the status of delivery, when they need to prepare for treatment, and more. Companies also could include educational, payment information and other support tools in these applications," the report says.
   
• Contracting with distributors and payers should account for the possibility of returned products. "Companies should consider what the 'return' process would look like in practice and how they might structure contracts with payers to account for this possibility. Companies could, for example, require the payer to cover the manufacturing costs of the product under certain circumstances beyond the biopharmaceutical companies' control," the report says.

3: Tailored commercialization and reimbursement models
 

Gene therapies can have high costs because they are not produced at large scale, so reimbursement models should be crafted to assure payers and patients that the therapies have value.

There are already examples of innovative pricing models, the PwC report says. "AveXis Inc., a subsidiary of Novartis Pharmaceuticals Corp., is offering payers a pay-over-time option for its new gene therapy treatment for spinal muscular atrophy in pediatric patients, a genetic disorder that causes muscles to atrophy. Under the plan, insurers would have up to five years to pay for the one-time therapy."

Opportunity for health systems and hospitals
 

Health systems and hospitals can generate revenue opportunities from gene therapy manufacturing, Karen Young, pharmaceutical and life sciences leader at PwC, told HealthLeaders.

"As gene therapy develops into the commercial space, health systems and hospitals may start to see gene therapy as a greater revenue opportunity. Some hospitals already discover, test, and administer gene therapies, which are key aspects to the overall process. Already, some academic medical centers have started investing in gene therapy manufacturing facilities and regulatory capacities, which could help them to control all aspects of the process. Even providers who aren't interested in making or developing gene therapies may wish to partner with companies that do, which could allow them to share in the costs—and rewards—of this space."

Medication management is the primary focus of a new care protocol for Parkinson's disease patients in the hospital setting.

Caring for hospital patients who have Parkinson's disease poses challenges for hospitals and health systems, but a new protocol drives significant clinical and financial benefits.

Research has shown that hospitalized Parkinson's patients have higher costs of care compared to patients without the disease, including longer lengths of stay and higher medication costs. A New Jersey-based healthcare organization has worked to improve these metrics.

In 2017, Hackensack University Medical Center in Hackensack, New Jersey, formally launched a strict medication adherence protocol for Parkinson's. In June 2018, the protocol was recognized by The Joint Commission as the Disease-Specific Certification in Parkinson's Disease.

Medication adherence is crucial in the care and daily functioning of people with Parkinson's, says Hooman Azmi, MD, director of the Division of Functional and Restorative Neurosurgery at Hackensack University Medical Center.

"Patients with Parkinson's have a significant reliance on their medication. Most patients with Parkinson's develop motor symptoms such as rigidity, stiffness, difficulty moving, and tremors. Rigidity and difficulty moving are particularly debilitating. The patients require their medicine for these symptoms to go away," he says.

In the outpatient setting, patients with Parkinson's work closely with neurologists to develop finely crafted medication regimens, Azmi says.

"When someone has Parkinson's for a long time, the management of symptoms becomes challenging because the effect of medicine is not long-lasting, and it becomes shorter and shorter. They end up requiring more and more doses of medicine. Sometimes, patients take medicine every three hours—or every two hours—around the clock. If patients don't take their medicine or the medicine is delayed, their symptoms can come out. Basically, they can go from being mobile to almost not being able to move," he says.

The strict medication adherence protocol for Parkinson's developed at Hackensack University Medical Center has generated statistically significant reductions in length of stay and hospital readmissions:

• Length of stay for all Parkinson's patients at the hospital decreased from 7.125 days in 2017 to 6.750 days in 2018.
   
• The readmissions rate for Parkinson's patients decreased from 13.9% in 2017 to 12.8% in 2018.

Hackensack University Medical Center's four primary components of the  medication adherence protocol are as follows:

1. Patient identification
 

Most patients with Parkinson's do not go to a hospital for treatment of the disease, so identifying them and their underlying condition can be a challenge, Azmi says.

"They come into the hospital for everything that everybody else comes into the hospital for—they come in for back pain, kidney stones, heart attacks, and all kinds of conditions. The Parkinson's can be missed in the shuffle because caregivers will focus on the main reason why the patient came to the hospital. So, strict adherence to the Parkinson's medication can be completely misplaced and patients don't get their medication on time, which compounds their problems in the hospital," he says.

HMHUMC is using the hospital's electronic medical record to identify Parkinson's patients, Azmi says. "Whenever a doctor or nurse opens a chart of a patient who has Parkinson's, a flag comes up identifying the patient with the disease. Then care plans are included in the electronic record including the timing of medication and contraindicated medicine."

Delays in administering Parkinson's medications as short as 15 minutes can "wreak havoc," he says.

2. Metrics to assess medication management
 

To help ensure strict adherence to medication regimens, the hospital monitors several metrics for Parkinson's patients, Azmi says. "The metrics include patient identification, making sure all of the medicines are in the formulary, and making sure that patients get medication in the customized fashion that they get at home—we don't put in default medication regimens. We also have a metric to make sure patients are not getting contraindicated medication."

Close monitoring of the metrics impacts patient care, he says.

"Every time an order is placed for a Parkinson's medication, we make sure the order is placed in a customized fashion. We track the number of customized orders versus non-customized orders, and we try to correct inappropriate orders in real time. With contraindicated medications, we follow up when these medications are ordered. We work very hard to prohibit the ordering of these medications for Parkinson's patients."

3. Metrics evaluation and implementation of action plans
 

When care teams find that metrics performance needs to be improved for patients, the hospital's Disease-Specific Committee for Parkinson's Disease develops improvements through implementation of action plans, Azmi says.

"Sometimes, there is an issue in a part of the hospital, so we educate the staff in that area. There may be an instance when the pharmacy needs to develop options when certain medications are ordered. Through the disease-specific committee, we have been able to look at the data, assess it, then come up with plans to improve the compliance," he says.

4. Education
 

Staff throughout the hospital has received education and training for the strict medication adherence protocol, including physicians, advanced practice practitioners, nurses, physical therapists, radiation technologists, and pharmacists.

"Everyone needs to be aware about the importance of medication and avoiding contraindicated medication," Azmi says.

The education and training have been provided in several formats such as webinars, surveys, and a variety of grand rounds for nurses and physicians. "It's an ongoing educational process—it must be continuous because managing these patients poses problems on a continuous basis," he says.

The key to success of the strict medication adherence protocol has been the hospital-wide approach, Azmi says.

"It's a cliché to say it takes a village to raise a child, but it takes an entire hospital to care for one patient. Everything we do for this patient population translates to the entire hospital. This protocol makes us better, and it takes many people from different parts of the hospital—from physicians, to nursing, to physical therapy, to the pharmacy, to regulatory staff, to quality officers. It is truly an interdisciplinary effort to launch an initiative like this throughout the hospital. But it translates into better patient care and financial savings for the hospital."

Azmi is the co-author of a 2018 book about Parkinson's care in the hospital setting, Parkinson's Disease for the Hospitalist: Managing the Complex Care of a Vulnerable Population.

Researchers focus on six categories of waste: failure of care delivery, failure of care coordination, overtreatment or low-value care, pricing failure, fraud and abuse, and administrative complexity.

Waste accounts for about 25% of U.S. healthcare spending, new research indicates.

No other country spends more on healthcare than the United States, with the gross domestic product share of healthcare spending estimated at nearly 18% and rising. Earlier research on U.S. healthcare spending has estimated that waste accounts for about 30% of the spending total.

Reducing wasteful spending is a promising avenue to curb annual increases in the country's healthcare spending, according to the co-authors of the new research, which was published today in the Journal of the American Medical Association. "Implementation of effective measures to eliminate waste represents an opportunity reduce the continued increases in U.S. healthcare expenditures," the researchers wrote.

The researchers examined data from 54 published reports. They tallied waste in six categories identified in 2010 by the Institute of medicine (IOM): failure of care delivery, failure of care coordination, overtreatment or low-value care, pricing failure, fraud and abuse, and administrative complexity. Pricing failure includes medication pricing, payer-based health services pricing, and laboratory-based and ambulatory pricing.

In 2019, total U.S. healthcare spending is projected at $3.82 trillion.

The JAMA researchers generated several key data points:

• Annual wasteful spending on healthcare is estimated from $760 billion to $935 billion.
   
• Interventions to reduce waste in the six IOM categories would result in annual savings from $191 billion to $282 billion.
   
• The annual cost of wasteful spending from administrative complexity accounts for the highest category of waste, estimated at $265.6 billion.
   
• The annual cost of waste from pricing failure is estimated from $230.7 billion to $240.5 billion.
   
• The annual cost of waste from failure of care delivery is estimated from $102.4 billion to $165.7 billion.
   
• The annual cost of waste from overtreatment or low-value care is estimated from $75.7 billion to $101.2 billion.
   
• The annual cost of waste from fraud and abuse is estimated from $58.5 billion to $83.9 billion.
   
• The annual cost of waste from failure of care coordination is estimated from $27.2 billion to $78.2 billion.

The impact of likely interventions to reduce wasteful spending are significant but limited, the researchers wrote.

"The best available evidence about the cost savings of interventions targeting waste, when scaled nationally, account for only approximately 25% of total wasteful spending. These findings highlight the challenges inherent in rapidly changing the course of a health system that accounts for more than $3.8 trillion in annual spending, 17.8% of the nation's GDP."

Assessing the data
 

The highest amount of wasteful spending was associated with the administrative complexity category. The development and adoption of value-based payment models has the most potential to impact this category of wasteful spending, the researchers wrote.

"In value-based models, in particular those in which clinicians take on financial risk for the total cost of care of the populations they serve, many of the administrative tools used by payers to reduce waste (such as prior authorization) can be discontinued or delegated to the clinicians, reducing complexity for clinicians and aligning incentives for them to reduce waste and improve value in their clinical decision-making."

Reducing spending the second-highest wasteful category—pricing failure—poses daunting challenges because of the rising prices of pharmaceuticals, the researchers wrote. "New high-cost specialty drugs, which will soon exceed 50% of pharmaceutical spending, are raising new questions about how to maintain affordability. This topic has thus received considerable attention from policy makers, and numerous proposals are currently under consideration."

The researchers say strategies to ease cost pressure in pharmaceuticals include increasing market competition, importing drugs from countries with lower medication prices, and reforming price transparency.

The big picture view
 

An editorial accompanying the new research says the findings are a significant contribution to the ongoing effort to rein in the country's healthcare spending.

"At a time when the United States is once again mired in a great debate about the future of its healthcare system, the data reported in the article … should become part of the national discussion. It would be nearly impossible for all waste to be eliminated in any healthcare system, just as it is impossible to know the true cost of any change in the delivery and financing of healthcare without understanding possible savings, and recognizing that there is complexity in knowing the savings," the editorial says.

Concentrating on wasteful spending is crucial, the editorial says. "While no single solution will solve the continuous increases in U.S. healthcare spending, identifying, reducing, and eliminating waste are important and appropriate places to start."

At MD Anderson Cancer Center, patient and family advisors are involved in a wide range of activities, including safety, process improvement, research grants, expansion of the organization's call center, and policy development.

The University of Texas MD Anderson Cancer Center is committed to involving patients and family members in a range of decision-making at the Houston-based organization.

After decades of providing directive care to patients, most health systems, hospitals, and physician practices across the country are trying to provide patient-centered care. Research has shown that patient-centered care is associated with several positive outcomes for patients, including better recovery from discomfort and concern, better emotional health, and fewer diagnostic tests and referrals.

Randal Weber, MD, chief patient experience officer at MD Anderson, says the organization's Patient and Family Advisor Program represents a leap forward in the organization's efforts to provide patient-centered care.

"If you are really committed to patient-centered care, you need input from your patients to change culture. Many institutions are provider-centered. The patient advisors are the best people to inform us about where we have opportunities to change our culture and make it more patient-centered and improve patient experience," he says.

MD Anderson launched its Patient and Family Advisor Program in 2014 with 20 founding members. Patients and family members who participate in the program are considered unpaid employees, and they receive extensive training in areas including patient confidentiality such as Health Insurance Portability and Accountability Act (HIPAA) compliance.

"They are fully vetted with HIPAA and everything else a regular employee receives training for, but they are not paid. They can sit on a range of committees such as process improvement and safety committees, and they can be privy to confidential information," says Elizabeth Garcia, RN, MPA, associate vice president of patient experience at MD Anderson.

The Patient and Family Advisor Program was almost immediately successful, and demand for advisor participation in institutional committees and research efforts soon outstripped the supply of advisors, says Kathleen Denton, PhD, MEd, director of patient experience at MD Anderson. "After a year and a half, we expanded the program, and we now have 80 advisors. We still have monthly meetings, but we also place patient and family advisors on institutional committees. They participate in focus groups. We send them electronic surveys."

Scope of involvement
 

The patient and family advisors have been involved in dozens of initiatives, policy decisions, and research projects, including:

• The advisors played an active role in developing MD Anderson's "Stop the Line" safety policy. Stop the Line gives staff, patients, and family members the ability to call for a pause whenever patient safety is in doubt. "We have had patients and family members say, 'stop the line,' when they were about to go into a diagnostic imaging test because they didn't think it was what the doctor ordered. Patients and family members are part of our healthcare team and can stop any procedure or process for safety concerns," Garcia says.
   
• Advisors sit on process improvement and quality improvement committees. For example, the advisors flagged the need to improve the functionality of call buttons in patient rooms. "We put a quality check in place for all our call bells throughout the institution. Now, every time we admit a new patient, the call bell gets checked," Garcia says.
   
• The advisors played an essential role in convincing MD Anderson's leadership to expand the organization's call center, Garcia says. "We extended our call center hours to cover weekends and holidays as well as evenings. We are also adding clinical services to the call center. We could have never gotten funding for our 24/7 call center without our patient advisors saying it was a need."
   
• The advisors requested better follow-up with patients after they are discharged from inpatient care. "In August, we launched a team that calls every patient when they go home. They ask whether the patient got home OK, how they are feeling, and whether they have their meds and their equipment. It's a mental thing—it shows that the institution cares enough to call the patient at home," says Ronnie Pace, a breast cancer survivor who is a founding member and former co-chair of the Patient and Family Advisor Program.
   
• Patient and family advisors were instrumental in improving parking at MD Anderson's main campus by urging adoption of a computerized red, green, and blue light parking system for guiding patients to parking spots similar to parking systems used at airports. "Because the suggestion came from the patients, that really gave the parking director the voice with the administration to get that in place. We also have a new kiosk that can tell patients exactly where their car is parked and how to get there," Denton says.
   
• The advisors have also helped MD Anderson researchers write grant applications.

Recruiting patient and family advisors
 

Patients and family members are eager to serve in the advisor program, says Pace. "The word got out that this council was the real deal, and that the institution wanted to know how we felt and how we thought about certain situations," he says.

There is a rigorous selection process for patients and family members who want to serve in the advisor program.

New members are recruited annually in July. There is an extensive recruitment outreach effort, which includes applications through My Chart and paper applications that are distributed at learning centers, the patient experience office, and the patient and family relaxation area. MD Anderson's internal communications team also advertises the advisor openings to patients and staff members.

There are three primary selection criteria to serve in the Patient and Family Advisor Program: disease-site representation, diverse representation from all populations, and experience with MD Anderson services. "If we have two candidates who are both breast cancer patients, but one has used chaplaincy, supportive care, and a variety of services, we will lean toward the more experienced candidate. We have a steering committee that makes the selections," Denton says.

The advisor selection process includes a 30-minute, on-site interview.

"You need be very careful to choose people who are in it for the right reasons. We have applicants who are going through the grieving process, and sometimes they have not worked through those issues. We want our advisors to understand that they are giving back. This is not a support group. This is about giving back to the organization and making things better for future patients and family members," Garcia says.

New advisors need to be prepared to work on meaningful projects, Pace says. "There is plenty of work to do for patient advisors who want to come in and do the job. There is no lack of opportunity. Our meetings are held once per month, and they're full of activity from start to finish."  

MD Anderson's Patient and Family Advisor Program was the focus of a featured presentation at this summer's HealthLeaders Innovation Exchange in California. The Innovation Exchange is one of six healthcare thought-leadership and networking events that HealthLeaders holds annually. While the events are invitation-only, qualified healthcare executives will be considered. To inquire about the HealthLeaders Exchange program, email us at exchange@healthleadersmedia.com.

Photo credit: Pictured above: Janice Finder, RN, MSN, MD Anderson Cancer Center's director of patient experience for clinical support, makes a point at this summer's HealthLeaders Innovation Exchange in California. (Photo: David Hartig)