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While definitions for “specialty drug” vary-and distribution and pricing/reimbursement factors remain complicated-this once-niche treatment market may be poised to steer the future of prescription medicine and patient care.
While definitions and classifications of what constitutes a specialty drug vary-and pricing and reimbursement factors remain thorny-there is little argument that this once-niche treatment market may be poised to steer the future of prescription medicine and patient care
Ask three people how they define specialty pharmaceuticals, and you will likely get three similar, but slightly different answers. “A [standard] definition of specialty drug has yet to be adopted,” says Sheila M. Arquette, executive director of the National Association of Specialty Pharmacy (NASP). “The FDA, employer groups, health plans, and pharmacy benefit managers (PBMs) all have their own ways of defining this drug category.”
The NASP views specialty drugs as more complex than most prescription medications and used to treat patients with serious and often life-threatening conditions. Those include cancer, hepatitis C virus, rheumatoid arthritis, HIV/AIDS, multiple sclerosis, cystic fibrosis, organ transplantation, human growth hormone deficiencies, hemophilia, and other bleeding disorders. These medicines, in some cases can be taken orally, but often must be injected or infused and may have special administration, storage, and delivery requirements. Many of the injectable medications are self-administered in the patient’s home. Infused specialty drugs are administered in various treatment settings, such as a patient’s home with the support of a home healthcare professional, a doctor’s office, or hospital, says Arquette.
At Cardinal Health, a distributor of specialty pharmaceuticals, it classifies this type of therapy in four ways. “Specialty products typically have one or more of the following characteristics: They are prescribed to small patient populations with rare, complex, or chronic conditions; they may be part of a complex treatment regimen or require ongoing patient monitoring; they may have special shipping and handling requirements, such as temperature controls; and they are usually more expensive than traditional pharmaceuticals,” explains Joe DePinto, president, Cardinal Health Specialty Solutions.
If anyone in your C-suite attended a specialty pharma conference 10 years ago, they might have been one of a handful of people in the room. But, as the annual Asembia Specialty Pharmacy Summit in Las Vegas earlier this year showed, this segment has grown exponentially.
Although organizers declined to comment for this article, the growing attendance was the headline of a company press release following the conference, boasting that the 2018 Asembia event set a new attendance record with over 6,500 attendees, and marked the thirteenth consecutive year of growth. It was also one of the most mentioned observations when Pharm Exec spoke with attendees, and was brought up during the general session, where one panelist recalled there being just several hundred people on-site in the summit’s first year, with specialty pharma a then-niche market.
What factors are behind the surge in this market? Quite simply, it’s innovation and money, experts point out. “This has primarily been driven by advancement of the science as well as a shift of capital investment to address patient and market needs,” says David Rosner, principal and digital life sciences leader, Deloitte Consulting. “In addition to the increase in the number of specialty-focused pharmaceutical firms, nearly all of the major big pharma manufacturers have added specialty products to their portfolios and consider them key to their growth.”
Randy Maloziec, vice president, biopharma relations, at US Bioservices, a part of AmerisourceBergen, echoed those observations.
“Nearly every pharmaceutical manufacturer has specialty therapies in their pipeline and it is universally accepted that the specialty marketplace will continue to see significant growth in revenue and overall pharmaceutical utilization,” he says. “This growth-real and anticipated-is driving nearly every stakeholder in the market to develop solutions or capabilities to support these therapies. This could be everything from an integrated delivery network (IDN) opening its own specialty pharmacy to a payer seeking new ways to manage overall spend.”
With so many companies entering the specialty pharma space, it begs the question, is what was highly specialized and rare now just becoming the norm?
Growth is almost always followed by challenges. “One of the biggest changes is that specialty products continue to become more targeted and patient populations smaller, particularly as more gene-based and cellular therapies come to market,” says DePinto. “These products face unique challenges in everything from recruiting patients into clinical trials, to determining how to efficiently distribute the product, to managing payer and reimbursement issues. Not only have the products themselves become more specialized, so has the approach to distributing and commercializing them.”
Another key change, according to DePinto, is the increased focus on security and traceability of products. Through advanced technology such as radio-frequency identification (RFID), he says it’s now possible to ensure supply chain integrity and to track products at every point from the manufacturing plant to the site of care.
That logistical part of the discussion is critical, especially when it comes to advanced therapies such as gene and cellular therapy.
“Managing the logistics of CAR-T therapies, [for example], is highly complex because of their high value, temperature sensitivity, and the precise timing in which they must be administered to the patient,” says DePinto. “It’s important to have a scalable distribution network that can compliantly and efficiently transport these products to sites of care nationwide, as well as technology systems to monitor the exact temperature of each dose of medicine the entire time it is in transit. The network also needs clear, standard operating procedures (SOPs) to detail how it would handle any potential logistics risk to the therapy.
“Because of the precise timing requirements, it is critical for the logistics provider to communicate with the pharmaceutical manufacturer, the site of care, and other stakeholders, such as the patient hub, to ensure that all parties are aligned in their efforts to get the therapy to the patient at the right time. Even a minor delay could impact the efficacy of the product and reduce the patient’s chance of experiencing the best outcome.”
Another major challenge facing specialty pharma is the shift from fee-for-service reimbursement to a value-based care model where physicians are reimbursed based on the quality of care and overall patient satisfaction.
“Under these new payment models, physicians are rewarded for reducing the costs of care and demonstrating improved patient outcomes,” says DePinto. “For specialty diseases, the approach of managing a patient across an ‘episode of care’ can be very complex because specialty patients are often treated with multiple medications and receive different interventions at multiple sites of care, making it challenging to track results. For manufacturers of specialty medications, it is not enough for therapies to be safe and efficacious, they must also show they can deliver improved outcomes at a better value.”
Adopting proactive digital health practices in a timely manner; connecting with patients while still staying HIPAA compliant; the volatile political climate that impacts regulations; and perfecting the possibly unachievable balancing act of keeping therapies affordable while also making a profit, were also all reoccurring themes at the 2018 Asembia Specialty Pharmacy Summit.
To hear more about these and other challenges, watch Pharm Exec’s video coverage from the event.
US Bioservices is not a newcomer when it comes to developing solutions to the challenges of specialty pharma. For example, the company exclusively dispenses a medication for a rare, inherited muscular disorder that typically occurs in boys and causes progressive muscular degeneration. It was a first-to-market treatment for this condition and was approved in other countries prior to its clearance in the US. Initially, patients in North America could try the medicine if caregivers facilitated patient participation in a clinical trial or expanded access program, explains Maloziec.
But, when the drug was approved by FDA, suddenly patients that were already on therapy confronted questions such as if the new product would be covered by their insurance and if there would be medication gaps while going through the prior authorization process. Even with coverage, patients still faced affordability and access questions. Further, the specialists that supported the small patient population indicated for the treatment were specialty-naïve and unfamiliar with manufacturer-limited distribution networks and the prescription referral and prior authorization processes requiring patient and clinical information.
“Working closely with the manufacturer, we designed a program focused on continuity of care as patient prescriptions were transitioned and filled through our pharmacy,” says Maloziec. “The program includes physician and practice education and a hub model that is clinically integrated with the pharmacy to ensure high-touch caregiver support and integrated data back to the manufacturer.”
According to Maloziec, pharmacy experts help secure prior authorization approvals and letters of agreement, as well as copay and foundation assistance, to expedite the start of therapy. “This is one of many examples of our specialty pharmacy working in close partnership with the manufacturer and other healthcare stakeholders to implement a program that addresses the unique clinical profile of the therapy and the specific needs of the patient population,” he says.
The specialty pharma trend in the life sciences doesn’t seem like it’s slowing down, or getting any easier. As scientists continue to innovate and develop novel therapies, the pricing, reimbursement, and delivery conversations will continue to get more complex.
“Specialty pharmacy will be the new pharmacy,” Arquette told Pharm Exec. “As more drugs are developed and approved by the FDA as specialty drugs and the focus of these medications is the treatment of diseases that have been historically managed by small molecule therapies, specialty drugs will be the mainstay of prescription drug therapy.”
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