Innovative Patient-First Approach Enhances Patient Journey, Mitigates Rising Costs for Rare Disease Drugs


A patient’s ability to pay for specialty medications will continue to be an issue because drug costs are prohibitive and add barriers to accessibility.

Specialty medications typically account for more than 50% of all drug spend but only comprise less than 2% of total utilization. For this reason, specialty pharmacy (SP) has gained increased attention from stakeholders across the care continuum, including patients, payers, life sciences, and providers.

For specialty drug manufacturers, this is a positive trend because greater focus in this area will help to ensure that specialty drugs can be seamlessly accessed by prescribed patients, and that drug therapy is monitored for efficacy, compliance, and adherence to treatment.

As the industry examines key trends for SP in 2022, it will be important to consider these issues:

  • Continued utilization and rising costs of specialty agents.
  • Emergence of ultra high-cost specialty agents and cell/gene therapies for rare and orphan diseases.
  • More focused management of high-cost specialty drug patients to ensure compliance and optimize outcomes.
  • Vertical integration of specialty pharmacies into health care organizations.

In this environment, a patient’s ability to pay for specialty medications will continue to be an issue because drug costs are prohibitive and add barriers to accessibility. In fact, out-of-pocket costs for specialty drugs have increased faster than gross domestic product (GDP) growth over the past decade. This signals greater need for financial assistance programs, which are essential for patients to start and stay on therapy as prescribed.

For SP businesses and practices, the appropriate delivery and management of specialty drugs is a key component of a patient-first approach to caring for patients with rare disease—in which every care decision is based on what is best for the patient. This strategy also allows all stakeholders to connect effortlessly, rather than operating independently to achieve a higher level of care continuity that strengthens communication, yields rich data for optimizing outcomes, and improves the overall patient experience.

Patient-First Addresses 2022 Trends

Continued utilization and rising costs of specialty agents

An innovative SP solutions partner will use patient care coordinators and pharmacists with the broad specialty therapeutic expertise needed to rapidly identify financial resources, such as co-pay and foundation assistance to help make specialty products more affordable. Some organizations can also offer unique financial solutions for gene/cell therapies to help offset the costs of these potentially curative therapies.

Emergence of ultra high-cost specialty agents and cell/gene therapies

The growing pace of innovation made possible by emerging scientific breakthroughs and new understanding of diseases have led to the development of novel therapies, although many rare diseases remain untreatable. The sheer number of new patients in the care pipeline is rising steadily, with some having multiple conditions that require a specialty drug.

Given the level of transparency and communication it fosters, a patient-first approach creates the best environment for managing rare conditions and ensuring that the appropriate therapy is prescribed, at the right time, and in the right setting.

More focused management of the high-cost specialty drug patients to ensure compliance and optimize outcomes

Patient-first SP solutions should be uniquely designed to engage the services of specialty-focused professionals who have the experience and capabilities to mitigate costs and improve outcomes. Specialized expertise is necessary to transcend the scope of competencies provided by traditional, legacy care organizations, which are often designed exclusively for scale.

The most innovative SP solutions organizations provide a boutique and consultative patient support/hub model that includes a dedicated, branded team. This is important for overcoming the limitations of the standard SP and hub service providers, which often rely too heavily upon technology solutions that fail to address human needs and variability, and customizations based on client needs.

It's also important that the solution offers care coordination with a team approach that includes SP-focused pharmacists, clinicians, nurses, and other support staff—all focused on the disease state, patient community, and therapy. This adds a layer of customized care coordination and telehealth solutions to foster discussion between the patient, pharmacist, and other clinicians.

By incorporating assessments and interventions at key points in therapy, the care team is able to manage side effects and capture real-world evidence around the therapy, condition and patient’s quality of life.

Vertical integration of specialty pharmacies into health care organizations

More than three-quarters of specialty drug dispensing revenues in 2020 were shown to be with the SPs owned by pharmacy benefit managers that are part of vertically integrated, consolidated operations. Notably, 9 in 10 large hospitals now have their own in-house SP.

In health care, a vertically integrated system can offer a broad range of different patient care and support services. For example, SPs are the primary provider of oncology-related medication therapy.

SPs within an integrated organization, however, may lack flexibility and can benefit by partnering with a patient-first SP solution to create more holistic decision support, reduce variation in care, enhance access to evidence-based information, build a better health care experience, and produce optimal outcomes.

Other Trends

With many plan sponsors now carving out specialty drugs from the medical and pharmacy benefit, it is evident that a primary goal is to reduce costs. But with expanded availability of specialty drugs and documented enhanced patient health outcomes, payers will require appropriate medication therapy management and utilization management programs that can be customized and aligned with each plan sponsor to accomplish their multiple objectives.

Additionally, in 2022, biosimilars are poised to trend upwards due to a number of additional blockbuster drugs losing patent protection. The rising burden of various chronic diseases, such as cardiovascular disease, diabetes, and cancer, will continue to fuel the biosimilar market with affordability impacting market adoption.

Patient-First, Pharmacy-First Services

It's important for pharmaceutical manufacturers to align with a patient-first, pharmacy-first hub provider that serves as a true collaborative partner—one that is backed by a team that has a thorough understanding of the payer mix and value across multiple specialty categories. Ideally, the partner should offer:

  • Deep bench experience and expertise in payer reimbursement and strategy development.
  • In-depth understanding of the competitive environment.
  • Programs designed to drive branded volume and provide data-driven cost savings analyses that result in optimal patient access to products in major specialty and rare disease categories.
  • Customized clinical support programs and interventions that result in better compliance to therapy.
  • Limited distribution drug fulfillment with national reach and centers of excellence across many specialty conditions.

High-touch and technology-backed capabilities to further improve the patient experience and enhance outcomes, ensuring that patients can seamlessly access medications and obtain the high-touch support they need.

A patient-first approach offers a more “human touch” for people facing chronic illness and uncertainty, transcends the limitations of the retail pharmacy approach, improves the level of care and clinical outcomes, and enhances the patient experience.

About the Author

Dea Belazi is president and CEO of AscellaHealth.

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