MedCity Influencers, BioPharma

Meeting the needs of rare disease patients: Improved access to specialty pharmaceuticals, gene and cell therapies

Specialty carve outs that move specialty drugs away from traditional prescription drug management to a pharmacy benefit administrator (PBA) or specialty administrator will continue and likely to provide cost savings.

Anyone who steps into the shoes of a person with a rare disease will experience not only the disheartening prospects of deteriorating health but also the pressure to arrange for effective treatment sooner and prevent morbidity and mortality. There are 7,000 known rare diseases affecting about 30 million people in the United States, or roughly one in 10 people. The exact cause for many rare diseases remains unknown. Still, for a significant portion, the problem can be traced to mutations or changes in a single gene.

Many patients face years of a difficult “diagnostic odyssey” before receiving a correct diagnosis and encounter numerous obstacles for obtaining the specialty drugs or innovative gene therapies which may be life-saving. The high cost of some drugs can sometimes hinder access for rare disease patients who often struggle to attain affordable, adequate and accessible health care coverage. Unfortunately, more than 95% of rare disease patients lack an FDA-approved treatment for their condition. Rare diseases represent a burden for individuals and families and pose a critical public health issue. The National Organization for Rare Diseases (NOTD) maintains a Rare Disease Database providing brief introductions for patients and caregivers to specific rare diseases.

Promising outlook: Specialty drugs

The growing pace of innovation made possible by emerging scientific breakthroughs and new understanding of diseases has led to the development of novel therapies. The sheer number of new patients in the care pipeline is rising steadily, with some having more than one condition that requires a specialty drug.

Specialty drugs are typically defined using elements of the following criteria, identifying an agent as a specialty medication:

  • Used to treat rare or orphan diseases or complex chronic conditions
  • High-touch disease state that involves one or more of the following for the medication:
  • Requires special handling
  • Requires special administration
  • Requires special distribution or storage requirements
  • Requires special monitoring
  • Involves high-touch or frequent patient care management or clinical monitoring
  • Distributed through a limited distribution network
  • High cost

As a result of the high cost, Specialty drugs are often prohibitive and therefore, add a barrier to patient access.  In fact, out of pocket costs for specialty drugs have increased faster than the gross domestic product (GDP) growth over the past decade.

These issues contribute to the increased attention and growth of Specialty Pharmacy (SP) among patients and health benefits payers, as well as specialty drug manufacturers that want to ensure that their drugs are seamlessly accessed by prescribed patients, and that drug therapy is monitored for effectiveness, safety, compliance and adherence to treatment.

Specialty drugs might be covered through either medical or pharmacy benefit and coverage is usually tied to where the patient receives the drug: if the patient takes a pill or self-injects the drug at home, it is more likely to be covered through pharmacy benefit. If the patient receives the drug at a doctor’s office, hospital or an outpatient clinic, it’s more likely to be covered through the medical benefits portion of existing health insurance coverage.

One initiative that can provide substantial savings to the patient and payer is to identify alternative site of care program. These programs offer more convenience for patient and caregivers/support team members and enhance opportunities for patient engagement and care in addition to providing a lower cost to the patient and payer.

Innovative copay coupon programs provide an additional means for the patient and payer to utilize manufacturer provided funds to offset the cost of therapy. Use of these programs is already in place, improving the ability of patients to access these high-cost medications.

Looking ahead, specialty carve outs that move specialty drugs away from traditional prescription drug management to a pharmacy benefit administrator (PBA) or specialty administrator are likely to continue. These arrangements provide cost savings, remove any incentives to dispense product and present opportunities for management and control of the utilization management programs that align with payer expectations.

These programs also improve patient care coordination activities, educate patients and provide opportunity to enhance adherence to therapy. Specialty clinical support programs can also identify and mitigate barriers for continued patient compliance, and monitor side effects and adverse events, pointing to the need to modify or discontinue therapy.

Going forward, Specialty Pharmacy networks are likely to draw wide appeal with specialty drugs being dispensed and coordinated through identified specialty pharmacies with high-touch capabilities such as personalized patient support services and advanced storage and shipping capabilities. These hand-selected pharmacies serve as Centers of Excellence across specialty conditions and assist with improved care coordination, minimizing waste by preventing excess drug accumulation, and increasing compliance to therapy for optimal patient outcomes.

Gene therapies

Throughout the industry, many are envisioning a world where genetic blindness, severe muscular dystrophy, cancer, sickle cell anemia, blood disorders and many other diseases are not simply treated, but actually cured by gene therapies. As some of the nation’s leading pharmaceutical and biotech giants ramp up the development and commercialization of products, what was once simply a futuristic concept is becoming reality.

Gene therapies are not simply another new class of specialty drugs to treat symptoms of a given disease — they aim to cure by correcting the underlying genetic abnormalities causing the disease. With more than 900 investigational new drug (IND) applications for ongoing clinical studies of gene therapy products underway, and the FDA predicting they will be approving 10-20 gene therapies per year, the availability of these groundbreaking drugs is becoming an emerging driver of change in specialty pharmacy benefit management.

The dawning of genetic therapies that represent new and potentially life-altering options for the people who use them represents steep costs as multi-million-dollar drugs have arrived. In fact, a new record was set recently when the FDA approved Zynteglo, a gene therapy for beta-thalassemia, a rare disorder requiring regular blood transfusions that carries a record $2.8 million price tag and making it the most expensive drug on a single-use basis in the U.S. and among the highest globally. Others such as Zolgensma which treats spinal muscular atrophy (SMA) cost $2.1 million for a one-dose treatment and Luxturna for genetic blindness runs $850,000.

Stakeholders are discussing these issues, and the payment models being developed for the newly approved specialty drugs and gene therapies provide an early indication of the flexibility and coordination that will be needed from manufacturers, payers, and policy makers to optimize patient access.

Currently, one of the potential solutions to payer coverage is a loan-based assistance program, which converts high upfront cost of ultra-expensive therapies into a pay-over-time loan in small, predictable payments. This opportunity protects payers from therapeutic failures by use of drug product warranty and/or value-based or risk-based agreement, provides a mechanism for patient mobility transfers from one payer to another, reduces the need for stop-loss or reinsurance and enhances appropriate patient access to ultra-expensive therapies.

In time, the gene and cell therapy market is expected to venture beyond targeting only rare conditions, with leading SP innovators introducing solutions that address concerns of monitoring patient health outcomes and affordability.

Photo: cagkansayin, Getty Images

Andy Szczotka, PharmD, is the Chief Pharmacy Officer for AscellaHealth.

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