Rare Disease Pharmacy Insights

This study, published in the Journal of Managed Care & Specialty Pharmacy, delves into the patient experiences and outcomes associated with insurance-mandated step therapy in treating dermatologic inflammatory conditions such as psoriasis and atopic dermatitis. It reveals that these insurance strategies, often intended to cut costs by requiring patients to try cheaper alternatives before accessing specialty medications, lead to significant delays in starting effective treatment. This delay isn't just a simple waiting game; for many patients, it translates to severe impacts on their quality of life as they struggle through ineffective treatments and administrative hurdles. Astonishingly, most patients eventually move on to the higher-cost specialty medications anyway, suggesting that these policies might be delaying the inevitable.

The investigation highlights that while formulary alternatives may lead to marginally faster access to medications, the step therapy requirement remains a clunky, bureaucratic hurdle in patients' treatment journeys. The findings encourage stakeholders in managed care pharmacy to reconsider the implications of these cost-containment strategies, potentially prioritising patient well-being over rigid protocol. This study is a clarion call for further research into the clinical and financial impacts of such pervasive insurance practices.

Selecting the right specialty pharmacy (SP) partner is crucial for the success of rare disease and orphan drug launches, as outlined by Richard Faris from PANTHERx Rare Pharmacy. The complexities of these therapeutic treatments necessitate strategic planning far in advance, with a good SP partner aiding in patient access, treatment outcomes, and the financial performance of a product. The landscape is not as straightforward as it might seem; manufacturers new to this niche must recognise that specialty pharmacies in rare diseases operate differently from traditional ones.

Key considerations involve understanding patient journeys, establishing collaborative partnerships, and opting for narrow or exclusive SP models, focussing expertise on rare populations. Furthermore, the decision to implement a patient services HUB can enhance care but adds layers of complexity and cost. A poorly chosen pharmacy can derail these efforts – illustrating the importance of diligence in pharmacy selection to ensure patient experience isn't a game of chance but rather a well-planned launch that maximises therapy access and outcomes.

The specialty pharmacy sector is expanding rapidly, driven by the increasing use of complex therapies such as cell and gene treatments, some of which cost over $1 million per dose. To navigate the financial and operational challenges associated with these high-cost medications, health systems are implementing collaborative, multidisciplinary approaches. By integrating pharmacists, clinicians, and financial specialists, these systems aim to enhance patient outcomes while ensuring sustainable access to specialty treatments.

Key industry trends are also shaping the future of specialty pharmacy, including the growing focus on rare and ultra-orphan disease treatments, the rise of cell and gene therapies, and the expansion of ambulatory infusion centers. These shifts require health systems and pharmacies to refine their strategies, ensuring they can meet evolving patient needs while maintaining financial stability in an increasingly complex healthcare environment.

Following the FDA's removal of tirzepatide from its drug shortage list on December 19, 2024, compounding pharmacies face new regulatory limits. Previously, both 503A and 503B pharmacies were allowed to compound tirzepatide under emergency provisions. However, with the shortage designation lifted, 503A pharmacies must stop compounding by February 18, 2025, and 503B outsourcing facilities by March 19, 2025.

After these deadlines, compounding tirzepatide will only be permitted under strict conditions. Pharmacies cannot compound versions identical to FDA-approved products unless a physician documents a medical necessity, such as an allergy requiring a modified formulation. Non-compliance could lead to FDA enforcement, particularly against pharmacies using non-compliant ingredients or failing to meet quality standards.