Rare Disease Pharmacy Insights

PANTHERx Rare has been chosen by Upsher-Smith Laboratories as the specialty pharmacy for VIGAFYDE, a concentrated oral solution used to treat infantile spasms in children aged one month to two years. This decision underscores PANTHERx's reputation as a pivotal entity in offering access and support services for rare diseases in the United States. Infantile spasms, characterised by brief seizures and muscle stiffness, require urgent attention due to potential risks like developmental delays and epilepsy.

PANTHERx, renowned for addressing rare conditions, offers integrated solutions, ensuring medications like VIGAFYDE reach those in need promptly. They are experts in managing rare neurological conditions, providing vital support to parents from the hospital to home care. The pharmacy's dual accreditations and multiple awards affirm its commitment to excellence in healthcare access for rare diseases. This collaboration with Upsher-Smith highlights the critical role of specialty pharmacies in transforming treatment access into practical home-based care.

Precision AQ, a leader in life sciences, has bolstered its Access Experience Team by appointing Julia Wermerskirchen, PharmD. With her vast expertise in rare disease access strategy, she will help the company address the intricacies of navigating payer scrutiny and policy shifts. As the rare disease sector grows, so does the demand for rigorous clinical and economic evidence, prompting the need for more robust reimbursement strategies.

Julia's impressive background includes leading national managed care contracting to improve access to orphan drugs. Her role will enhance Precision AQ's ability to anticipate market access challenges and foster sustainable access to innovative treatments. This strategic expansion underscores the company's commitment to bridging the gap between payers and life sciences firms, ensuring that transformative therapies reach those who need them most.

Kedrion Biopharma broadens access to Ryplazim, a critical treatment for plasminogen deficiency type 1 (PLGD-1), a rare but serious condition causing mucosal lesions. The disorder can lead to severe issues such as vision loss and airway obstruction. With the FDA greenlighting tech transfer and manufacturing capacity expansion, Kedrion enhances its distribution network. This results in a wider reach across the US, incorporating new partners like CuraScript SD and specialty pharmacies CVS Health and Soleo Health, ensuring timely treatment for patients.

Kedrion aims to bolster the identification of PLGD-1 patients, smoothing their path to acquiring this FDA-approved therapy. Ryplazim, however, comes with notable safety considerations, including potential bleeding and hypersensitivity reactions. The initiative underlines Kedrion's commitment to improving patient care through robust distribution and collaboration, ensuring essential therapies are readily available to those in need. This move signifies a step forward in rare disease management, reflecting a dedication to health accessibility and patient support.

The implementation of an electronic early treatment assessment and monitoring (eTEAM) questionnaire for oral anticancer medications seeks to streamline the identification of adverse effects necessitating pharmacist intervention. Embedded within the electronic health record patient portal, the eTEAM questionnaire is sent to patients 7 to 14 days post-counselling. Notably, it achieved a response rate of 47% from 182 patients, leading to 29 pharmacist interventions mostly due to adverse effects or adherence issues. Pharmacists endorsed the questionnaire for its practicality and positive influence on practice, suggesting enhanced documentation of medication start dates.

The RE-AIM framework evaluated the eTEAM’s reach, effectiveness, and permanence, revealing its favourable reception among patients and pharmacists alike. Recommendations for improvement included standardising documentation practices. Overall, the eTEAM questionnaire emerged as a valuable tool in early identification and management of treatment complications in patients beginning oral anti-cancer therapies, signalling a promising future in enhancing patient care efficiency.