Gene therapy isn’t on the horizon anymore; it’s here. Precision medicine demands a level of personalization that few Specialty Pharmacies are equipped to handle. PANTHERx®’s pure focus on rare and orphan conditions has propelled the company to the forefront of the industry for program personalization, making it the ideal partner to bring new gene therapies to the market.
Gene therapy is characterized by the targeting of DNA or RNA as a pharmaceutical agent to treat or cure disease. The DNA/RNA either encodes a functional gene that replaces an existing mutated gene, or encodes a therapeutic protein rather than a naturally occurring human gene.
Gene therapy was first proposed conceptually as a therapeutic option to treat disease during the 1970s and the first gene therapy clinical trial was conducted in 1990. Over the past twenty-eight years, more than 2,500 clinical trials have been conducted using numerous gene transfer vectors to assess the efficacy of potential gene therapies. Today, gene therapy is one of the fastest growing therapeutic areas in biomedical science and has a rich therapeutic pipeline.
Over 600 gene therapies are currently in the pipeline for the treatment of a wide variety of cancers and more than 500 are in development to treat rare disease. Furthermore, with greater than 4,000 diseases linked to genetic disorders with no existing effective treatment options, gene therapy offers an opportunity to revolutionize treatment paradigms and redefine care for patients suffering from such devastating conditions. With only a small number of gene therapies approved by the FDA thus far, the current healthcare marketplace has not assessed how to effectively integrate the delivery of these novel medications to patients.
As a nationwide, quadruple-accredited specialty pharmacy that has won back-to-back Zitter Health Insights Patient Choice Awards, PANTHERx® is uniquely qualified to serve as a partner in the commercialization of novel gene therapies. PANTHERx®, with its focus on rare and orphan conditions combined with customized patient programs delivered by specialized RareCareTM teams, provides tailored solutions to all marketplace stakeholders including patients, providers, payers, and partners.
The successful commercialization of gene therapies requires unique program designs. Customized clinical patient programs, a broad and deep understanding of innovative payer reimbursement models, expert supply chain management, modified pharmacy staffing models, and proficient revenue cycle management are all necessary as parts of a comprehensive program. As THE Rare Disease SP®, PANTHERx® has experience delivering the most complex and innovative therapies ever developed. In fact, PANTHERx® has been selected to exclusively handle, distribute, and dispense a growing number of transformational products, each with differing profiles, that are delivered in concert with ultra-high touch patient services. Critical to the company’s success has been prioritizing the patient’s needs coupled with the ability to effectively collaborate, offer flexible solutions to partners, and maintain the nimbleness necessary to adapt to a rapidly evolving marketplace.
Long term clinical trial data for patients who have received gene therapies is lacking. As a result, pharmacies, patients, prescribers, and payers are all now challenged to develop reimbursement models that are based on patient outcomes. These treatments will require longitudinal patient monitoring and an ongoing dialogue between all parties long after patients receive treatment. Recognizing this marketplace need, PANTHERx® has developed a proprietary patient care management platform (SWFT) and has demonstrated industry excellence in the ability to capture patient outcomes. The platform’s unparalleled customizability has been used across several disease states and therapeutic categories, and is a perfect solution for the complexities associated with managing a gene therapy.
Due to the cost and complexities associated with gene therapy administration, Centers of Excellence (COE) are the ideal patient care sites for gene therapy administration. Proficiency coordinating patient site visits and the on-time arrival of the gene therapy at the COE for patients requiring gene transfer are also critical. PANTHERx® has an established, proven track record of successfully working with COEs nationwide for patients afflicted with rare and orphan conditions where associated therapies have extremely stringent cold chain shipping requirements.
The transition of gene therapies from the bench to the bedside has now become a reality and modern medicine has only just begun to unlock the potential for these transformative, lifesaving treatments. In the coming years many more precision medicines currently in clinical trial will be approved by the FDA. These advanced therapies will bring hope to patients who previously had none. PANTHERx®’s mission is to transform lives by delivering medical breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions. The emergence of gene therapy will play a crucial part towards fulfilling that mission. PANTHERx® is excited by the growth of gene therapy and is uniquely qualified to support its evolution into the healthcare marketplace like no other specialty pharmacy.
