Blastic Plasmacytoid Dendritic Cell Neoplasm

For years, many manufacturers have assumed that pre-market physician education was not strictly necessary unless their brand was the first to market or had a novel mechanism of action. With the advent of precision medicine, however, ongoing disease state education has become increasingly critical. Todays molecular-targeted therapies have very different treatment paradigms, not to mention cost structures. While it stands to reason that purportedly curative therapies, like those for hemophilia, would require more intensive education campaigns, the need for greater pharma investment in physician education is not limited to precision medicines. Evidence-based physician education is now an essential element of market access planning for any new drug, regardless of whether it is the first therapy in an indication or the fourth. The role of first-to-market manufacturersManufacturers of first-to-market therapies reap the benefits of providing the first therapeutic option for patients in need, but they also assume the tremendous responsibility of comprehensive disease state education. Until the advent of the first therapy, specialists are just treating symptoms. They may be familiar with the diagnosis and know which protocols are best, but they are unlikely to know the latest research on disease progression, or which clinical factors matter most.Manufacturers of breakthrough drugs often rely on real-world data to find diagnosed patients and their treating physicians. To develop a strong physician education program, manufacturers must ensure that the physicians involved with their clinical trials become true key opinion leaders (KOLs). They should be tapped to provide disease state education to their peersand specifically to potential prescribersin published research and public commentary.Using real-world data to select optimal trial sites and improve enrollment is also pivotal for a manufacturers physician education initiatives. Physicians who enroll multiple patients in a clinical trial are also poised to become KOLs for the new therapy. Once a brand comes to market, they can provide first-hand anecdotes about how therapy impacted their individual patients. Fortunately, much of this physician disease state education can be done before a drug is approved. If a manufacturers KOLs have successfully raised awareness about the latest research and the imminent release of a breakthrough drug, physicians will be eagerly awaiting its launchand the manufacturer will not experience a lag in sales. In fact, research indicates that pre-launch medical KOL education increases treatment adoption in the first six months after launch by 150%. Ongoing education on brand differentiators For subsequent therapies in an indication, disease state education remains critical. Developers of these therapies shouldnt rely on the broad education provided by the first-to-market manufacturer, as research will have evolved considerably in the interim. Along with additional education on the overall disease, manufacturers should also stress their products differentiators. As a third-to-market therapy is likely treating a nuance of the disease that other drugs did not, its important to fully explain how it benefits patients, and which patients are ideal candidates.This is especially true for cell and gene therapies, which are changing the structure of a patients gene or their messenger RNA. Using real-world case studies, physicians must be taught how to identify a particular subset of the total disease population and apply eligibility criteria for new medications. While a drugs label may state that patients should take an FDA-approved genetic test to determine eligibility, the exact test is not always specified. Manufacturers must educate physicians on the testing processincluding policy and reimbursement requirementsso they can quickly diagnose patients and begin treatment. Refining a brands value proposition Early physician education also helps pharma companies zero in on the most persuasive value proposition for their new drug. Long before FDA approval, manufacturers should take the pulse of relevant physicians to understand their perceptions of the available treatment options. Are physicians reluctant to use a specific therapy because of its side effects? Charting treatment pathways and provider preferences can help pharma companies better determine an agents key differentiators. Targeting well-known and influential physicians who specialize in the relevant therapeutic area can also give manufacturers perspective on how their brand will likely be received. Before a drug hits the market, pharma companies should research how physicians are currently treating patients in this indication. What treatment protocols have already been tested, and whats been most effective thus far? How will the new brand be more beneficial than its competitors? Physician education and research also helps manufacturers refine their messaging for their existing portfolio. After a drug has been on the market for a while, its important to keep an eye on HCP perspectives. Are new side effects now being discussed in the physician community? Do physicians need additional education on eligibility requirements or prescribing scenarios?Avenues for reaching targeted physiciansPharma companies arent always sure how to go about physician education, even when they understand that its necessary. Physicians learn about new medications in myriad ways, from continuing education workshops to industry journals and manufacturer communications. Which is the best avenue for disease state education? Research shows that physicians place a high value on in-depth disease state conversations with medical science liaisons (MSLs), who are becoming a primary driver of HCP engagement. Specialists are often receptive to discussing their therapeutic area with an MSL, who can point out relevant clinical data and offer a more research-oriented view. Multi-channel engagement models, in which manufacturers offer just-in-time content personalized to the physician, are now common.Online medical communities, in which physicians speak directly to each other in specialty- and indication-specific groups, are also an immensely valuable resource for pharma companies. KOLs can share their patient treatment experiences with other specialists, detailing which factors lead them to prescribe one drug over another and discussing outcomes. They can dialogue with peers about the best treatment paths for certain subsets of the population, share clinical trial information, and spread awareness about new therapies on the horizon.Peer-to-peer physician education can be particularly useful for complex disease states and indications which are commonly misdiagnosed. Physicians who are struggling to diagnose an individual patient often seek advice from their peers in a specialty community. For example, a specialist might share specific lab tests or secondary symptoms to help other specialists differentiate between blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML).Fulfilling the demand for evidenceManufacturers are used to providing evidence-based education to payers. Along with a drugs clinical efficacy data, they share cost-benefit analyses on the long-term financial impact of treating a patient population. They track comorbidities and disease progression, amassing evidence to educate payers on why an expensive drug is more cost-effective than it first appears. Manufacturers must also practice the same kind of evidence-based education with physicians. Early and continuous disease state education can help physicians recognize the clinical factors that indicate a patient will respond well to a particular therapy. Physicians are aware that the standard of care is always evolving, and theyre curious about new treatments. Pharma companies should invest in education initiatives that help HCPs apply the right treatment paradigm to the right patient, without delay. Ready to engage with target HCPs? Drive disease state education on Skipta, the online network of medical micro-communities focused on specific conditions and specialties. '

According to the latest statistics, cell and gene therapies are rapidly emerging as a hot topic in the pharmaceutical field due to their unique therapeutic advantages. A recent report has revealed the top 10 best-selling cell and gene therapy products globally in 2023. At the top of the sales ranking is Yescarta, Gilead's CAR-T cell therapy product, which leads the global CAR-T cell therapy market with sales of $1.498 billion, making it the overall sales champion in the cell and gene therapy field. Closely following are two products from Novartis, Zolgensma and Kymriah, which generated sales of $1.214 billion and $508 million, respectively, ranking second and third. In addition, several other products that made it to the TOP 10 also performed remarkably well, including Carvykti co-developed by Johnson & Johnson and Legend Biotech, Abecma co-launched by BMS and 2seventy bio, and Tecartus from Kite Pharma. It's worth mentioning that if the two antisense oligonucleotide (ASO) therapies for Duchenne muscular dystrophy, Exondys 51 and Amondys 45, developed by Sarepta Therapeutics, were included in the ranking, their 2023 sales of $540 million and $270 million, respectively, would also place them among the top ten. Looking at the sales ranking, CAR-T cell therapies occupy half of the market, fully demonstrating their outstanding position in the treatment of malignant hematological cancers. Meanwhile, gene therapies based on viral vectors, such as Zolgensma and Luxturna, have also shown impressive market performance. Looking forward, with the continuous emergence and launch of more innovative therapies, the cell and gene therapy field will embrace a broader development prospect and unlimited market potential. Menarini Group, as a leading pharmaceutical company in the Italian industry, has weathered 140 years of storms and emerged as an important force in the European and global pharmaceutical industry, thanks to its deep accumulation and excellent technology in the biopharmaceutical field. As a pharmaceutical giant with unique technology, products, and a huge R&D system, Menarini has always focused on the research and development of drugs for cardiovascular diseases, inflammatory pain, gastrointestinal diseases, oncology, and metabolic diseases. Headquartered in the historic city of Florence, the company has a team of over 17,700 excellent employees and operates in more than 140 countries around the world, with numerous subsidiaries, distributors, and franchisees. In recent years, Menarini has actively expanded its international business footprint and strengthened and expanded its position in the global market through a series of strategic acquisitions and collaborations. In the fields of oncology and infectious diseases, Menarini, with its rich product line and innovative technology, has provided many high-quality treatment options for patients worldwide. Take Tagraxofusp as an example, it is the first approved drug for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) and the first approved CD123-targeted drug, which has brought unprecedented hope for the clinical treatment of BPDCN patients. Overall, Menarini, with its 140-year deep accumulation and the spirit of continuous innovation, has achieved remarkable results in the fields of oncology and infectious diseases, and continues to provide patients with better treatment options. At the same time, the company also demonstrates strong competitiveness and broad development prospects in the international market.

As a traditional power in European biopharmaceutical manufacturing, Italy possesses outstanding technology, excellent products, and a comprehensive R&D system in the pharmaceutical field. Among them, the Menarini Group, as a leading company in the Italian pharmaceutical industry, ranks among the top European pharmaceutical companies and is among the global TOP50 pharmaceutical companies. Since its establishment in 1886, the Menarini Group has always focused on pharmaceutical R&D in areas such as cardiovascular diseases, inflammatory analgesia, gastrointestinal diseases, oncology, and metabolic diseases. The company's headquarters is located in Florence, with over 17,700 employees worldwide, and has established subsidiaries, distributors, and franchises in 140 countries, forming a widespread and in-depth international business network. In recent years, Menarini has continuously expanded its international business footprint. In 1992, it successfully entered the German and Central and Eastern European markets through the acquisition of Berlin-Chemie; in 2011, it acquired the Invida Group, further consolidating its market position in the Asia-Pacific region; and in 2020, it successfully entered the US oncology market through the acquisition of the biopharmaceutical company Stemline Therapeutics, demonstrating its strong market expansion capabilities and strategic vision. In the fields of oncology and infectious diseases, Menarini is highly regarded for its innovative drugs and technology products. Its product line covers innovative drugs such as small molecules and biologics for the treatment of hematological malignancies and solid tumors. At the same time, the company is also actively investing in the development of diagnostic technologies, aiming to develop high-speed, high-precision diagnostic instruments covering clinical chemistry, diabetes, immunology, hematology, histology, and point-of-care testing. Taking Tagraxofusp as an example, this drug is an important innovative achievement of Menarini in the field of cancer treatment. As a CD123-directed cytotoxin, it has been approved for the treatment of adult and pediatric patients (2 years and older) with blastic plasmacytoid dendritic cell neoplasm (BPDCN). As the first approved treatment for BPDCN and the first approved CD123-targeted drug, the launch of Tagraxofusp has brought new hope and choice for the clinical treatment of BPDCN patients. In summary, as a leading company in the Italian pharmaceutical industry, Menarini, with its 140-year historical tradition and continuous innovation spirit, has achieved remarkable results in the fields of oncology and infectious diseases. The company is committed to providing patients with better treatment options and has demonstrated strong competitiveness and broad development prospects in the international market.