Pharma Tech Outlook: Specials Magazine

Currently, most gene therapies are being developed for rare and serious diseases. In vivo gene therapies tend to be one treatment only and many use invasive delivery methods. On the other hand, ex vivo gene therapies involve complicated and lengthy preparation processes. These and other complications made it very difficult to design place bo-controlled trials. So far, all approved gene therapy products have used historical data as control groups, either data from a natural history study (e.g., Zolgensma, Libmeldy) or historical data prior to treatment (e.g., Roctavian, Luxturna, Zynteglo). FDA opens to use historical (external) controls in specific circumstances. “FDA regulations recognize historical controls as a possible control group… however, inability to control for certain biases could limit the ability of externally controlled trials to demonstrate substantial evidence of effectiveness. However, bias may be mitigated in certain situations where the disease course is predictable and the treatment effect dramatic.” (FDA Draft Guidance “Rare Disease: Natural History Studies for Drug Development”)

Independent pharmacies across the U.S. are running into a wall with the Drug Chain Secuirty Act (DSCSA). The law now brings wholesalers fully into scope and changes how pharmacies must electronically receive and reconcile serialized shipments. Each unit carries a unique serial identifier, supported by interoperable digital transaction data that trace its path from manufacturer to pharmacy shelf. It is good for safety but hard on small teams. Chain pharmacies manage this transition with scale. They have integrated systems, a dedicated compliance staff, and a budget to maintain scanners, portals, and data storage. However, independent owners do not have that leverage since they are the pharmacist, the buyer, and the IT support all in one. A single mismatched data reconciliation can stop a shipment from being received. Incomplete or mismatched data files trigger exceptions that take time to fix. And since verification tools are still evolving, pedigree checks often add more delays than answers. What independent pharmacies need is simple. They need products that arrive already validated and easy to confirm. They also need a distributor that sources what big systems overlook, such as niche strengths and specialty generics that matter in real clinics. That kind of support does more than fix logistics. It keeps the personal side of pharmacy alive, where care is local, names are remembered, and patients are more than profiles. That is the belief that drives True Marker Pharmaceuticals. The company is a national wholesaler built around the realities of independent pharmacies. It treats compliance as part of daily work, not an added burden. Each shipment is processed in accordance with DSCSA serialization and verification requirements, so by the time it reaches the counter, pharmacists can stay focused on what matters most—the people they serve. “Independent pharmacies shouldn’t have to choose between meeting DSCSA requirements and serving their patients. We have built our distribution systems so that compliance happens quietly in the background, allowing pharmacists to keep doing what they do best while we handle the rest,” says Davin Deb, CEO..