Only a small percentage of the rare diseases that have been identified have approved therapies available to treat them. However, more and more orphan /rare disease (ORD) drugs are being developed and approved for use in the U.S. market. In fact, according to the FDA’s Orphan Drug Designations and Approvals database, there have been 668 orphan drug approvals since the passage of the Orphan Drug Act in 1983. With increased ORD drugs entering the market, there is increased scrutiny on their effectiveness and cost, making the overall product strategy even more important to get right.
For ORD products, it is not only a matter of getting product approval and promoting the product to prescribers. For many of these products, just finding the right patients can be a monumental challenge. Once the patient has been identified, challenges begin to mount related to product supply, sites of care, reimbursement and affordability. As manufacturers develop the commercialization strategy for an ORD product, it is important that they focus across each of the 5 Ps of commercialization: Product, Prescriber, Payer, Pharmacy and Patient:
For some indications, the act of getting the product approved can be the biggest challenge. As the design of the trial sets the guidelines for storage, handling and administration that the FDA approves, it is critical to consider the practicalities of trial design, and not just race for approval. There is a long list of products that suffer post-approval from short-term decisions in trial design.
Sometimes the most difficult part in treating an ORD patient is getting them diagnosed and then in the right treatment center. With limited patient populations, it is important to focus on the right physicians, and even more specifically the right sites of care, as treatment options and the path to treatment can vary across each of them. For example, the path to treatment can be very different for a patient seeking treatment in a specialist’s office vs. a hospital, and the reimbursement may also be substantially different. With so many rare diseases, most physicians are not familiar with many of them, and both awareness and diagnosis become important educational elements of any commercial strategy.
With small patient populations comes high costs for treatment, as the cost of development does not care if it is for one million potential patients or for one hundred. However, payers do care as the annual costs of several therapies have now exceeded $500,000 per patient. Given the number of ORD drugs entering the market, and the increasing focus on precision medicine and gene therapy, ORD drug costs may only continue to rise. Partnering with payers early is critical, as no payer wants to deny coverage for an ORD patient, but they need to plan and prepare for how these drug costs will be paid. Manufacturers that start talking early to payers and setting expectations on drug pricing have been greeted with more acceptance than those that avoid the conversations and are surprised by the level of payer resistance and access barriers.
Getting the product from manufacturer to patient can be a complicated process, and even more so for some complex ORD products. Some ORD drugs may have a very limited shelf life or require cold chain distribution. Others may involve special device requirements or new patient starter kits to be delivered with them. Regardless of the eventual path to the patient, there is often significant need to manage and coordinate across distribution and dispensing partners, HUBs or other patient services programs, physicians and the patient. For example, with the new CAR-T therapies, we are seeing completely new supply chains outside of traditional or specialty distribution channels, outcomes-based pricing and long monitoring periods all necessitating new patient support services.
Underpinning most every strategy for a rare disease product is the patient and oftentimes also a properly executed patient services strategy. Given the small number of patients involved in ORD clinical trials, positive data may be difficult to achieve at a level sufficient to result in a positive FDA decision and approval. Additionally, given the rarity of these diseases, it is often necessary to combine a product strategy with some form of diagnostic or patient identification strategy, whether or not it is part of the approval pathway. Finding the rare disease patient can be a difficult challenge, and once they have been identified, every step should be taken to ensure that patient is carefully managed through the process of diagnosis, treatment and reimbursement, and that they (and their caregiver in many cases) receive the support and care needed. For ORD strategies, the emotional component is a huge factor for the patient and caregiver as many of these patients have been through a long process already—multiple doctors, multiple sites of care—that may have already resulted in long periods of frustration to get to treatment.
For an ORD drug, the commercialization strategy needs to include each of the component strategies independently, as well as in combination. Only by taking a holistic approach that centers on the patient and looks at all the interdependencies as well as the impact of each strategic decision point on the other strategies will a manufacturer develop the most effective ORD drug strategy.
By focusing on each of the 5 Ps (Product, Prescriber, Payer, Pharmacy and Patient), both independently and holistically, we can successfully bring these drugs to market and to the patients that so desperately need them.