Patients should be at the center of strategies to commercialize rare and orphan disease treatments. Consider the following questions to set products up for maximum reach and impact.

1. What are the essential elements in any rare and orphan drug commercialization strategy?
It starts with the patient and their family and hearing stories of the daily burden of living with the disease. A life sciences company needs to understand the patient's journey before the rare disease was even diagnosed and potential treatment options identified.

Understanding the patient goes beyond their medical history and into the financial stressors. Are they paying for multiple drugs? What is their benefit design and what happens if their coverage changes given this is most likely a lifetime therapy? How can life sciences companies center their strategy around what a patient and their family needs and help them access care when they need it?
Communication with payers is another essential element to commercialization. Do payers understand the specific disease and the burdens patients face in terms of cost of care? What is the current standard of care?

2. What are important channel strategy considerations for rare and orphan products?

Fundamentally, rare and orphan products are going to have a smaller patient population and they are generally going to be higher cost products. When life sciences companies look at overall distribution across the market that points to specialty distribution with inventory strategically stocked at fewer locations. Downstream customers will most likely not be buying and stocking rare and orphan products for a period of time, costing them money. Life sciences companies should work with a specialty distributor who can deliver their products the following day, on-time.

Life sciences companies must also consider targeted customer classes of trade that will have access to these products. The uniqueness of the class of trade and access strategy for downstream customers requires partnering with a specialty distribution partner who brings a high level of expertise and a high level of customer account management and identifiers.

3. For maximum success, when should life sciences companies begin commercialization planning?

Engagement with patients and their families must start early. Life sciences companies must also develop rapport with physicians and patient advocacy groups to gain an understanding of treating the rare disease. All of this relationship-building starts before a life sciences company even approaches a commercialization strategist. Working with that commercialization partner at least 12-to-24 months pre-launch to develop the appropriate go-to-market strategy is ideal.

4. What should life sciences companies keep in mind when commercializing rare and orphan products?

Life sciences companies need to look at rare diseases through the lens of health equity issues and how those impact patients. There are outcomes disparities between patients who have an abundance of support, financial resources, and access to care from specialists and those who lack those resources.

Another key consideration around health equity is access to clinical trials. Diversity issues related to who is enrolled in the clinical trial are huge. Patient advocacy organizations like the National Association of Rare Diseases, the Rare Diseases Clinical Research Network, and the International Rare Diseases Research Consortium can address the design of clinical trials to help their constituents get greater access. One outcome of COVID-19 was how it shifted workflows and created the opportunity to re-think access with at-home clinical trials, giving life sciences companies opportunities to reach patient segments underserved by traditional clinical trial set-ups.

Life sciences companies must also consider the implications of treatments which require gene therapy, such as cost, access, and the need for patient registries to track outcomes after a single treatment. What happens after that single treatment must be carefully analyzed, as do the long-term outcomes of patients on gene therapy.

5. What should life science companies look for in a specialty distribution partner?

The most important things are previous experience and processes that are flexible to adapt to any unique needs. Does the specialty distribution partner have the resources to collaborate with them to find solutions that will provide a successful launch? Life sciences companies are usually trying to reach a specific site of care for rare and orphan products. They need a distributor with depth of visibility who can control access and target that customer base. Rare and orphan products often receive additional indications and approvals beyond the initial launch and life sciences companies need a partner that partner can expand with them and reach additional customers immediately.

They also need to look for a partner who has business continuity and infrastructure plans in place. The COVID-19 pandemic really put a big spotlight on business continuity, something that was previously taken for granted or overlooked. Rare and orphan products serve serious diseases and therapies that can’t be interrupted. Does their distribution partner have a network of facilities across the country with redundant systems that ensure immediate response if there is ever a shut down?

6. What specific barriers might life sciences companies face in commercializing rare and orphan therapies?

Barriers exist in several different areas. One of them is awareness among the medical community. How does the disease manifest itself, how is it diagnosed and what treatment options are going to be available when the product is approved? It's critical to identify patients who may have been seen by multiple physicians who have been lost in understanding the diagnosis. The more healthcare providers know about a disease, the more likely they are to get patients to the right diagnosis and treatment.

Another barrier is financial. Insurance companies or life sciences companies may need to offer assistance programs to help patients shoulder the out-of-pocket costs. Uninsured patients face an even greater financial barrier, especially those from underserved communities facing additional health equity issues.

Geography is a third barrier. Do patients live in a region where they have convenient access to care or are they miles away from an appropriate treatment center? Can treatment be brought closer to them? Life sciences companies should think through the appropriate wraparound services to offer, such as transportation or telehealth nursing services.

7. How does the distribution of rare and orphan products specifically differ from other specialty products?

There’s a real focus on just-in-time shipping. Which customers will have access? Is it going to be specific institutions? Is it going to be specific areas within a health system? Is it going to be a specific practice type in the physician or community space?

Another difference from other specialty products is, because of the diseases these products treat, there’s a need for increased visibility and following the product in the patient throughout the course of their therapy. Life sciences companies should look for a partner who can capture specific patient identifiers, giving them visibility of data through the entire distribution process, so they can tie a specific order to a specific patient or provider.

One additional area with differentiation from other specialty products is 340B access for orphan drugs. 340B is entirely dependent on each individual life sciences company’s strategy for the product, so they will need solutions that are flexible to meet any discount strategy.

8. What role should data play in rare and orphan commercialization?

Data creates an opportunity for a much better quality of life for those patients who thought it was hopeless. Evaluating EHRs and analyzing diagnoses and symptoms, a physician can pull together the puzzle pieces for final diagnosis.

Every patient's journey offers essential data that supports identification of potential barriers to care driving requirements for orphan product commercialization. This is a different approach from broader patient populations and life sciences companies need more data, not less. There should never be a patient left behind for treatment and support.

Understanding when patients go on therapy and tracking adherence is an important component of data gathering. Are there trends around when patients fall off therapy or never fill the prescription in the first place? A patient support program can fill in a lot of those gaps. Life sciences companies need to track the continuous cycle of treatment. For example, did the patient move and not receive their medication in their new location? That's why a robust data strategy is needed for close observation of any potential adherence barriers.

9. Where does payer strategy fit into the picture?

Life sciences companies need a robust communications strategy that educates payers on the underlying cause(s) of the disease, how healthcare professionals diagnose and treat it, and patient cost burden. What, if anything, is the current best practice protocol for treatment and how is this new therapy an improvement? When a new therapy is approved, it's going to be important for payers to understand the value of the treatment and the benefit for the patients. Life sciences companies need to be advocates for those patients and lift barriers related to payer coverage.

Ideally, those conversations with payers should begin early before final approval of a new rare disease product. Participating in pre-approval information exchange gives payers the ability to review collateral around the course of the disease and patient journeys. It's important to educate payers on the disease first. This puts them in a stronger position to understand the rare disease and the product’s indication. . Then minimizing delays on coverage approval from the payer as patients go on the therapy and products move from clinical trial to full commercialization.