Advancing Rare Disease Drug Development: Four Key Considerations for Market Access

Our industry is witnessing increasing growth in the rare disease market, thanks to financial and regulatory incentives to develop orphan drugs. This has been good news for both sponsors and patients, but the fact remains that rare disease trials are inherently challenging to run. In addition, completing a complex study and reaching regulatory approval does not necessarily translate to market success.

John D. McDermott, Jr., Vice President of Market Access Services, recently shared his insights on the market access challenges in rare disease drug development and discussed key considerations for sponsors and stakeholders.

Provide early education about the disease

Even though rare diseases as a whole are getting more attention, sponsors cannot assume that their potential payers know much about the particular condition they are targeting and its importance to patients.

By developing evidence that covers the natural history of the disease and highlighting patients’ burden of illness, sponsors can inform payers about the unmet medical need and its consequences for patients. These data can also define the subsequent value proposition. Sponsors should consider “marketing” the disease to payers to overcome potential payment hurdles and ensure greater acceptance at launch because payers have to understand the disease that they are providing drug coverage for.

Focus on the patient perspective

Rare disease patients are critical for gathering clinical trial data, but data about their personal experiences while on the treatment can be equally valuable. Sponsors are in a unique position to learn more about the patient perspective in rare diseases to better understand their needs beyond the clinic.

Patient-reported outcomes (PROs) and other health-related quality-of-life instruments can help sponsors generate crucial evidence to appeal to patients. These tools improve understanding of the treatment’s benefits and inform patients about the everyday impact of the drug on their lives. Coupled with partnerships with patient advocacy groups, a strong connection with patients can ease future recruitment burdens, guide the choice of relevant endpoints in clinical studies, and build strong demand for the drug after launch.

Evaluate potential payment hurdles and payer sensitivity

When orphan drugs launch, they are often priced many times higher than non-orphan drugs to offset the development costs of treating very small populations and generate sufficient return on investment. Even with comprehensive insurance, the co-payments or out-of-pocket expenses for rare disease treatments can be prohibitive. Sponsors need to carefully consider the financial burden on patients and ensure that they can afford to pay for the drug.

Co-payment assistance programs can direct patients to resources such as non-profit foundations that assist with co-payments and help improve access and retention. Manufacturers may also consider offering their own patient assistance programs for insured or uninsured patients who cannot afford out-of-pocket expenses. Regardless of the final price, sponsors should create robust market access support programs that consider patients’ needs for these crucial reimbursement case management services.

Understand the route to market and its implications

Sometimes, sponsors have the option to pursue either orphan or non-orphan indications for the same product. Orphan drug development can be very attractive to sponsors due to potentially expedited approval, longer exclusivity, and lower levels of investment needed to take a drug to market. However, an orphan pathway to market is not always the optimal choice.

Sponsors choosing between orphan and non-orphan indications must evaluate multiple dimensions of their market to choose the path with the highest commercial potential. Some factors to consider are the comparative sizes of the patient populations, the expected prices, the investment needed, the duration of development, and the impact on follow-on indications. Careful planning and investment modeling can help sponsors select the most attractive route to market.

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Effectively running a rare disease clinical study and bringing a treatment to patients is a challenging process. We offer over 25 years of market access consulting experience for rare and orphan products. We can leverage our track record supporting more than 50 indications to help you realize the full market potential of your rare disease drug development program.

Learn more about our rare disease efforts and our market access solutions.

Advancing Rare Disease Drug Development: Four Key Considerations for Market Access

Provide early education about the disease

Focus on the patient perspective

Evaluate potential payment hurdles and payer sensitivity

Understand the route to market and its implications

John D. McDermott, Jr, MBA

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