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Despite rare disease market growth, pharma leaders see development obstacles ahead

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Life sciences companies are leaning into the rare disease space, with the market expected to grow at a compound annual growth rate of 12.8% between now and 2030. Still, more than 90% of the 7,000 known rare diseases lack a treatment option.

And, as the industry is all too aware, there’s no silver bullet for filling these gaps. Despite the increased investment, numerous roadblocks along the drug development pipeline continue to hamper efforts to bring therapies to patients.

In a recent PharmaVoice poll, industry leaders were divided over where the biggest barrier to rare disease drug development lies — designing and enrolling patients in clinical trials, pushing treatments through regulatory approvals or launching the drug into the market.

Industry leaders weigh in on obstacles in rare disease space

% of the 174 respondents who say the biggest barrier to rare disease drug development is …

Recently, regulatory and research advancements spurred by COVID-19 have helped overcome some of the most glaring obstacles to rare disease drug development.

Clinical trial concerns

With restrictions on in-person clinic visits during the pandemic, pharma companies were forced to think outside the box and utilize decentralized trial approaches and real-world evidence to keep research flowing. Once the infrastructure kinks can be worked out, these new techniques could provide a more effective framework for designing rare disease clinical trials with small patient populations

According to Hassan Kadhim, senior director and head of clinical trial business capabilities at Bristol Myers Squibb, DCTs could allow companies to recruit more patients in trials because “they don’t have to be geographically located with the different sites.”

Similarly, Will Maier, the vice president of rare disease at ICON, told us in August that real-world evidence can be used to supplement the small patient cohorts that participate in rare disease trials.

“In rare disease, where you might not have much in the way of patient numbers or where diagnosis might be quite difficult, we often will just look at medical records to figure out who the patients are,” Maier said. “Sometimes those registries evolve into a post-approval safety study that is used as part of the condition of approval.”

Regulatory requirements

Operation Warp Speed allowed the federal government to accelerate the development of needed vaccines and therapeutics at the onset of the COVID-19 crisis. The same tactics implemented through that initiative could soon be applied to expedite the approval of rare disease medications, Dr. Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research, told attendees of the 2023 Biopharma Congress.

Among the regulatory challenges drug developers face in the rare disease space is collecting enough evidence in small population clinical trials to show efficacy and safety, and then communicating the information quickly with regulators.

In October, the FDA announced a pilot program to help sponsors address these problems and better determine endpoints to assess the efficacy of a drug. Under the Rare Disease Endpoint Advancement (RDEA) Pilot Program, the FDA will accept one proposal for an active pre-IND or IND for a rare disease and will conduct ongoing meetings with the sponsor to evaluate the endpoint. Eventually, findings from the program could be used in an agency guidance document or workshop to improve the industry’s overall understanding of rare disease endpoints.

Additionally, Marks said the FDA hopes soon to “allow the clinical development (of rare disease drugs) to happen in constant communication and sharing of potential results before a submission of an NDA or BLA. That’s the idea — to move things as fast as possible.”

Market viability

Rare disease treatments that make it to launch often come with a hefty price tag. For instance, CSL Behring’s gene therapy Hemgenix for hemophilia B was approved in November and given a $3.5 million price tag.

While the high list prices aren’t likely to change, companies and governments are searching for better financing mechanisms to reduce out-of-pocket costs for these types of medications. Some of the innovative methods include voucher programs, value-based pricing, public-private partnerships, patient assistance programs and targeted grants. In the case of Bluebird Bio’s Zynteglo for the blood disorder beta thalassemia, which entered the market last year with a list price of $2.8 million per treatment, the biotech opted for an outcomes-based pricing approach, meaning it will refund up to 80% of the drug’s cost if it is not effective for the patient.

Still, as patient healthcare costs rise, developing new financing models to improve patient access to rare diseases is likely to become more important for both governments and private entities. 

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