Regulations enable approvals and opportunities in rare disease drug development

By Maggie Lynch

- Last updated on GMT

(Image: Getty/BCFC)
(Image: Getty/BCFC)

Related tags Fda Regulations Rare disease Drug development

Regulatory actions by the FDA have enabled rare disease drug development to become a compelling space with orphan drug designations and approvals trending upward, says life sciences industry attorney.

The US Food and Drug Administration (FDA) has taken steps to increase patient participation in rare disease clinical trials, though recruitment remains a considerable challenge.

The agency published a draft guidance in June 2019​ suggesting the removal of certain logistical barriers to increase participation. This included suggestions to use adaptive clinical trial designs and mobile technology tools to supplement clinic visits.

In 2018, the agency also issued a Memorandum of Understanding​ (MOU) defining the framework of a collaboration between the US Department of Health and Human Services, the FDA, and the National Organization for Rare Disorders (NORD).

This MOU saw the FDA and NORD agree to collaborate on promoting scientific progress in innovation, patient education, drug safety communications, and risk evaluation and mitigation strategies.

According to Khelin Aiken, counsel in the life sciences and FDA practice group at McDermott Will & Emery, opportunities in rare disease research are increasing and collaborative work to progress innovation is present, but small patient populations remain a challenge to study recruitment.

Aiken explained further, “With respect to rare disease clinical trials, the biggest impact on patient recruitment is the challenge of finding subjects that are appropriate for inclusion because of the limited number of people that are suffering from the disease, thus making the pool of potential participants smaller.”

The FDA’s 2019 draft guidance to improve patient recruitment suggests that engagement with patient advocacy groups, particularly rare disease patient advocacy groups, can improve the patient-centric communications needed for clinical trials.

“Requests for orphan drug designation and FDA approval of products indicated for rare diseases are trending upward,” ​she said. Orphan Drug Designations are granted to drug products​ developed to treat a rare disease and provide various development incentives.

The designation does not alter standard regulatory requirements or the process for obtaining marketing approval, and safety and effectiveness of a drug must still be established through standard well-controlled studies.

Aiken noted that there is a “huge opportunity in the pediatric space,”​ as “the potential to receive a priority review voucher is very compelling.”

Relating to rare pediatric diseases, the Advancing Hope Act​ (AHA) of 2016 that amended the Federal Food, Drug, and Cosmetic Act to revise the priority review voucher program for rare pediatric disease therapies.

With the AHA sponsors intending to request a voucher for a rare pediatric disease therapy must notify the FDA of the intent upon submission. Additionally, vouchers may not be issues for a rare disease therapy if one was granted to the medication under another program.

“The agency has committed significant efforts to create opportunities for product development, particularly with the AHA of 2016,” ​she told us.

Aiken further explained, “Pediatric orphan drug designations have historically been under-explored. But, the opportunity for exclusivity and drug pricing flexibility coupled with the agency’s enthusiasm to ensure innovation and access is a collision of commonality that is resulting in an increased designation demand and approval.”

Vouchers can be applied to any drug product application and can be used by any applicant, according to Aiken. She said, “Priority review vouchers have been sold to the highest bidder for millions of dollars. The ability to buy and sell PRVs provides another layer of business strategy that manufacturers should consider.”

Advancing adaptive techniques

The 21st​ Century Cures Act​, enacted in December 2016, also takes aim at advancing rare disease treatments, encouraging the use of in silico​ tools in clinical trials to improve the drug development process.

In 2017, the FDA released a draft guidance outlining the ways in which drug development for rare pediatric diseases can be expediated​. In collaboration with the European Medicines Agency (EMA), the FDA developed the guidance from patient and industry feedback.

Using Gaucher disease as a model, the guidance​ describes the use of statistical models and simulations that can eliminate the need for certain clinical studies, as well as the development of multi-arm, multi-sponsor clinical trials, which can reduce the total number of patients needed.

In 2018 the FDA released an additional draft guidance to provide clarity on the use of adaptive trials and approaches. “Adaptive Designs for Clinical Trials of Drugs and Biologics​” advised industry stakeholders on the specific information the FDA needs to evaluate results from clinical trials with adaptive designs.

Aiken added, “I think there is an opportunity for technology and science to build on observed synergies. Apps that identify the right patients or health communities for clinical trials are an area that shows promise.”

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