Rare Disease Blogs

U.S. Senator Kay Hagan (NC) has introduced proposed legislation that NORD believes could help rare disease patients by encouraging the development of safe, effective treatments. It is known as the Transforming the Regulatory Environment to Accelerate Access to Treatments or TREAT Act.

In introducing this bill, Sen. Hagan said her goal was to empower the Food and Drug Administration (FDA) to ensure a clear and effective pathway that will encourage innovative products to benefit patients and improve public health.

In her statement introducing the legislative proposal, Sen. Hagan mentions a study that NORD conducted earlier this year cataloguing FDA flexibility in the review of 135 non-oncologic orphan drugs approved since 1983. That study showed that in two-thirds of the cases, flexibility was employed in the review process, either based on specific policies adopted over the years or on a case-by-case basis.

After releasing this study at its annual conference in October, NORD commended FDA for demonstrating flexibility in the review of products for small patient populations. But NORD went on to encourage FDA to establish written documentation to ensure that the flexibility is applied in ways that are consistent and transparent.

Specific features of the TREAT Act that NORD feels are important are:

• It enhances and codifies the accelerated approval process.
• It addresses concerns of the rare disease community related to conflict of interest provisions regarding participating on advisory committees.
• It provides greater clarity, consistency, and transparency in the review process.
• And it encourages innovation and the adoption of modern scientific tools in regulatory science.

At the NORD/DIA Conference in October, National Institutes of Health Director Francis Collins, MD, said the science of rare diseases is advancing faster than at any other time in history. The global body of knowledge and understanding related to rare diseases is expanding at a very impressive rate.

NORD believes the TREAT Act has the potential to provide FDA with the authorities and tools needed to promote a similar level of innovation and advances in regulatory science and the review process. It would promote the use of modern scientific tools and methodologies, while maintaining the necessary controls related to safety and efficacy.

And if the goal of bringing innovative, safe, effective new therapies to market is achieved, the ultimate winners will be patients, their families, and patient organizations.