Commissioner of Food and Drugs – Food and Drug Administration
Thank you for inviting me to be with you for this important meeting. I’m sorry we can’t be together in person.
I want to express my gratitude to each of you for your energy, focus, and commitment to helping patients with rare diseases, particularly during the COVID-19 pandemic.
I especially want to recognize and thank the patients and their families who live with the challenges of rare diseases every day, and who are the inspiration and moving force behind the critical work being done to find new treatments and cures.
The work of NORD in marshalling this power takes several different forms.
Most obvious is the immediate and long term support you provide for patients through your efforts to find and advance new treatments and cures.
You also play an important role in shining a light on, encouraging, and forging opportunities to advance new and groundbreaking scientific research, in areas where there might otherwise not be attention and action.
Which leads me to a third critical role you play — promoting collaboration.
Collaboration occurs here at this conference, of course, with so many different stakeholders — including researchers, representatives from developers, investors and potential investors, healthcare organizations, government officials, and, most importantly, patients and caregivers — coming together to share information, learn, and support each other in the efforts to develop and bring new and better treatments to patients.
But your ability to bring the strengths of the rare disease community together and to build collaborative partnerships goes beyond this meeting, and is an important means of finding treatments and cures for these diseases.
The challenge of rare diseases has been an important part of my career, and its focus on researching and treating cancer. I’ve had the opportunity to witness and, in some cases, be part of some of the extraordinary developments of products to treat these diseases.
I understand that while we face great challenges, we also have enormous opportunities for the development of products and therapies to treat rare diseases.
That potential is captured in the title of this meeting – “Entering a New Era, Bringing Transformational Therapies to Patients with Rare Diseases During Challenging Times.”
“Challenging times” can be interpreted in many ways. Today, of course, the global pandemic we’re working to overcome is front and center. It affects everything, but it has a special impact on rare disease product development. I’ll speak more about that shortly.
First, I want to note how, even in the face of this unprecedented public health crisis, we’ve nonetheless seen some important developments for rare diseases. And that’s a tribute to your work and focus.
This year, the FDA (through July) had approved 51 orphan indications, covering a broad range of rare diseases. Let me give you a small sampling.
In August, our Center for Drug Evaluation and Research approved risdiplam to treat patients two months of age and older with spinal muscular atrophy, or SMA, a rare and often fatal genetic disease affecting muscle strength and movement. This is the first oral drug approved and third drug approved for SMA.
We also approved a number of drugs to treat different types of cancer, such as Selpercatinib, for the treatment of patients with RET mutant medullary thyroid cancer and RET fusion positive thyroid cancer.
Among the treatments approved by our Center for Biologics Evaluation and Research were SEVENFACT, a product for hemophilia treatment that includes an active ingredient genetically engineered to produce a protein necessary for blood coagulation.
As these approvals, along with our many others this year indicate, I would suggest we are already part of “a new era” of transformational therapies.
This success is due in great part to the collaboration that NORD promotes. It is also an essential aspect of FDA’s work, both internally and externally, especially in rare disease product development.
We accomplish this through communication with sponsors, researchers and others, as well as through a number of our programs and outreach efforts.
For example, the Office of New Drugs launched a new division for Rare Diseases and Medical Genetics. The new division’s mission is to advance treatments for all patients with rare diseases by taking innovations learned on the front lines of drug development for rare, fatal, pediatric diseases like inborn errors of metabolism, and leveraging them to expedite drug development for rare diseases in every division in the office of new drugs.
Our Office of Orphan Products Development continues to be busy, once again experiencing large increases in the number of requests to our orphan drug designation and rare pediatric disease designation programs, continuing a multi-year trend.
And our Orphan Products Grant program, which provides important funding in support of rare disease research, funded 6 new awards in FY 2020 that focus on unmet needs, including for indications such as Stargardt disease, pancreatic cancer, and Graft vs host disease.
A new initiative, called Orphan Grantees Unite, provides a forum for grantees to share their experiences, and challenges in support of common solutions.
Another promising effort, intended to help medical technology innovation meet the unique and complex needs of children and small populations, is an initiative out of our Center for Devices and Radiological Health called SHIP, or System of Hospitals for Innovation in Pediatrics.
The vision of SHIP is for a national ecosystem that will focus on safe innovation by optimally engaging our nation’s pediatric health systems that have the breadth and depth of infrastructure to support safe evaluation of technologies for children. It will be designed to transform traditional business thinking around investing in the pediatric medical device market.
We welcome your input on this initiative, and I hope that you will join us this February for a virtual public meeting and workshop.
In another exciting development, last year CBER awarded a contract to NORD to design and conduct a pilot rare disease natural history study. This will be a source of control data for clinical trials of therapies for a rare disease for situations such when it is not feasible or ethical to enroll and randomize patients to a control arm in a clinical trial. You’ll hear more about this project during the breakout session later today.
I could tell you more, but I want to switch gears now and talk a little bit about the FDA’s response to the pandemic.
As you know, developing a product for a rare disease is challenging in the best of times and circumstances. The circumstances we find ourselves in right now, are far from that.
The COVID-19 pandemic has had far-reaching effects, and patients with rare diseases may be especially vulnerable to the impacts of COVID-19.
It is essential that even as we continue to support the development of safe and effective treatments and vaccines for COVID-19 during this public health emergency, we also advance the FDA’s other public health priorities, including the development of treatments for patients with rare diseases.
I’m happy to report that, in the face of this unprecedented challenge, the FDA’s centers and offices have continued to fulfill the Agency’s mission-critical responsibilities.
For example, with NORD as a partner, we’ve held virtual Patient Listening Sessions, to help ensure that a larger group of patients and caregivers can share their perspectives. Recent sessions have focused on a variety of rare diseases, including Smith-Magenis Syndrome, progressive multifocal leukoencephalopathy, Hunter Syndrome, Von Hippel Lindau, Valosin Containing Protein Disease, and inclusion body myositis.
Earlier this year we participated in a Listening Session with NORD and another with NORD’s rare cancer coalition regarding the impact of COVID-19 on patients with rare cancers.
We are also continuing our work with NORD as an important partner in building the Rare Disease Cures Accelerator, a cooperative scientific initiative for the development of common, standardized platforms that can improve the characterization of rare diseases, incorporate the patient’s perspective through the development of standard core sets of clinical outcome assessments and related endpoints, and build clinical trial readiness in the pre-market space.
In other words, COVID-19 may be placing incredible demands on the Agency, but it hasn’t stopped us from fulfilling our commitment to our many other priorities.
But we’ve also been focused on addressing the challenges of this pandemic.
For example, we are working with medical product companies performing clinical trials to address some of the additional challenges they face with the COVID-19 pandemic. This included issuing a guidance to provide greater clarity to medical product companies about how to proceed with these trials and deal with necessary protocol deviations created by the pandemic.
Our Center for Biologics has worked closely with sponsors of cell and gene therapy products to address challenges to recruitment and trial design caused by the COVID-19 pandemic. Many of these therapies are being developed for patients with rare diseases.
And we’ve also worked to address unexpected increases in clinical study costs in our Orphan Products Grants, and to support the development of virtual or digital health platforms and models.
And, of course, we continue to focus on the development of safe and effective medical countermeasures, from working to ensure that our front-line health care workers have the necessary protective equipment, to providing essential regulatory advice, guidance, and technical assistance to advance the development of tests, therapies, and vaccines.
For each of the decisions we make, it is critical that the American public has complete confidence in the process we use to reach those decisions. Nowhere is that more important than for any decision we might make on whether to approve or authorize a vaccine.
I want to assure you that the FDA will not authorize or approve any COVID-19 vaccine before it has met the Agency’s rigorous standards for safety and effectiveness.
In this effort, and in everything we do, science will guide our decisions.
As we move forward, continuing to find answers and develop strategies and solutions to address the extraordinary challenges of this pandemic, we are also applying the lessons learned to future actions.
For instance, we recently launched the COVID-19 Pandemic Recovery and Preparedness Plan (or PREPP), which focuses on identifying avenues for improvement by coordinating scientific leadership, agency operations, communications, and programming across the agency for all regulated products.
We can emerge from this pandemic only if we work together, learn from each other, and continue to be driven by the facts and data.
Our efforts to find solutions to rare diseases is a long-term priority at FDA. And we look forward to continuing to engage and collaborate with NORD on these issues.
I hope you have a productive meeting, and I thank you for your commitment, and for inviting me to be here with you today.