Our Policy Work

Advancing a Middle Ground Pathway
For Cell Therapy in the United States

What Problem are we Trying to Solve?

Cell therapy—which involves the use of human cells to restore healthy function in the human body—represents one of the most promising areas for the next generation of groundbreaking treatments.

Cell therapies show enormous promise in the areas of cardiology, neurology, oncology, and ophthalmology. Thousands of clinical trials are now underway that are focused on cancer, heart disease, urologic diseases, kidney disease, trauma-related burns and wounds, and diabetes. A number of trials are also underway to address diseases for which there is no cure, such as Alzheimer’s, MS, and Parkinson’s disease.

Despite the benefits of these treatments, cell therapies are generally not accessible to patients in the United States due to a regulatory approach that has not kept up with scientific advancements.

Increasingly, patients are forced to go to other countries to access treatments. And U.S. companies are also increasingly moving to and making investments in other parts of the world, including Europe and Japan, who have modernized their regulatory frameworks to bring safe and effective treatments to patients.

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Today’s Broken Regulatory Approach for Cell Therapy

Today, there are only two pathways to bring these life-saving therapies to patients in the United States.
Under current law, a very narrowly defined set of HCT/Ps—regulated under section 361 of the Public Health Service Act—can be offered to patients with no pre-market review by clinics that follow requirements of 1271.
All other HCT/Ps—those regulated under section 351 of the Public Health Service Act—are treated like drugs—even if they involve the use of a patient’s own cells. They require a full Biologics Licensing Application (BLA) and take up to $1 billion and 10 to 12 years before they can be made available to patients.

As a result, since 2001 when the regulatory structure for HCT/Ps was created:
Only a handful of cell therapies have been approved by the FDA, using an approach traditionally used for chemical drugs, requiring more than 10 years and $1 billion; and
There is a proliferation of more than 500 clinics offering cell therapies, as practice of medicine, some of which may require greater regulatory review and approval.

The regulatory system for cell therapies is clearly broken, and change is needed.

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What’s the Solution?

Informed by research, guidance from an expert panel, and discussions with multiple public and private sector organizations across health care, the Bipartisan Policy Center (BPC) and the Regenerative Medicine Foundation recommend that a “middle ground” pathway be created for cell therapies.

To create such a middle ground pathway for cell therapy, the Food and Drug Administration (FDA) should be given the flexibility to use “expedited programs” that already exist under current law, for cell therapies. FDA uses it discretion when deciding whether to apply expedited programs to drugs going through the approval process. In fact, a majority of drugs approved in 2014, 2015, and 2016 to date, were approved under these expedited programs.

FDA’s Guidance, Expedited Programs for Serious Conditions – Drugs and Biologics, provides a summary of its expedited programs, which are intended to facilitate and expedite the review of new drugs to address unmet medical need in the treatment of serious conditions.

Again, it is important to note that the use of expedited programs is at the sole discretion of the FDA. Giving FDA the flexibility to use expedited programs for cell therapies does not force FDA to do so. Also, under the law, expedited programs do not dictate the number of phases of clinical trials that a drug must go through—again, this is at the sole discretion of the FDA.

Regarding the definition of a serious condition, the Guidance states the following, “whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that if left untreated, will progress from a less severe condition to a more serious one.”

Because many regenerative cell therapy treatments hold the promise of reducing the need for artificial knees or hips by restoring cartilage, etc., which could be defined as “available therapies” to address a medical need, clarifications may be needed to address the unique aspects of cell therapies.

In March 2016, both Democrats and Republicans introduced the Reliable and Effective Growth for Regenerative Health Options (REGROW) Act, in both the House and the Senate, essentially creating a middle ground pathway in the form of a “conditional approval program”, similar to laws recently passed in Japan and Europe. This middle ground pathway was also recommended in BPC’s December 2015 report, Advancing Regenerative Cellular Therapy: Medical Innovation for Healthier Americans.

In April 2016, the Senate Health, Education, Labor, and Pensions (HELP) Committee unanimously approved bipartisan legislation (S. 2700) laying the groundwork for a modernized regulatory framework for cell therapy, by calling for the establishment of standards to support development, evaluation, and review, and provide greater regulatory clarity and predictability.

Providing FDA with the authority to utilize an existing FDA expedited program (vs. creating a new conditional approval program) to support a middle ground pathway, informed by such standards, provides a reasonable, stepwise approach toward improving the regulatory framework for cell and other advanced therapies. There is growing support among members of Congress to advance this compromise approach.

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Next Steps

Members of the House and the Senate are currently considering the advancement of a middle ground pathway, by giving FDA flexibility and the authority to apply—at the FDA’s sole discretion—existing expedited programs, such as the accelerated approval program, to cell therapies.

This incremental step represents a compromise approach that preserves the gold standard for safety and efficacy and will enable the U.S. to regain its leadership on these innovative therapies that show great promise for those with Alzheimer’s disease, cancer, heart disease, stroke, trauma-related wounds and burns, and many other debilitating diseases for which there is no cure.

Hundreds of organizations representing patients, clinicians, researchers, and industry are expressing their support for the middle ground pathway by reaching out to members of the House Energy and Commerce Committee, the Senate HELP Committee, the House Leadership, and the Senate Leadership. They are also expressing their support by signing on to a support letter being organized by the Regenerative Medicine Foundation.

The Regenerative Medicine Foundation invites you to join with other members of the patient, clinical, research, and industry communities to express your support today. Contact us to demonstrate your leadership and show your support.

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A Long Track Record of Policy Work

In the absence of a supportive legal and regulatory environment,
the promises of cell therapies and regenerative medicine will be delayed, thereby prolonging the anguished wait of those in need of lifesaving treatments and cures.

Regenerative Medicine Foundation has a track record fostering a supportive societal framework for advancing scientific research and accelerating regenerative medicine.

Previous Work (highlights)

In 2004-2005 Regenerative Medicine Foundation (at the time called Genetics Policy Institute) built and led a global coalition that defeated a proposed United Nations treaty that would have banned stem cell research worldwide. The grassroots coalition attracted many notable patient advocates, including Christopher Reeve.

2010-2012  Our organization submitted amicus curiae (“friend of the court”) briefs in the United States District Court for the District of Columbia and the Court of Appeals, supporting federal funding for embryonic stem cell research in Sherley v. Sebelius, a case challenging President Obama’s Executive Order and National Institutes of Health (NIH) guidelines. (Read pdf: GPI brief, GPI and CAMR brief)

Today we continue this essential work in partnership with Bipartisan Policy Center and other respected, nonpartisan organizations.

If you are interested in more details, or would like to discuss ways to foster a supportive legal and regulatory environment for regenerative medicine, please contact us.