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Understanding

Dopamine neuron (iPSC sourced)

The FDA and

Regenerative Medicine

Therapies

Updated: 09/30/2020

This page is dedicated to provide you with information and an understanding of the Regenerative Medical Therapies and the FDA:

Regarding the research project launched by Summit: autologous dopamine neuron replacement therapy

Please note: Regenerative Medicine is a new field. Please understand that the work being done to develop this therapy is the first of its kind and leads the world in the development of an autologous dopamine neuron replacement therapy. We would like to share with you that the scientific team has been working with FDA consultants and the FDA throughout the research project's development and have established a good working relationship. There have been no FDA precedents or guidelines to follow. The clinical trial process is subject to the discretion of the FDA for your safety.

The FDA has created a "Framework for the Regulation of Regenerative Medicine Products" (05/21/2019) addressing how the agency plans to support and expedite the development of regenerative medicine products. The guidance documents underscore the agency's commitment to help bring new and innovative treatment options to patients with unmet medical needs.

How long does it take for a Regenerative Medical (Stem Cell) Therapy take to receive FDA approval to begin clinical trials?

All studies required to determine safety and efficacy for a stem cell therapy MUST be completed first.

The clinical trial process cannot begin until studies for the autologous dopamine neuron replacement therapy are completed.  

 

For REFERENCE - The typical timeline for a pharmaceutical drug to be publicly accessible:

                                           12+ years for a pharmaceutical drug to receive FDA approval to begin clinical trials 

                                           Average cost of US$350 million. 

                                Once FDA approval is received, the “investigative new drug” then begins three phases of clinical trials:

                                           Phase I - Establishing a drug’s safety and profile (approximately 1 year).

                                           Phase II – Assess a drug’s effectiveness (approximately 2 years).

                                           Phase III – Determining a drug’s effectiveness and identify adverse reactions (about 3 years).                                                        Once Phases I,II, & III are completed

                                           A 100,000+ page application is submitted

                                           2.5+ years additional to processing

                                Only then is final FDA approval given for physicians to prescribe.

However, the FDA is working to meet the unmet needs of people with diseases having no cure. Parkinson’s disease is a disease with unmet medical needs. Although the induced pluripotent cell-based therapy has not received its classification as of this date, the FDA has developed new policies to address the pace at which scientific discoveries in regenerative medicine are being made today.

Aspen Neuroscience is currently working with a newer FDA division called: “Center for Biologics Evaluation and Research (CBER)”.

 

CBER is working with the Regenerative Medicine field to develop new policies to “expedite” Regenerative Medical therapies availability. The following policies and programs have been created in an effort to meet patient’s needs and the challenging pace of new scientific discoveries: 

                            Fast Track Designation: to facilitate the development and expedite review of drugs and

                            biologics intended to treat serious or life-threatening conditions and that demonstrate the

                            potential to address unmet medical needs. 

 

 

                            The 21st Century Cures Act: modifies the FDA Approval process. The development

                            and bring new innovations and advances to patients who need them faster and more

                            efficiently.

 

 

                            RMAT designation: Regenerative Medicine Advanced Therapy (RMAT): a drug is eligible

                            for regenerative medicine advanced therapy (RMAT) designation if:

                                      1. The drug is a regenerative medicine therapy, which is defined as a cell therapy,

                                           therapeutic tissue engineering product, human cell and tissue product, or any

                                           combination product using such therapies or products meeting FDA criteria.

                                       2. The drug is intended to treat, modify, reverse, or cure a serious or life-threatening

                                           disease or condition; and 

                                       3. Preliminary clinical evidence indicates that the drug has the potential to address

                                           unmet medical needs for such disease or condition.

                            Right To Try: This law is a way for patients who have been diagnosed with life-threatening

                            diseases or conditions who have tried all approved treatment options and who are unable to

                            participate in a clinical trial to access certain unapproved treatments.

                            The Right to Try Act permits/allows eligible patients to have access to eligible investigational

                            drugs. An eligible patient is a patient who has:

                                   - Been diagnosed with a life-threatening disease or condition

                                   - Exhausted approved treatment options

                                   - Unable to participate in a clinical trial involving the eligible investigational drug

                                     (this must be certified by a physician who is in good standing with their licensing

                                     organization or board and who will not be compensated directly by the manufacturer

                                     for certifying)

                                   - And has provided, or their legally authorized representative has provided, written

                                     informed consent regarding the eligible investigational drug to the treating physician

 

                           An eligible investigational drug is an investigational drug:

                                   - For which a Phase 1 clinical trial has been completed

                                   - That has not been approved or licensed by the FDA for any use

                                   - For which an application has been filed with the FDA or is under investigation in a

                                     clinical trial that is intended to form the primary basis of a claim of effectiveness in

                                     support of FDA approval and is the subject of an active investigational new drug

                                     application submitted to the FDA.

                                   - Whose active development or production is ongoing, and that has not been

                                     discontinued by the manufacturer or placed on clinical hold by the FDA.

                           If you are interested in Right to Try, you should discuss this pathway with

                           your licensed physician.

                           For more information on Right to Try: 

Compassionate Use or Expanded Access: Extended Use, sometimes call "compassionate use", is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Expanded access may be appropriate when all the following apply:

        - Patient has a serious disease or condition, or whose life is immediately threatened by their 

          disease or condition.

        - There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the

          disease or condition.

        - Patient enrollment in a clinical trial is not possible.

        - Potential patient benefit justifies the potential risks of treatment.

        - Providing the investigational medical product will not interfere with investigational trials that

          could support a medical product’s development or marketing approval for the treatment

          indication.

If you are interested in Expanded Access (Compassionate Use, you should discuss this pathway with 

your licensed physician.

 

For more information on Right to Try: