The Perinatal Stem Cell Society and its President, Kyle Cetrulo believes that it will only take three changes to the Right to Try Law to revolutionize access to stem cell therapy and advanced regenerative medicine technologies overnight while maintaining safety and oversight.
On May 22, 2018, President Donald Trump signed the Right to Try Act into federal law enabling terminally ill patients to access drugs that are currently being investigated in FDA approved clinical trials. The Right to Try Bill was an attempt to provide life-saving therapeutic approaches for terminally ill patients. However, the Right to Try Act in its original form has major flaws that severely limits patient access. Although the Right to Try Bill has been law for 7 years only 16 products have been approved for use and likely only a few hundred patients have benefited. Due to the limitations in the original legislation, new versions of the Right to Try Act are being promoted in Nevada and Montana. However, these new bills also fail to fully address the problems of the original bill and are also too limited in scope.
Perinatal Society Proposed Change #1
Eligibility: Any individual shall be eligible to access an investigational treatment under this Act, regardless of their current health status, medical diagnosis, or condition.
Current eligibility of each bill:
Federal Right to Try Act (S. 204):
- Patient must have a terminal illness.
- Considered all FDA-approved treatments.
Montana Right to Try Bill (SB 422):
- Patient must have a terminal illness.
- Considered all FDA-approved treatments.
Nevada Right to Try (GoldWater Institute )
- Patient must have a life-threatening or severely debilitating illness (per 21 CFR 312.81).
- Considered all FDA-approved treatments.
The first needed change is to extend access to any American citizen, not just the terminally ill or those who have exhausted other modalities, affirming the right of every citizen to take their health into their own hands by removing eligibility limitations.
Perinatal Society Proposed Change #2
Manufacturers Right to Profit from Sale of the Drug:
Compensation: Allow companies to negotiate directly with the patient and to sell the drug at fair market value with no limit on maximizing profit.
Current Version of Manufacturers Compensation:
Federal Right to Try Act (S. 204):
- Manufacturers may provide treatments with or without *compensation but are not required to do so.
Montana Right to Try Bill (SB 422):
- Manufacturers may provide treatments with or without *compensation but are not required to do so.
Nevada Goldwater Institute
- Right to Try for Individualized Treatments Act: Manufacturers operating within an eligible facility (under FWA) may provide treatments with or without *compensation but are not required to do so.
*Compensation is limited to the cost of goods.
The current Right to Try Law and proposed Bills provide zero incentive for companies to engage with patients and is the main limitation in all three bills. The current reimbursement for the company only covers the cost of goods for the drug. This excludes hard costs and the years of investment in research and development, the cost to operate a laboratory, salaries for the skilled scientists, salaries for the administration and executives, and all of the overall operations that go into actually providing the drug. The cost of goods is a tiny fraction of the actual costs to manufacture the drug.
The goal is to create a marketplace where companies can negotiate and sell the drugs at fair market value and for maximum profit. This small step will revolutionize healthcare in the United States by allowing companies to raise funds for Phase I safety trials and to sell therapeutics profitably during the Phase II and III clinical trial periods.
If the only barrier to get to the marketplace is a Phase I clinical trial there will be 1000s of competing companies entering the market and prices will match the supply and demand of the market driving costs way down and making stem cell therapy affordable.
Perinatal Proposed Change #3:
Company's Clinical Trial:
At the companies discretion, the Right to Try patient can be included in the Phase II and Phase III clinical data set.
Allow companies to fund the cost of the Phase II and Phase III clinical trials through sales of the drug through the Right to Try Law patients. This will enable the company to be profitable during the clinical development stages of the drug development. This will encourage the company to file additional Phase I Safety trials with their drug and include more indications that patient seeking the Right to Try will be eligible for and have access to.
Perinatal Proposed Change #4:
Clinical Trial Process Fees:
Remove the $1,000,000 (Million Dollar) Fee imposed by the FDA to launch a Phase I Clinical Trial.
Although not directly related to the Right to Try Law removing the $1,000,000 fee required to launch a clinical trial will drastically lower the cost for companies to conduct the Phase I Safety trial. This will expand the pool of eligible products for patients seeking any therapeutic to improve their health as more companies will be able to raise the funding needed to conduct the Phase I Clinical Trial for their product.
On May 22, 2018, President Donald Trump signed the Right to Try Act into federal law enabling terminally ill patients to access drugs that are currently being investigated in FDA approved clinical trials. The Right to Try Bill was an attempt to provide life-saving therapeutic approaches for terminally ill patients. However, the Right to Try Act in its original form has major flaws that severely limits patient access. Although the Right to Try Bill has been law for 7 years only 16 products have been approved for use and likely only a few hundred patients have benefited. Due to the limitations in the original legislation, new versions of the Right to Try Act are being promoted in Nevada and Montana. However, these new bills also fail to fully address the problems of the original bill and are also too limited in scope.
Perinatal Society Proposed Change #1
Eligibility: Any individual shall be eligible to access an investigational treatment under this Act, regardless of their current health status, medical diagnosis, or condition.
Current eligibility of each bill:
Federal Right to Try Act (S. 204):
- Patient must have a terminal illness.
- Considered all FDA-approved treatments.
Montana Right to Try Bill (SB 422):
- Patient must have a terminal illness.
- Considered all FDA-approved treatments.
Nevada Right to Try (GoldWater Institute )
- Patient must have a life-threatening or severely debilitating illness (per 21 CFR 312.81).
- Considered all FDA-approved treatments.
The first needed change is to extend access to any American citizen, not just the terminally ill or those who have exhausted other modalities, affirming the right of every citizen to take their health into their own hands by removing eligibility limitations.
Perinatal Society Proposed Change #2
Manufacturers Right to Profit from Sale of the Drug:
Compensation: Allow companies to negotiate directly with the patient and to sell the drug at fair market value with no limit on maximizing profit.
Current Version of Manufacturers Compensation:
Federal Right to Try Act (S. 204):
- Manufacturers may provide treatments with or without *compensation but are not required to do so.
Montana Right to Try Bill (SB 422):
- Manufacturers may provide treatments with or without *compensation but are not required to do so.
Nevada Goldwater Institute
- Right to Try for Individualized Treatments Act: Manufacturers operating within an eligible facility (under FWA) may provide treatments with or without *compensation but are not required to do so.
*Compensation is limited to the cost of goods.
The current Right to Try Law and proposed Bills provide zero incentive for companies to engage with patients and is the main limitation in all three bills. The current reimbursement for the company only covers the cost of goods for the drug. This excludes hard costs and the years of investment in research and development, the cost to operate a laboratory, salaries for the skilled scientists, salaries for the administration and executives, and all of the overall operations that go into actually providing the drug. The cost of goods is a tiny fraction of the actual costs to manufacture the drug.
The goal is to create a marketplace where companies can negotiate and sell the drugs at fair market value and for maximum profit. This small step will revolutionize healthcare in the United States by allowing companies to raise funds for Phase I safety trials and to sell therapeutics profitably during the Phase II and III clinical trial periods.
If the only barrier to get to the marketplace is a Phase I clinical trial there will be 1000s of competing companies entering the market and prices will match the supply and demand of the market driving costs way down and making stem cell therapy affordable.
Perinatal Proposed Change #3:
Company's Clinical Trial:
At the companies discretion, the Right to Try patient can be included in the Phase II and Phase III clinical data set.
Allow companies to fund the cost of the Phase II and Phase III clinical trials through sales of the drug through the Right to Try Law patients. This will enable the company to be profitable during the clinical development stages of the drug development. This will encourage the company to file additional Phase I Safety trials with their drug and include more indications that patient seeking the Right to Try will be eligible for and have access to.
Perinatal Proposed Change #4:
Clinical Trial Process Fees:
Remove the $1,000,000 (Million Dollar) Fee imposed by the FDA to launch a Phase I Clinical Trial.
Although not directly related to the Right to Try Law removing the $1,000,000 fee required to launch a clinical trial will drastically lower the cost for companies to conduct the Phase I Safety trial. This will expand the pool of eligible products for patients seeking any therapeutic to improve their health as more companies will be able to raise the funding needed to conduct the Phase I Clinical Trial for their product.
