Frequently Asked Questions
The Heartland Institute’s Free to Choose Medicine (FTCM) project is devoted to offering patients with terminal illnesses the opportunity to safely access treatments that have already passed Phase I safety testing by the U.S. Food and Drug Administration (FDA) and at least one Phase II efficacy test. In the 1990s, FDA successfully implemented a similar exception for HIV/AIDS patients during the height of the AIDS epidemic. Fortunately, this resulted in the approval of several drugs that improved the quality and length of life for millions of HIV/AIDS patients across the globe.
Heartland’s FTCM proposal would simply apply the same proven reform for millions of Americans now struggling with debilitating diseases. FDA’s current approval process is far too lengthy (10–12 years) and expensive ($2.9 billion). Patients with life-threatening conditions deserve the opportunity to access safe, promising treatments. FTCM would accomplish this urgent and pressing objective.
- On average, it takes 10–12 years at a cost of $2.9 billion for a new drug to go from the research lab to the market, with much of that time and cost devoted to satisfying U.S. Food and Drug Administration regulatory requirements.
- The excessive time required for new drug approval unnecessarily delays access to promising new drugs and, in some cases, prolongs illnesses and results in patient deaths.
- The costs and time required to win approval for a new drug eliminate many new drug candidates from further development, which results in less competition, less innovation, and higher prices.
- FDA’s regulatory monopoly mandates hugely expensive, often unnecessary clinical testing and data analysis that stifles competition and inflates drug costs.
- After several years of product development in private research labs, FDA requires a drug to enter Phase I testing for basic safety.
- Safe products then must pass multiple Phase II tests to determine general efficacy and Phase III tests to refine that information.
- These latter phases can stretch out for up to a decade. Upon completing all phases, a producer may file a “New Drug Application,” which can take as many as two years to approve. This all adds up to 10–15 years.
- Yes! FDA is overly cautious, but this caution does not make us safer. We all want safe medications, but we also want them in a timely manner. FDA has little incentive to approve drugs quickly.
- If FDA approves a treatment that does not perform as effectively as promised, it could suffer poor public relations coverage and possible congressional inquiries.
- However, FDA receives little criticism or penalties for requiring years of dubious tests that results in years of patient suffering and unnecessary deaths.
- No! FDA’s failings were made clear during the AIDS epidemic of the 1980s. Individuals suffering from that ailment rightly argued that with only a few years or even months to live, they could not wait for very promising drugs to pass seemingly endless rounds of efficacy tests. They were facing death.
- Although some FDA reforms were made to allow expedited and compassionate use, FDA’s problems have continued and arguably worsened.
- Smaller companies cannot afford a decade or more of costs before their products finally reach the market, allowing them to recover their investments. FDA’s draconian system prices them out of the market.
- Often, pharmaceutical giants gobble up smaller companies. While this might save some promising research from being shelved, a large, bureaucratic company often can dampen the efforts of visionary, innovative researchers.
- Free to Choose Medicine is an innovative plan to reform FDA’s current drug approval process. FTCM’s goal is to create a system that provides better drugs, sooner, at lower cost.
- FTCM creates a parallel and more dynamic track for testing and approval of new drugs. After a new drug passes FDA Phase I safety tests and at least one round of Phase II efficacy testing, a drug manufacturer can choose to proceed on the FTCM parallel track.
- On the FTCM track, manufacturers make their drugs available to physicians and their patients. While using the drug, the physician enters patient treatment information, anonymously, into a newly created Tradeoff Evaluation Drug Database (TEDD).
- The information and data in TEDD would be accessible online to other physicians so that they can determine whether to advise the drug for their patients.
- The information and data in TEDD would also be available to other researchers and pharmaceutical companies to better inform their work and allow them to bring additional new drugs to market faster.
- Under FTCM, patients, advised by their doctors, can make an informed decision as to the use of a not-yet-approved drug that has been allowed on the FTCM track. The characteristics and medical history of the patients as well as the results of the medication are entered into TEDD, with patient privacy protected.
- Researchers would also have access to this data, which would spur more drug innovation
- Sure. In late 2000, 21-year-old Abigail Burroughs was diagnosed with neck cancer. By March 2001, traditional treatments had failed her. Her physicians then recommended a safe drug stuck in FDA-mandated efficacy tests. Abigail and her family did everything they could to access the promising drug, even making appeals on local TV shows and pulling political strings. Sadly, nothing worked. Abigail died in June 2001. The drug was later approved. The tragic case of Abigail Burroughs inspired the development of FTCM by Bartley Madden.
- Right to Try (RTT) laws allow terminally ill patients to access new drugs that have passed Phase I FDA safety testing but about which no determination of efficacy or effectiveness in treating an illness has been made.
- FTCM allows early access to a new drug only after it has completed one or more Phase II round of FDA efficacy testing.
- RTT laws do not require physicians to report patient treatment data while on the new drug. On the other hand, FTCM requires physicians to report treatment data for their patients on a new drug, thereby offering physicians and patients the data needed to make an informed choice on whether to use a new drug.
- FTCM allows market pricing of not-yet-approved drugs. If a new drug generates compelling treatment results, it could receive a new FDA Observational Approval, which would expedite insurance reimbursement.
- No, Free to Choose Medicine offers a second track for drug development, testing, and approval.
- Drug companies can choose to develop, test, and have new drugs approved on FDA’s standard drug approval track or the FTCM track.
- No! To get on the FTCM track, a product must pass Phase I safety tests and at least one initial efficacy test to show the product’s promise for safely treating patients.
- The new frontier of medicine includes bio-hacking and engineering cells, blood, or other so-called “biologics” to treat human ailments.
- The best ways to test the efficacy of these cutting-edge treatments might not be the same process FDA uses for testing traditional chemical-based medications.
- New ways of testing might be necessary, and FTCM could offer viable alternatives.
- No! Japan recently adopted this approach for regenerative treatments.
- About 26.5 percent of Japan’s population is older than 65, the largest proportion of elderly people in any country’s population. Life expectancy in Japan is 83.7, one of the world’s highest.
- Japan implemented FTCM to protect its most vulnerable citizens by allowing them to access drugs that could possibly save and prolong their lives.
Conclusion
Like all industries, when the U.S. health care system has improved, it’s been because of competition and innovations that flow from a free market, not because of government regulation. Free to Choose Medicine offers choice and competition in the marketplace and moves the dial in favor of patient freedom.
The antiquated FDA drug approval process is holding back innovation the private sector is demanding. The government ought to act like a bridge to innovation, not a barrier. FTCM would be an important and lifesaving move in that direction.