We are ambitious and want to try and change significantly the current research culture. We want to make better drugs and new treatment methods available to patients faster and at lower costs. This we do by assisting clinical medical investigators in the design and execution of short lasting phase 2 studies.
Phase2Therapy (P2T) founded in 2013 is an open source organization. We enhance knowledge transfer by making anominized results of P2T studies available to others on login and password while the study is still on going. P2T harbors knowledge (intellectual property) of scientists based in The Netherlands, The United States, Great Britain and Lebanon. This diversity in languages spoken, talents, educational backgrounds and cultures is essential to our success.
We are a not-for-profit organization and obtain our operating costs by way of donations, grants and crowd sourcing.
We enhance knowledge transfer by making anominized results of P2T studies available to others on login and password while the study is still on going.
Clinical-medical investigators can use new P2T drugs or patented P2T devices for production costs plus a 30% surcharge after accreditation. The surcharge is necessary to help cover P2T’s operating costs.
P2T has instituted an accreditation procedure to secure the quality and safety of research performed with P2T’s drugs and devices. Investigators who successfully complete the accreditation will be able to use P2T drugs and devices in their research.
New investigators joining P2T will contribute scientifically to the whole group, because the newcomers are required to also adopt the open source method and share their results with other accredited users of P2T intellectual property.
P2T selects new drugs/devices after phase 0 and 1 studies indicate high efficacy and a low incidence of side effects.
We formulate a simple experimental question, the so-called ‘null hypothesis’, which can be confirmed or rejected objectively in a short, cheap clinical study. Only drugs/devices, which improve the medical condition of at least 1 out of 3 patients studied and do not cause any or only a minimal amount of serious adverse effects, will continue in further tests and developments.
Most P2T studies/projects will be limited to 30 patients. New drugs effective in at least 1 out of 3 patients do not have to be compared in a double blind randomization to 30 patients receiving a placebo. This streamlined approach guarantees that patients can benefit faster from the new drug. Moreover the new drug will be considerably cheaper than drugs developed by the pharmaceutical industry.
In some P2T projects randomization will be used. Not double blind randomization, but the so-called staggered wedge method: at the end of the study all patients will have received the new drug/device, but the sequence in which the two groups of patients get the new drug/device differs.
P2T raises the bar for phase 2 studies: 33% efficacy, instead of the cut-off efficacy point for the pharmaceutical industry: 15%. The new drugs or devices studied by P2T that fail to make the grade are dropped, but results are published in the open medical literature.
We admit P2T intentions are to change the development of new drugs/devices drastically, but doing so we will always adhere to the existing rules and regulations for safe and ethical drug development.
The Pharmaceutical Industry has learned how difficult it is to identify and develop new drugs. Before the industry starts a phase 2 study it has already incurred more than one third of the costs it takes to complete the development of a given new drug. The costs of a phase 2 study add another 15% to the total costs. The P2T approach diminishes costs and development time of drugs drastically.
A placebo, “I will please you” in Latin, is a drug that is similar to the new drug under study in color and shape, but is inert. Neither the patient nor his physician knows whether the patient is in the placebo arm or the proper drug arm (‘double blind’).
Ten to fifteen percent of the patients receiving a placebo report they are getting benefits from the drug/placebo, while another ten to fifteen percent report ‘significant’ side effects, a so called ‘nocebo’ effect. “I shall hurt you” in Latin. Patients do not mind getting side effects, because they think the side effects indicate they are on the non-placebo study arm.
P2T opines placebo randomized trials take too long. They have to be conducted in several different institutions and the end result of the study is not an important step forward: The benefits are limited and experienced by less than 1 in 5 patients.
The bottleneck in the development of new cancer treatments is the investigator, not lack of funds or overly restrictive rules and regulations for clinical research. Investigators, worried about their patients with cancer, should get together and develop more effective and cheaper ways of drug development.
Huib Vriesendorp, Chairman P2T