COMPASSIONATE USE OR EXPANDED ACCESS POLICY
Laboratoris Sanifit S.L. (“Sanifit”) is committed to developing innovative, safe and effective therapies for vascular calcification. The company is advancing therapy for treatment of calciphylaxis as well as cardiovascular calcification for patients with end stage renal disease on hemodialysis. We are conducting clinical trials to assess the safety and efficacy of investigational product, which if proven, will allow us to obtain the regulatory approvals to provide patients with broad access to these medicines.
In general, Sanifit believes that participating in clinical trials is the best way for patient to access medicines prior to regulatory approval because they are conducted in a well-controlled environment. Clinical trials collect the necessary data to evaluate the safety and efficacy of an investigational product and allow for regulatory review and approval of the product. In some extreme circumstances when this is not possible, patients or health care providers are interested in special access to investigational product prior to regulatory approval and outside of clinical trials settings. This type of special access is usually referred to as compassionate use, expanded access, early access, pre-approval access or emergency use.
Compassionate use and expanded access to investigational products must balance several factors such as regulatory considerations, strength of the clinical data, benefit-risk profile, the impact on clinical development program, the phase of development and probability and timing of regulatory approval.
Several factors were considered to determine whether or not to support compassionate use for a given investigational drug:
- Is there substantial scientific evidence to support the safety and efficacy of the investigational product so that it can be provided to patients in an environment that is less controlled than a sponsored clinical trial?
- Will it compromise the clinical development to bring the product to market as quickly as possible?
- Is there adequate supply of the investigational product?
After taking these factors into consideration, Sanifit has determined that compassionate use cannot currently be offered for its investigational product on a fair and sustainable basis. We believe that the best path to potential regulatory approval, and subsequent access to the greatest number of patients, is by executing our current and future clinical trials as efficiently as possible
We will continue to evaluate the possibility of compassionate use and pre-approval access mechanism.
For information on current clinical trials, click on www.clinicaltrials.gov, the Federal Government’s website containing information about Sanifit’s current clinical trials.