As snow begins to fall around Clinuvel’s European office, the team in Australia is preparing for a long hot summer. The seasons are at the forefront of our minds at Clinuvel, since our lead drug SCENESSE® appears to have a dramatic impact on the ability of patients to expose themselves to sun. We try to test the drug under the most extreme conditions, meaning trials must be conducted in spring and summer. As the seasons change, we begin to see more requests and enquiries from the southern hemisphere, in particular from patients with erythropoietic protoporphyria (EPP), seeking access to the drug outside of formal trial programs.
We’ve made it clear publicly that we support the administration of SCENESSE® for these severely affected patients beyond clinical trials. EPP, an absolute intolerance to light, forces many patients to lead virtually nocturnal lives for much of the year. These individuals can only allow the briefest exposure of their skin to sunlight for the risk of suffering debilitating and painful phototoxic reactions; often lasting for days. Clinical results to date suggest SCENESSE® is well tolerated (safe) in EPP patients and effective in reducing the number and severity of phototoxic reactions caused by the disease. Anecdotally, we’ve also been aware for some time that the drug does have the potential to change the quality of life of patients. Despite all of this, continuing to supply a product free of charge represents a conundrum.
Two factors dominate our decisions on this matter: the response from regulatory agencies and the cost of freely supplying the drug. Drugs which are yet to be approved for marketing by pharmaceutical regulators can, in some instances, be supplied by companies under what is known as a ‘compassionate use’ protocol (also known as a ‘special access scheme’ protocol in some regions); particularly in the case of severe diseases for which no other treatment currently exists. These protocols allow for the ongoing treatment of a patient beyond a formal study or, in some instances, where a patient was ineligible for study participation.
The process of applying for compassionate use varies, but generally involves a formal request by a physician to a drug company on behalf of a patient, expressing a need for treatment. The company or physician (country dependant) in turn approaches the relevant regulator and applies for ongoing treatment of the patient under a specific compassionate use protocol. Physicians, patients and companies must adhere to the existing national framework; rarely are two countries’ frameworks for compassionate use identical, but there are similarities. Generally, limits are set on the treatment period and the physician and company must agree to continued monitoring of the patient’s treatment progress and safety with regular clinical visits.
Clinuvel believes there is much to be gained from these programs and that they should be viewed as an investment. While the patient ideally benefits from the ongoing treatment, companies also benefit from having extended data on the safety profile and use of the drug available to them outside of a formal study protocol. It also indicates that patients believe there is benefit to continuing use of the drug, despite the potential inconvenience of frequent clinical visits; this is an encouraging indication, to say the least.
From the most recent Phase III study in EPP (CUV017) a great number of patients eligible for compassionate use across the study sites have maintained their treatment under a compassionate use protocol. Applications have been made for patients from other sites to the relevant regulators, to allow for treatment with SCENESSE® in the coming spring and summer. In Italy, the patients who had completed their compassionate use protocol took their regulatory request for ongoing treatment a step further and successfully applied for a world first reimbursement and supply of SCENESSE®, an unapproved product, through the national health system – a remarkable event.
As the first patients from our confirmatory EPP program in Europe and the US (Phase III and II trials respectively) complete their treatment under the formal study protocols, we are in discussions with national agencies – including, for the first time, the FDA – for ongoing treatment. While no decisions have been finalised, it is my hope that we will be able to continue these compassionate use programs for as many patients as is possible until such a time that we are able to completely satisfy the regulators of the need for treatment in this severe disease.
Image reference
‘Four Seasons – Longbridge Road’ uploaded to Flickr.com by joiseyshowaa on the 28 October 2008, <http://www.flickr.com/photos/joiseyshowaa/2775011897/>
