How Brazilian Patients Might Access Sickle Cell Disease Drug Faster
Executive Summary
Masters Speciality Pharma is in the process of registering sickle cell disease drug Siklos in Brazil. The company revealed how it would ensure the product gets to patients sooner rather than later.
Masters Speciality Pharma has signed a licensing deal with the French firm Addmendica to bring its sickle cell anemia treatment, Siklos (hydroxycarbamide), to Brazil. Zulf Masters, the company’s founder and CEO outlined how the company planned to get the drug to patients.
Siklos was authorized in 2007 in the EU where it has orphan status and has since been approved in 11 countries across the world, including the US earlier this year. The product now looks set to become the first registered drug to treat this disease in Brazil, says the company.
Sickle cell disease is a public health priority in Brazil, where it is the most prevalent hereditary disease. Around 2,500 children are born with the disease there every year. As such, Masters expects that the authorities will likely ask the company to submit its registration application through a fast-track procedure that is available for treatments for rare diseases. This means that registration could take around six months from the date of submission, rather than the lengthier standard process, which can take between 360 and 480 days, said Masters.
Moreover, Siklos could reach patients even sooner as the government is considering buying the drug while the registration process is ongoing, in line with local regulations.
Pricing and Reimbursement
Meanwhile, talks with Cmed, the body that regulates pharmaceutical pricing are under way. Cmed applies a “rigorous” pricing formula based on a basket of prices paid in markets around the world, according to Masters. He believes that the price agreed in Brazil will not be higher than the lowest price paid in Europe, though he declined to say what that was.
Masters also confirmed that talks have also recently begun with Conitec, Brazil’s health technology appraisal (HTA) body. An HTA evaluation is a prerequisite for any product that will be funded through the national health system.
Strained healthcare budgets are one of the challenges for achieving market access in Brazil. As a general response to the problem, Masters said the company puts a strong value proposition together. “We try and offer a solution and we try and keep pricing low. We do not overprice things, we see if we can help the government try and cover as many patients as possible with their budgets.”
Once Siklos is approved in Brazil, Masters expects that the drug will be made available to patients at specialist centres, mainly in the states of Rio de Janeiro, Minhas Gerais and Bahia, as well as locations throughout the north and north east of the county, where the disease is most prevalent.
To access the drug, sickle cell anemia patients will have to attend specialist pediatric or hematology clinics, where Masters will train health care professionals to administer it. Siklos is administered according to how much a patient weighs, which means that dosing is more accurate. “This is a factor critical to successful treatment given the narrow therapeutic window that exists between safe and effective dosing, and disease control,” the company said in a statement.
Masters also plans to hold discussions with key opinion leaders to educate and raise awareness of the disease. “Specialists in the treatment of complex blood disorders, will of course be an important part of our strategy, however, we will also be engaging with the wider group of healthcare professionals involved in the treatment of patients with SCD, such as nurse practitioners, pharmacists and procurement managers, ” he said. Key KOLs also include patient organizations. “We also believe it’s important to recognise the role played by the various patient advocacy groups in Brazil and intend to work closely with them to help further patient education and access to treatment,” said Masters.