HME Business

What are the market needs and technical challenges?

For patients who must manage chronic diseases, such as hemophilia, multiple sclerosis, rheumatoid arthritis and diabetes, medication issues can present significant challenges regarding safety, ease of administration, cost, compliance and other factors. Fortunately, continual advances and breakthroughs are delivering tremendous improvements.

However, these medications typically require frequent injections or infusions. Depending on the nature of the disease and the patient’s individual condition, those treatments could be weekly, daily, or even multiple times a day. Advances in drug technology, therefore, need to be matched by innovations in systems and devices for administration.

Why do we need reconstitution systems?
Many new drugs are initially marketed in lyophilized form for two reasons: shelf-life and time-to-market. A lyophilized drug maintains its stability and potency over time, extending its shelf-life for prolonged storage. Some drugs marketed in lyophilized form may eventually be available as liquid, but lyophilization provides the fastest route to market for many drugs and is the only option for those not stable in a liquid form.
These drugs — often packaged in powder form in vials — require an additional preparation or reconstitution step prior to administration. Traditional reconstitution requires multiple needles, syringes and diluent/drug containers. This rudimentary process presents several challenges:
•    A Lack of Expertise. In most instances, reconstituted drugs are typically administered at home by patients or caregivers who are not trained health care professionals.
•    Added Risks. Risks of accidental needle stick or exposure to hazardous materials are increased.
•    Compliance Concerns. If the process is complicated, dosing accuracy may suffer. And if the process is difficult, unpleasant, or painful, it can become an impediment to patient compliance.
•    Waste. Pharmaceutical manufacturers often overfill the vial to ensure that there is a sufficient quantity of the reconstituted drug to administer the correct dose. From the patient’s perspective, there’s a risk of mishandling or contamination that can necessitate throwing out very expensive drugs.

Making challenging conditions easier
A number of newer, advanced products on the market can provide non-professionals with safe, convenient and easy-to-use systems for reconstituting and administering injectable drugs. These systems can be provided either as a total packaged solution or as components for specialized use. Such systems usually consist of a needle-free plastic component that joins the drug vial to the diluent container that can be either a prefilled syringe, vial or infusion bag.

Reconstitution devices can be sterile and fully supported by appropriate regulatory filings.

Let’s take a look at a case history. Every hemophiliac knows and lives with a shared reality: Administering expensive medications to control these serious conditions can be a complicated, time-consuming process. For a child, simplicity and safety are of even greater importance.

For 6-year-old Brock, hemophilia is more than a chronic disease. With its need for regular dosages of intravenous medications, hemophilia is also a chronic annoyance. Brock was on high-dose Immune Tolerance Therapy that required a two- or three-vial dose. Each vial reconstitution needed a set of more than 10 different steps just to prepare a dose. According to his mother, Katherine, 42, it was getting to be too great of a burden.

Katherine says that, as Brock grows older, she wants “to help foster a greater sense of independence and control in him.”
“He’s battling a difficult disease and if I could find a way to put him more in control, it would be good for his self-esteem and easier for all of us,” she says. “With his previous medication, we’d have to pop off tops, get a syringe packet, puncture the vials with a double ended needle, transfer the water, dispose of the needle in a sharps container, then open a filter needle to draw up the solution only to have to remove it to administer the product.”

“So we consulted our physician and chose Helixate FS and the determining factor was the much easier system for preparing and administering the doses,” Katherine explains.

Helixate FS, manufactured by Bayer HealthCare for CSL Behring, is an advanced recombinant FVIII (rFVIII) factor product for the treatment of hemophilia A, which uses an easier drug-delivery system.

“Brock liked the Helixate/Mix2Vial system because it was just so much easier for him to figure out. He even taught his 3-year-old sister how to do it,” Katherine said. “It requires just four steps to reconstitute the drug and has a built-in filter.”  (Mix2Vial was designed by Medimop Medical Projects, a West company.)

Katherine and Brock also appreciate the fact that there are no needles involved in the reconstitution process. “This would be a great option for any patient, but it has been particularly well suited for Brock,” she explained. “The simplicity of the process takes out some of the frustrations and that’s a really good thing.”

Patient training will ensure safe use
Reconstitution systems are especially beneficial for products that are used to treat chronic conditions that are administered in a home setting. For the person administering the drug, advanced reconstitution systems can help promote safe and effective drug delivery and compliance with a dosing regimen. Advanced reconstitution systems represent an important leap forward in usability and safety.

However, it’s also undeniable that they introduce change for people who are not health care professionals. Manufacturers must ensure that patients receive complete and clear training on using the new reconstitution systems.