Pharmacist help for rare diseases

Fümany people with a rare disease and there is no finished product. With the patient-matched formulations, pharmacists make the supply safe

Hand: let the capsules individually with a medicine f&uuml materials;cases

It is a single is by no means destiny. A rare disease, around four million people suffer in Germany. It meets every Twentieth. "However, since there is a total of more than six thousand of such diseases, numbers of patients in each disease is very gering", the Berlin pharmacist Dr. Dennis Stracke, of the care of these Pa&shy says;­patients specializes.

As rare a disease is when less than one out of 2000 people suffers from it. Häcommonly, it is genetically Stoffwechselst&ouml conditional;retention, neurological diseases or congenital immune defects. They are usually chronic and sometimes life-threatening course.

Research is not worth it

"Because of their rarity they are also in Ädoctors hardly bekannt", Stracke says. Therefore, it often takes years until the correct diagnosis is made. But l&auml is still;ngst no Lösung für the problems in sight. Stracke: "Less than ten percent of all rare diseases can be pharmacologically treated."

Even if it für the disease in question is an effective therapy in the majority of Fäcases, no approved finished drug products to the Verfü. "The Pa­patient numbers are often so low that it für the Pharma­industry is not worthwhile, dafüto entwickeln&quot r drugs;, erklärt VizepräPresident of the land pharmacists Association of Baden-­Württemberg, Christopher Gulde. Despite the simplified authorisation procedure, the research and development costs are still too high.

According to the needs of befüfilled capsules

This Versorgungslücke can schlie&szlig the local pharmacy;en. "Für individual data, we make individual recipes: with special combinations of active ingredients as well as doses and dosage forms on the market verfüavailable sind", Gulde, owner of a pharmacy in Stuttgart says. "So, for example, capsules individually with the prescribed bef&uuml active ingredients;cases."

Also Säuglinge and small children köcan rare diseases be affected and benöterm drugs in age-appropriate dosages, and child-friendly, mostly flüsingle-level formulations. "Children with congenital heart defects need, for example, the gäan independent beta-blocker Metoprolol low Dosierung", says Gulde. "But since he is in this dosage, and für this purpose, not as a Fertig­medicines verfüis available, we manufacture custom formulations."

Individual advice including

Such regulations usually come from centers of expertise, or Fachädoctors who have specialized in rare diseases. Also Dennis Stracke was due to the location of his pharmacy gegenüber of the Berlin Charité motivated, intensively with these Suffering beschäbusy. "From there, patients with neurological diseases such as amyotrophic lateral sclerosis, or rare Epilepsieformen&quot come;, erlähe explained. "You müwe left kind of field."

A sechsköpfiges Team under his leadership, küGod is to meet the needs of these patients, supplies you with the recipes and accompanied them until the right dosage was found. Außerdem berät Stracke Angehörige, PflegekräRTD and Ädoctors. "We see ourselves as an interface between the patient and their treating Ädoctors are often difficult to achieve."

Zeitaufwäof incomplete Service

Grundsäin addition verfügen all pharmacies üabout the nötransparent requirements, to individual recipes. In addition, füyou do Identitätsprütests of the raw materials used and ei­ne comprehensive documentation. "There’s quite a lot of time zusammen&quot comes;, says Gulde. "However, formulations gehören to our ­basic Täactivities we do as part of our supply order."

Information für Affected, and your Angehömembered, but also für Ädoctors and therapists there are in the Alliance of Chronic Rare diseases – AXIS e. V.