Questions After Questions
But before a research project for a drug begins questions must be answered, such as: Which diseases are in urgent need of innovative treatments? and: Are new findings on the cause of the disease available? Answers to these questions must be sought in discussions with healthcare experts. The hunt for “targets”—like enzymes or receptors—then begins. Once a target is identified, researchers develop an array of compounds designed to address exactly this target. As a rule of thumb, only ten out of 10,000 synthesized compounds finally make it to the development stage; of these ten, only one will clear the final hurdle and end up on the pharmacist’s shelf. By the final stage, development of the drug will have involved investments of up to over one billion euros—counting all the failures along the way.
A Testing Time
After developing the compounds, researchers begin to investigate their efficacy. To do this, they conduct extensive tests on the compounds, primarily investigating whether they have harmful effects. Over five years generally elapse before the potential compounds have completed all the tests. Only compounds that have proved their worth in terms of efficacy and tolerance go on to the next stage of drug development.
Success Or Failure?
After testing, the compounds that have successfully passed the pre-clinical phase are initially trialed on a small group of healthy adult volunteers (test phase 1) to identify the stage when the drug takes effect and the dosage from which side-effects develop. The drug is then trialed for its efficacy, tolerance and dosage on around 100 to 500 voluntary patients (Phase 2), proposed by physicians. Large-scale trials are then conducted over several years involving hundreds, or even thousands of patients, to verify whether the previous findings can be reproduced over a larger number of patients (Phase 3). Schedules of trials on children are drawn up once all trials on adults are completed.
Onto the Market
Once the efficacy of a drug has been verified, the researchers collate their results and apply for approval. To do this, they pass their research data to the Federal Institute for Drugs and Medical Devices, which examines and evaluates all trial and test results. If the Institute reaches a positive conclusion, the drug can go into industrial production and distribution, and becomes available to patients.