167 General Discussion required to guarantee effectiveness. No assumptions are necessary in traditional dose-finding models, because the dose is related directly to concentration and effect. - Having chronic, broad or rare disease indications: For instance, the indications of inflammatory bowel disease, rheumatoid arthritis and malignant haematologic or oncologic disorders have chronic and expensive biopharmaceuticals in their medicinal arsenal prescribed to many patients. Additionally, rare and orphan indications (e.g., bleeding disorders or metabolic diseases, such as Fabry or Pompe) often have high priced biopharmaceuticals due to small patient groups. - Preferably exhibiting pharmacology with a linear dose–concentration relationship and an effectiveness plateau at conventional dosing: that is, the drug-target binding is saturated, and the maximum effect of the drug is achieved, respectively. 2. Research team creation If the hypothesis is verified via a quick literature scan, a multidisciplinary research team can be developed based on it. The members of this team should have research experience and preferably perform more than one role. The team should include one PhD candidate, who is the driving force for continuous output; a clinician involved in the regular care of the affected individuals who receive the biopharmaceutical; and a pharmacist with experience of using the required laboratory techniques and clinical pharmacology. Other team members who could prove beneficial to the research team include experts in the disorder (i.e., other clinicians, medical staff or researchers), the clinical laboratory (e.g., laboratory analyst), health or pharmaco-economics, and academic supervisors (e.g., postdoc, co-promotor or promotor). Furthermore, patient and public involvement (PPI) is recommended and often required by subsidising parties or journals. The PPI significantly contributes to research and probably increases its quality [57-61]. 3. Stakeholder involvement Stakeholder involvement should begin early, preferably during the conceptualisation of the hypothesis. The primary stakeholders to involve are the research departments of the hospital, university, institution, clinic or pharmacy where the research team members are employed. The members must engage with their colleagues to gain knowledge and awareness, and to use the infrastructure. Furthermore, hospital financial departments often have internal financial programmes for supporting cost-efficient research because these efforts directly benefit the hospital. 9
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