Clinical studies

Clinical studies represent a major part of a new therapy’s overall time to market and can typically run for at least six to seven years across the industry as a whole. The clinical research phases require a significant investment in time and funding.

However – and this is particularly relevant to Sobi as a rare disease specialist – for rare diseases where there is an unmet medical need, the process can be accelerated if a candidate treatment demonstrates transformative results for study patients.

Both the US Food & Drug Agency (FDA) and the European Medicines Agency (EMA) can allow such breakthrough therapies to be fast-tracked through the approval process, to be expedited through the approval process and be reviewed more quickly. Regulatory authorities can also grant longer market exclusivity to a company bringing an orphan drug to the market.

Patient-centric approach

When we conduct clinical research, we engage with patients whenever possible throughout the process: patients, as well as physicians, payers and other healthcare professionals, all have a voice in designing the studies. For example, patients are consulted on the study design concept, including the clinical design with a specific emphasis on patient-relevant measurements. Patients are also consulted on how a study is run, whether it is too burdensome to participate in, and how educational support materials can be developed to support patients and their families during the clinical study experience. By adopting this patient-led approach we aim to derive outcomes that will be meaningful and helpful to patients.

Additionally, consultation with payers is also essential during drug development to ensure that there are payer-relevant endpoints beyond efficacy, safety and tolerability. In this case, payers prefer to see comparative efficacy data vs. another agent as well as the implications for healthcare system resource use as a result of the new therapy.

Patient safety is a priority for Sobi. There are, rightly, stringent requirements regarding patient safety in clinical studies, which we support and comply with. We collect, analyse and act upon any new information about the benefits and risks of our products whether in clinical research or once a therapy is on the market.

Phases of clinical research

The Sobi pipeline involves three phases of clinical research leading up to registration. The three phases are widely regarded as standard in the pharmaceutical industry.

The same therapy will usually undergo more than one clinical study – concurrently or at a different time – particularly if it is being assessed for more than one indication.

Phase 1 clinical studies: Screening for safety

Phase 1 studies are usually the first tests of a therapy under development in healthy volunteers. These studies may typically involve about 20 to 100 volunteers, although in the case of rare diseases the numbers may differ.

The tests determine a therapy’s safety profile, including the safe dosage range, as well as how it is absorbed, distributed, metabolised and excreted, and the duration of its action. These studies may also provide preliminary evidence of whether the treatment could have therapeutic or preventive properties. As a rough guide, across the industry as a whole, phase 1 studies can typically take about up to one year.

Phase 2 clinical studies: Establishing the preliminary efficacy of the therapy in a treatment group

Phase 2 studies are slightly larger studies, usually conducted among patients who have the disease for which the therapy is intended. The studies generally involve up to several hundred patients and are controlled in design. There will usually be a placebo control group. Again, in rare diseases, the numbers may be significantly lower, and the study design may also differ.

During this phase we are seeking to understand what the ideal dosing will be for patients taking the treatment for its intended therapeutic use. As well as providing a preliminary assessment of efficacy, phase 2 studies also assess safety including potential side effects.

Due to the nature and severity of rare diseases, and as a result of consultation with the regulatory authorities, pharmaceutical companies can sometimes use the outcomes of phase 2 data to file for regulatory approval.

Phase 3 clinical studies: Final confirmation of safety and efficacy

Phase 3 studies are definitive, larger, randomised studies, the results of which are submitted to the regulatory authorities such as the FDA, EMA and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), together with results from previous studies, to obtain approval of a therapy. This phase examines the efficacy as well as the safety (adverse events) of the new therapy, compares it with other treatments or a placebo, and collects information that will allow it to be used safely. Also vital to the future use of the drug as well as assessment by payers, the study collects patient-relevant and patient-reported outcomes as well as information on healthcare resource use as a result of the new therapy.

In general across the pharmaceutical industry, phase 3 studies may involve 300 to 3,000 patients in clinics and hospitals. Again, however, this is not possible in the case of a rare disease with so few patients that a truly large-scale clinical study cannot be conducted.

Post-approval studies

After a therapy is approved and launched, studies continue into what is called phase 4, gathering real-world clinical experience and evidence about the treatment in clinical use.  In addition to prospective real-world evidence (RWE), additional data can be mined from existing data registry sources or payer claims databases to understand the patient burden of illness from a specific disease area as well as the clinical and healthcare resource-use implications of the new therapy. 

Our R&D is focused on late-stage research in haematology and immunology.