Risk mitigation in a drug’s path to market, and comparison of EMA and FDA approval processes
It can typically take 10 to 12 years or more for a new therapy to go from initial discovery to the patient. Yet far from all therapies make it all the way. The long timescales from concept to approval, and the risks involved, make getting therapies to the market an expensive and complex business.
The European Medicines Agency (EMA) and the US Food & Drug Administration (FDA) are two of the most important regulatory bodies that Sobi deals with. Both can influence markets beyond their own formal jurisdictions. Whilst the EMA and FDA share similar objectives, there are differences between the EU and US regulatory processes.
The FDA is a centralised agency operating in a single country that evaluates and monitors the efficacy, safety and tolerability of medicines and accordingly grants marketing authorisation for therapies within the borders of the US. The EMA is a centralised agency carrying out the same functions for the 27 member states of the European Union. The FDA monitors the drug development process in its own right. In the EMA, assessments are conducted by a Rapporteur and Co-Rapporteur appointed by the EMA from EU member states for each assessment; the rapporteur and co-rapporteur deliver their assessments and recommendations to the Committee for Medicinal Products for Human Use (CHMP) where the decision to recommend authorisation of a therapy for use in the EU is taken. The CHMP draws its membership from all the national regulatory agencies of the EU as well as invited observers.
The EMA and the FDA signed an initial collaboration agreement in 2003 to share confidential information on medicine assessments, which has since been extended indefinitely. The two agencies work in close cooperation as they strive to oversee and maintain safety and quality standards in a global marketplace.
The EMA, the FDA and the various other regulators have broadly similar processes. Below is a simplified summary of a typical path to market.
Pharmaceutical companies, doctors and academics research new medicines.
Thousands of compounds may be potential candidates for development as a therapy. After early testing, only an estimated 12 per cent of compounds that enter clinical studies will eventually become available in healthcare systems. 1
Developers have to demonstrate that their medicine is safe, effective and of good quality. Before testing a medicine in humans, researchers must evaluate the potential risks with its use. The three types of preclinical research are in vitro (tests in test tubes), in silico (computer modelling) and in vivo (tests on living organisms).
The developer must provide details of the way the medicine is manufactured, its effects in laboratory studies, benefits and side effects observed, how risks will be managed, and what information will be provided to patients and doctors.
If a medicine clears the preclinical studies, it can move on to clinical research – that is, research involving studies in healthy volunteers and patients. Developers must receive clearance from regulatory bodies and ethics committees before clinical research may begin.
Standards for clinical studies must be met, which cover the manufacturing and quality control of the medicine and the performance of the study including the professionals involved in the scientific testing, the selection criteria of the patients being tested, and the setting in which the clinical studies will be carried out. The clinical studies are closely monitored for product safety from the first day. Ensuring patient safety by minimising adverse events is a priority.
Patient safety monitoring, also known as pharmacovigilance (PV), is a constant activity that continues throughout the life cycle of a therapy. The World Health Organization (WHO) defines pharmacovigilance as “the science and activities relating to the detection, assessment, understanding and prevention of adverse effects or any other drug-related problem”.
Under the Sobi patient safety and pharmacovigilance system, we apply the precautionary principle and continuously monitor the benefit/risk profiles of our products. We run annual training for all our employees to raise awareness and ensure that all safety information concerning our products is reported.
While the details of regulatory approval differ between the EMA, the FDA and other authorities, the underlying principle is similar. Regulatory authorities make an assessment of the data generated during development, describing the quality, risks and benefits of the product for its recommended use. If the benefit outweighs the risk, and good quality can be assured, the therapy can be approved.
Once a therapy is granted marketing authorisation, the decision-making processes regarding health technology assessment, pricing and access will begin. In the US and Europe, the procedures are quite different.
In the US, therapy pricing and reimbursement decisions are taken across a complex set of payers varying from national/public (Centers for Medicare and Medicaid Services, or CMS), state/public (Medicaid and Medicare), payer group/private insurance funds and pharmacy benefit managers (PBMs), and local (hospitals).
In Europe, unlike the centralised regulatory procedure to grant a single European marketing authorisation, when it comes to facilitating local patient access at the EU member state level, each market has its own regulations and procedures for assessment of value based on the therapy’s evidence and value profile. Local health economic assessment follows, which considers the therapy’s relative value versus the local market’s existing treatment/standard of care to assess both the incremental value (ICER) and the impact on the healthcare system’s budget. Following this process of value assessment, a value-based price is agreed upon as well as a volume of patients who will be granted access and reimbursement for the therapy.
A value-based price reflects the benefits to patients, healthcare systems, societies and payers. Sobi is committed to giving patients sustainable access to medicines – and doing so is contingent on the long-term sustainability and affordability to health systems of our therapies.
The clinical studies are closely monitored for product safety from the first day.
Only a tiny proportion of investigational therapies in the pharmaceutical industry make it all the way to the patient. Across the pharmaceutical industry as a whole, only about one in every 5,000 to 10,000 new compounds entering R&D pipelines will make it to the market. 2
Sobi has been able to refine its therapy identification and development strategy to mitigate risk.
We follow a strategy of strengthening our R&D pipeline with candidate therapies that are in the later stages of clinical research, known as (late-stage assets), thus mitigating the risk associated with a long time to market.
We specialise in orphan drugs and serve the rare-disease market, which is characterised by high unmet medical need. Regulatory authorities such as the FDA and the EMA have put incentives in place to encourage pharmaceutical companies to develop treatments for rare diseases. These regulatory bodies have also created accelerated assessment tracks for medicines that treat diseases with high unmet need that reduce the time it takes for the treatments to reach patients.
We provide access to innovative treatments that transform life for people.
Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care.
We contribute to societies by improving access to treatment of rare diseases.
Every day, we work actively to find better ways to understand and meet patient needs.
Find out more about our business and financial performance.
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