#EAPM – Orphan Regulation developments ahead of race to the White House

Greetings one and all – summer is now well and truly with us as we land in mid-August, and ahead of the weather there have been storms internationally as well, in Lebanon and Belarus, as well, of course, as the upcoming race to the White House in the US, which reaches a climax in just 88 days. On the European Alliance for Personalised Medicine (EAPM) health front, there are updates on the state of play with the Orphan Drugs Regulations, writes EAPM Executive Director Denis Horgan

Commission update on pediatric and orphan drug rules

On 11 August, the European Commission published its evaluation on the legislation for medicines for rare diseases and for children. This is the first comprehensive evaluation of the two regulations since their adoption in 2000 and 2006 respectively. They are evaluated together, given that the majority of rare diseases may appear already in children and many children’s diseases are also rare. The evaluation was conducted in line with the Commission’s Better Regulation Guidelines. It aimed to assess the strengths and weaknesses of the two regulations. The evaluation consisted of several steps, including the publication of a road map, various recent studies on paediatric medicines and on medicines for orphan diseases and extensive consultation of stakeholders. The European Commission is now reflecting on follow-up actions, and has published its staff working document evaluating EU orphan and pediatric legislation, its first major assessment of the regulation. 

Major changes

The therapeutic landscape for patients in the EU has undergone major changes. Still, considerable unmet needs remain. About 30 million European Union citizens are affected by one of the over 6000 rare diseases currently recognized. The European Union considers diseases to be rare when they affect no more than five per 10,000 people in the EU. 80% of these diseases are of genetic origin, and they are often chronic and life-threatening; almost 90% can begin in childhood. For these patients, and for more than 100 million European children, treatment was either limited or non-existent before the introduction of EU legislation on rare diseases and on medicines for children (in 2000 and 2006 respectively). That situation represented a huge unmet medical need and a significant public health challenge. There were often no medicines at all available for doctors treating patients with rare diseases. 

Children were regularly prescribed medicines indicated for adults, which had not been tested or adapted specifically for use in young patients. When these policy challenges were identified, the EU already had a well-established legislative framework for medicinal products that had developed considerably since its inception in 1965. It covered the whole life-cycle of medicines, from clinical research to post-marketing surveillance (pharmacovigilance). Its main aim was, and still is, to ensure that all medicines in the Union are authorised by demonstrating their safety, quality and efficacy before they reach patients. Decisions on product development were generally left to the market and were subject to commercial decisions driven by considerations of return on investment. 

Some points policymakers could consider: 

Since development is global, more should be done to align EU and other big markets’ regulatory practices: notwithstanding efforts and cooperation between US and EU aimed to harmonize their strategic plans in the field of orphan drugs, regulatory criteria and procedures to gain the designation, terms and classifications should be still harmonized. Aligning the criteria of prevalence and support to orphan medicines in the various jurisdictions would facilitate patient recruitment eventually at global level, so as to gain the data and the biological insights required to identify biomarkers and appropriate endpoints needed for progressing clinical development.

The economic support for clinical development costs is not set out in the orphan legislation, and the implications of this gap might be explored. Efforts could be undertaken to reduce the bureaucratic burden (eg. insurance multiple steps, costs and limitations, legal implications, ethical committees judgement, approval timelines) which notoriously limit the implementation and conduct of clinical trials in such difficult areas.

Further alignment across other regulatory areas to reinforce mutual reliance on paediatric development (eg, pediatric investigation plans in the European Union and pediatric study plans in the United States) to integrate discussions on orphan medicines for children within a global context is also planned, and, from an international perspective, the US Creating Hope Act is offering priority review vouchers for drugs specifically developed for children, while its Research to Accelerate Cures and Equity (RACE) for Children Act promoting drug development is also under way. 

A conducive regulatory environment can further support the development of medicines to treat rare diseases. Overall there is a need for joined-up regulatory process co-ordination. Better integration of regulatory pathways and better integration of regulatory systems, such as scientific tools and methods to generate evidence, would be helpful.

Pharma Framework or Strategy forward...

After being kept under wraps for a year, an expansive review of European regulations designed to spur development of drugs for rare diseases and children has found the number of medicines has increased. But at the same time, drug makers often did not address some of the most urgent needs.

The publication of the European Commission’s New Industrial Strategy for Europe in March has recently been followed by the road map to develop an EU Pharmaceutical Strategy.

The strategy is intended to create the right environment to ensure treatments and technologies actually reach the patients who need them. The Commission has identified a range of areas that need to be addressed, including market failures. The strategy will create a regulatory framework and policy landscape to promote research and technologies that will respond to the therapeutic needs of patients. 

The debates around access, availability (including shortages) and affordability of medicines are not new. The current commercial reward is insufficiently attractive to mobilize research especially in the field of paediatric oncology. The orphan reward has not had any impact on the development of new paediatric anticancer drugs. An effective evaluation should explore why the current legislation fails to remove barriers and constraints for pharmaceutical companies to develop their oncology drug in children.

Paediatric indications of orphan drugs, particularly in oncology, have not been developed consistently and in a timely manner, resulting in key challenges.

The road map itself references Council conclusions from 2016 that suggested measures to reconcile innovation with the need to ensure wide access to innovative products for unmet needs and the financial sustainability of health systems. Orphan Medicinal Products were a major focus. The European Parliament also adopted a resolution in 2017, on possible EU options for improving access to medicines. As anticipated, the roadmap focuses on the availability, sustainability and security of supply of pharmaceuticals - a need highlighted by challenges around COVID-19. 

The Commission asserts that “there is a need to build a holistic, patient-centred, forward-looking EU Pharmaceutical Strategy which covers the whole life-cycle of pharmaceutical products”. The Commission is undertaking a series of consultations with all stakeholders, including a public consultation on the strategy, and will also continue to hold targeted meetings and workshops with stakeholders.

Study reveals some 6% of people infected with COVID-19 in UK

Nearly 6% of people in England were likely infected with COVID-19 during the peak of the pandemic, researchers studying the prevalence of infections said on Thursday (13 August), millions more people than have tested positive for the disease. A total of 313,798 people have tested positive for COVID-19 in Britain, 270,971 of which have been in England, or just 0.5% of the English population. However, a study which tested more than 100,000 people across England for antibodies to the coronavirus showed that nearly 6% of people had them, suggesting that 3.4 million people had previously contracted COVID-19 by the end of the June.

The results are consistent with other surveys, such as those conducted by the Office for National Statistics, which suggest higher levels of COVID-19 in the community during the pandemic than implied by daily testing statistics. Health care and care workers were most likely to have been previously infected. Prevalence of infections appeared to be be highest in London, where 13% of people had antibodies, while minority ethnic groups were two to three times as likely to have had COVID-19 compared to white people. Prime Minister Boris Johnson had early on hailed antibody tests as a potential game-changer in tackling the pandemic. But while helpful for population studies, scientists say the margin of error for the tests makes them unreliable for use at the individual level.

Spanish region bans smoking over fears of increased COVID-19 transmission risk

The Spanish region of Galicia has effectively banned smoking in public places over concerns it increases the risk of COVID-19 transmission. It has issued a blanket ban on smoking in the street and in public places, such as restaurants and bars, if social distancing is not possible. The north-western region is the first to introduce such a measure, although others are considering following suit. It comes as Spain faces the worst infection rate in western Europe. Daily cases have risen from fewer than 150 in June to more than 1,500 throughout August. It recorded 1,690 new cases in the latest daily count on Wednesday (12 August), bringing the country's total to almost 330,000.

And that is all for this week, stay safe, stay well, and have a lovely weekend.

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