A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem.

People with rare lysosomal storage diseases face challenges in their care that arise from disease complexity and heterogeneity, compounded by many healthcare professionals being unfamiliar with these diseases. These challenges can result in long diagnostic journeys and inadequate care. Over 30 years ago, the Rare Disease Registries for Gaucher, Fabry, Mucopolysaccharidosis type I and Pompe diseases were established to address knowledge gaps in disease natural history, clinical manifestations of disease and treatment outcomes. Evidence generated from the real-world data collected in these registries supports multiple stakeholders, including patients, healthcare providers, drug developers, researchers and regulators. To maximise the impact of real-world evidence from these registries, engagement and collaboration with the patient communities is essential. To this end, the Rare Disease Registries Patient Council was established in 2019 as a partnership between the Rare Disease Registries and global and local patient advocacy groups to share perspectives on how registry data are used and disseminated. The Patient Council has resulted in a number of patient initiatives including patient representation at Rare Disease Registries advisory boards; development of plain language summaries of registry publications to increase availability of real-world evidence to patient communities; and implementation of digital innovations such as electronic patient-reported outcomes, and patient-facing registry reports and electronic consent (in development), all to enhance patient engagement. The Patient Council is building on the foundations of industry-patient advocacy group collaboration to fully integrate patient communities in decision-making and co-create solutions for the rare disease community.

Partial stream digestion of residual municipal solid waste.

Anaerobic digestion of residual municipal solid waste (MSW) has become more important than the digestion of source separated biowaste. More than 52% of the capacity available in Europe was designed for digestion of residual municipal waste by the end of 2006, while this was only 13% in 1998. Partial digestion of residual waste organics, by which only a part of the organics is digested, has been implemented to reduce the need for dewatering and subsequent wastewater treatment. The digestate coming from part of the organics is immediately mixed with the non-digested organic fraction. This organic fraction is drier and still contains a lot of energy which can be used to dry the digestate during the aerobic composting of the mixture of digested and undigested organics. Such a MBT-plant has been operating for over a year whereby 2/3 of the organics (including sludge cake) are digested (25,000 t/year) and mixed after digestion with the remaining 1/3 of the organics. Biogas production averages 125.7 Nm2 per ton fed and contained 56.2% of methane. The mixture of digestate and non-digested organics is aerated in tunnels during 4 to 6 weeks. The stabilized end product is landfilled, meeting the stringent German standards for inert landfills. By using a dry fermentation able to produce a digestate at 35% solids, there is no need for dewatering the digestate so that no wastewater is produced.

Will anaerobic digestion of solid waste survive in the future?

Anaerobic digestion has captured a significant share of the European market for the biological treatment of the organic fraction in municipal solid waste. Almost 4 million ton per year in digestion capacity has been installed through the construction of more than 120 full-scale plants. Not all plants have been equally successful, due to poor planning, design or bad operation. This, besides higher than expected investment and operating costs, may have slowed down the growth of anaerobic digestion of solid waste. However, an evaluation of the development of anaerobic digestion over the last 15 years shows that there is now a greater diversity in application, a wider range in types of systems and suppliers, and a continued increasing rate of implementation throughout most parts of Europe. New alternative treatment techniques have not seen the same level of success as anaerobic digestion. Anaerobic digestion has been established as a viable treatment technology for the organic fraction of municipal solid waste and will most likely play an even more important role in the future.

Solid waste digestors: process performance and practice for municipal solid waste digestion.

The most common types of anaerobic digesters for solid wastes have been compared based on biological and technical performance and reliability. Batch systems have the most simple designs and are the least expensive solid waste digesters. They have high potential for application in developing countries. Two-stage systems are the most complex and most expensive systems. Their greatest advantage lies in the equalisation of the organic loading rate in the first stage, allowing a more constant feeding rate of the methanogenic second stage. Two-stage systems with biomass accumulation devices in the second stage display a larger resistance toward toxicants and inhibiting substances such as ammonia. However, the large majority of industrial applications use one-stage systems and these are evenly split between "dry" systems (wastes are digested as received) and "wet" systems (wastes are slurried to about 12% total solids). Regarding biological performance, this study compares the different digester systems in terms of organic loading rates and biogas yields considering differences in input waste composition. As a whole, "dry" designs have proven reliable due to their higher biomass concentration, controlled feeding and spatial niches. Moreover, from a technical viewpoint the "dry" systems are more robust and flexible than "wet' systems.

Anaerobic digestion of solid waste: state-of-the-art.

In order to make a correct assessment of the state-of-the-art of the technology, a study was made on the development of digestion capacity for solid waste in Europe. The study was limited to plants in operation or under construction that were treating at least 10% organic solid waste coming from market waste or municipal solid waste. A total treatment capacity for solid waste organics, excluding the tonnage used for sewage sludge and manures, evolved from 122,000 ton per year in 1990 to 1,037,000 ton available or under construction by the year 2000 in 53 plants across Europe, an increase by 750%. Both mesophilic and thermophilic technologies have been proven, with about 62% of capacity being operated at mesophilic temperatures. Wet and dry digestion are almost evenly split, while a clear choice was made for one-phase systems instead of two-phase systems, which represent only 10.6% of capacity. The capacity provided by co-digestion systems is limited, while there is a rising interest in digestion of mixed household waste. The reliable performance has been demonstrated for all types of anaerobic digestion systems. On the basis of the Dranco technology, a single-phase thermophilic dry digestion process, performances were reached similar to high-rate wastewater digestion. An annual average loading rate of 18.5 kg COD/m3.day, resulting in a biogas production of 9.2 m3/m3 reactor.day was obtained at a full-scale plant. The plant operated at a retention time of 15.3 days. Feedstocks range from clean organic wastes (31% dry matter) to heavily polluted grey waste organics (57% dry matter). Average dry matter concentrations of the digested residue of 41% were obtained.