Session Summary from VI HTA Symposium (11-12 June 2018)
DAY I, SESSION 3:
Innovations in healthcare
The panellists: Aleksandra Mościcka-Studzińska, Ewa Kalinka-Warzocha, Grzegorz Mączyński, Adam Krenke, Karolina Skóra, Marcin Czech
The moderator: Wojciech Szefke
The third session of the first day of the Symposium was devoted to access to modern therapies and sources of financing research and development activities in healthcare.
Emergency access to drug therapies (RDTL)
One of the mechanisms of financing innovative technologies in healthcare is emergency access to drug therapies (RDTL). This instrument may be used when, after exhausting all possible therapeutic options for a given patient, a given drug is necessary to save the patient’s health and life. A healthcare provider submits the application for funding under the RDTL mode to the Minister of Health; the application is sent along with the opinion of a national or regional consultant. The Minister of Health has 14 days to consider the application. In certain cases, the Minister of Health is obliged to ask the AOTMiT for an opinion on whether it is justified to finance the drug in the given indication from public funds. It is worth to note one of the provisions of the Act, pursuant to which, after receiving a positive opinion of the AOTMiT, the Ministry calls on the applicant to submit a reimbursement application for the drug in this indication within 90 days. This makes the patients’ access to a life-saving drug technology dependent on whether the pharmaceutical company does apply for reimbursement. Consent to cover the costs of the drug under emergency access to drug therapies is issued for a period of 3 months or three treatment cycles.
An analysis of processes under RDTL which were assessed by the AOTMiT in the period from 23 July 2017 to 6 June 2018 included 41 processes. The main therapeutic areas for which emergency access applications were concerned are haemato-oncology and oncology (over 70% of all analysed processes). The President of the Agency issued a positive opinion in 60% of cases, which is a larger percentage of positive decisions compared to the standard reimbursement procedure. In the vast majority of processes, the opinion of the President of AOTMiT and the opinion of the Transparency Council are the same. The conducted analysis did not demonstrate prevalence of the orphan status in this mode (about 50%). In addition, the amendment to the Pharmaceutical Law Act introduced a change which is negative from the patients’ perspective, excluding orphan drugs from the targeted import procedure. The biggest problem with emergency access to drug technologies is the lack of a separate budget. Currently, RDTL is financed under a lump sum, which in practice limits patients’ access to modern drug technologies, which often is the last resort.
Funds of the National Centre for Research and Development for financing innovations
Before a new product is introduced onto the market, an innovation which must be tested, proven and safe is developed. In this context it is important to look for funds which will make financing of the entire process of innovation development possible. One of the options consists in co-funding granted by the National Centre for Research and Development (NCBR). At its disposal, the Centre has structural funds from the European Commission as well as national resources from the Ministry of Science and Higher Education. The average annual funding from NCBR is PLN 4 billion, while in the years 2008-2017, a total of PLN 43 billion was allocated to support research and development works of Polish enterprises, universities and research institutes. The NCBR programme offer includes both national programmes and competitions from European funds and international programmes. In the field of health technologies, the NCBR finances over 400 projects with a total value of over PLN 3 billion, with subsidies for beneficiaries at the level of PLN 2 billion. The main programmes in the area of health protection are STRATEGMED – a strategic programme, INNOMED – the PPTIM (Polish Platform for Innovative Medicine Technology) sector programme and InnoNeuroPharm – the PZPPF (Polish Association of Pharmaceutical Industry Entrepreneurs) sector programme. The offer of international programmes is very broad and regularly updated.
Financing innovations within public-private partnerships
The introduction of innovative therapies on the market can be implemented as part of a public-private partnership (PPP). PPP allows for an effective use of the strengths of both sectors. The role of the public sector within PPP is to provide knowledge about where innovation is needed, what the needs of patients are, the concentration of experts and valuable scientists in research centres, and the justification for the concept of a new therapeutic strategy. In turn, the private sector has qualified human resources which make it possible to logistically plan clinical trials leading to obtaining marketing authorisation. Due to the chance of profiting from the marketing authorisation, the private partner may incur the financial risk of such research, which is completely unjustified for the public sector. In 2017, on the basis of cooperation with pharmaceutical companies, the first centre of early phase drug research was opened in Poland at the Institute of Oncology in Warsaw. The creation of such an entity means that Poland joins the group of countries which participate in the research of the earliest phases, and thus automatically also later phases, that way building the image of a country in which a drug can be tested from the beginning to the very end. The establishment of such a centre allows for eliminating administrative, formal, logistic and organisational barriers hindering cooperation between a scientific institution and a pharmaceutical company. Conducting studies is not possible without the public sector, which has access to patients, qualified medical staff conducting experiments and access to hospital facilities to address possible adverse effects. Thanks to clinical research, the scientific rank of the centre grows, the staff gains experience with new molecules long before the drug is granted marketing authorisation, while patients gain access to a drug which potentially is more effective than standard treatment. In addition, participation in clinical trials brings significant financial benefits to the research centre, and the staff are given the opportunity to increase their salary, which helps bring a stop to the brain-drain phenomenon. The private sector incurs the cost of statistical analysis, however, the creation of a valuable publication which meets EBM standards and is useful for HTA analyses requires the participation of clinical experts from the public sector. In conclusion, innovations in the field of new therapies are a great example in favour of PPP in Poland.
Crowdfunding and crowdsourcing
Ways to finance the development of innovative health technologies include crowdfunding and crowdsourcing. Crowdfunding is a form of financing various types of projects by a community which is, or will be, organised around these projects, while crowdsourcing is a process in which the organisation searches for ideas outside its structure. In other words, crowdfunding is about searching for resources, and crowdsourcing is looking for a solution to a defined problem. Examples of projects implemented as part of crowdfunding in the world include: “Treatment of Batten’s Disease” – searching for the causes of the disease, “Telerobotic Ultrasound Distance Imaging (TRUDI)” – financing the development of a telemedicine device to be used in care for patients with heart disease, as well as “Mind-App” – creating an application to help patients with memory deficits caused by to cancer or brain damage. The key terms in the context of crowdfunding and crowdsourcing are: the concept of “presumers”, i.e. future consumers, ready to finance future services, as well as the concept “prosumers”, i.e. consumers who want to proactively participate in the process of creating an innovative technology. The presented methods offer tools for obtaining financing necessary for research and development of medical devices, commercialisation and creating interest in new products, they enable reaching business partners and investors, are an opportunity to promote the brand, recruit the best specialists for cooperation and open the project to an international perspective.
The discussion highlighted the progress made in accessing and financing healthcare innovations in our country, at the same time noting the limited financial capacity of the state budget. One potential source of additional financial resources could be the introduction of co-payment on the hospital market, however, it should be borne in mind that this is a political issue, and additionally co-payment also raises further questions, including regarding the possibility of introducing complementary health insurance. In reference to the subject of PPP, the great potential of this type of cooperation was highlighted, which requires better and safe tools as well as more specific legal provisions guaranteeing the inviolability of the public entity’s interest. The panellists also discussed emergency access to drug technologies (RDTL), wondering whether there is a possibility to expand this idea to include medical devices, which was the subject of the initial bill. Attention was paid to the complicated system of reimbursement of medical devices, where the main problem consists is the lack of a transparent guaranteed benefits basket which would specify what can be financed outside this mode. The RDTL funding mode under the hospital lump sum has been assessed as imperfect and in need of changes.