Advocacy and Access in a Complex Marketplace

It’s now a given that access in all disease areas is complex but, a bit like George Orwell’s Animal Farm, some areas are more complex than others. None more so than human plasma-derived medicinal products (PDMPs) whose complexity has created a unique set of challenges for patients access to these treatments. PDMPs are the products of costly and complex technologies using plasma obtained from large numbers of blood or plasma donors. The plasma derivatives market is growing and is expected to reach 23 billion dollars in 2021 and the European market is currently at 3.7 billion rising to 5.1 billion by 2021[1].

Unique challenges

High global clinical demand of finished products (PDMPs)

Public and Private sector have different goals and practices regarding the collection and usage of blood or blood components (e.g. plasma)

Plasma proteins are separated into types then used according to indications

Numerous connected factors contribute to the distinctive challenges of PDMPs. Firstly, the raw material, blood derived human plasma, is a unique, complex raw material. The global demand is high and increasing plasma collection is not straightforward, causing supply tensions of finished plasma-derived products in some countries[2][3][4][5][6][7]. The two key players, the public and private sectors have a different focus; the public sector is principally focused on blood collection (used for transfusion of red cells and blood platelets) whereas the private sector is focused mainly on plasma (used to purify and concentrate a large array of human plasma proteins), and they do not agree on many policies related to plasma collection and usage[8]. Different blood plasma components are divided into numerous types such as antibodies (immunoglobulins), albumin and coagulation proteins and then further distributed according to medical need in complex acute and chronic indications spanning e.g. haematology, immunology and neurology.

Illustrative list of the main proteins coming from plasma and examples of conditions in which they are used

Type of plasma proteins

Therapeutic areas

Polyvalent Immunoglobulins (antibodies)

Immunodeficiencies; autoimmune neurological disorders; haematology

Anti-D specific Immunoglobulin

Haemolytic disease of the newborn; haematology

Concentrated specific Immunoglobulins

Specific immunological challenges

Coagulation Factor VIII

Haemophilia A

Coagulation factor IX 

Haemophilia B

Factor X

Congenital coagulation disorder

von Willebrand factor (vWF)

Congenital von Willebrand coagulation disorder

Coagulation factors preparations

Haemophilia A immunological complication (inhibitors); massive bleedings; haematological coagulation issues

Protein C

Congenital coagulation disorder

Antithrombin III

Congenital coagulation disorder

C1 Esterase inhibitor

Hereditary angioedema

Alpha-1 antitrypsine

Congenital lung disease

Fibrinogen and Thrombin

Surgical sealants

“For many of these components (e.g. immunoglobulins) there is no biotech-derived alternative, and in some cases (e.g. factor VIII) there are recombinant synthesis alternatives but these do not always represent the best clinical option for all patients”, said Prof. Cédric Hermans, Head Haemostasis and Thrombosis Unit, Haemophilia Clinic, UCL, Brussels.

Access challenges

Data production

Reimbursement system issues

Blurred guidelines on access to plasma derived products

Some specific access challenges can be highlighted. Generally speaking, healthcare authorities are willing to receive or produce more data (cost effectiveness studies, patient registries, optimal dosages, diagnosis delay…) for the usage of plasma derived medicinal products, although these are quite difficult to produce due to the number of indications, complexity of clinical usage or other hurdles. Though, it is uncertain if authorities would change their access systems based on new data. In many countries, current reimbursement system for PDMPs is based on a classical pharmaceutical model (e.g. applying linear taxes or regular price decreases) that is not customized for the complex and costly evolving biotechnologies related to the production of PDMPs[9]. In some cases conflicting national authority guidelines on plasma collection make access to the final products (PDMPs) difficult, if not impossible (for example country X cannot have access to a plasma-derived product from country Y) ⁴.

Advocacy challenges

Limited patient groups representation

Highly, fragmented, small, target populations, often rare diseases

Limited advocacy at the national and EU level for the priority challenge of plasma collection

As an example, at the EU level and in many countries in Europe there is no dedicated neurology patient advocacy group for the conditions involving the usage of immunoglobulins, although these indications (Chronic Inflammatory Demyelinating Polyneuropathy-CIDP; Guillain-Barré Syndrome-GBS; Multifocal Motor Neuropathy-MMN) account for the biggest usage of immunoglobulins in volume. The highly fragmented, advocacy environment contributes to the lack of voice for improving the access challenges. With some notable exceptions, the target patient populations are small and suffer from rare disorders. Acute indications are obviously not represented by patient groups (e.g usage of albumin for temporary intensive care). In some other case, the patient groups have limited capacity or country representation, or conversely overrepresented by two groups for the same indication diluting advocacy effort.

Fortunately some have a clear, coordinated strategic EU and national approach addressing the full spectrum of short and long-term patient issues including improving plasma collection policies.

As with all new therapeutic advances, new biotechnologies and gene therapies used in this area raise scientific interest and public interest, improving treatment options, but sometimes at the expense of the original traditional treatments which still have value.

“The multiple developments in biotechnology to treat certain plasma disorders are receiving much more attention that those made in the field of production of plasma-derived proteins. This creates an extra challenge of visibility and awareness for the plasma derivatives”, said Prof. Cédric Hermans, Head Haemostasis and Thrombosis Unit, Haemophilia Clinic, UCL, Brussels.

What can be done?

From the time of diagnosis, many patients requiring plasma-based therapies will be on a life-long intravenous injection regimen. To ensure patients continue to have access to these treatments three broad approaches can be recommended. All feature the traditional advocacy, policy informing role of patient groups.

• Facilitate the mobilization of patient groups to discuss and address key issues with compliant support from companies

• Multistakeholder workshops with patients and the medical community to create a common agenda

• Increase awareness of the current challenges related to plasma derived proteins in the general population, among health professionals and decision makers

Nick Hicks, director of Commutateur Advocacy Communications, develops strategic partnerships with patient groups. Bruno Santoni develops Government Affairs and Communication plans for organizations and companies.

References

[1] Market Insider August 2017

[2]https://marketingresearchbureau.com/list-of-reports/china-albumin-forecast-2013-2020/

[3]https://www.ft.com/content/40ba0978-6abc-11e7-bfeb-33fe0c5b7eaa

[4]https://ec.europa.eu/health/sites/health/files/blood_tissues_organs/docs/20150408_cc_report_en.pdf

[5]http://www.pharmabiz.com/ArticleDetails.aspx?aid=86664&sid=3

[6]https://www.sps.nhs.uk/wp-content/uploads/2015/05/Guideline20use20human20albumin20solution20and20demand20management20final.pdf

[7]https://www.nsd.scot.nhs.uk/Documents/clinimmumoMarch12.pdf

[8]https://ec.europa.eu/health/blood_tissues_organs/latest_updates_en

[9]https://e-news.ipopi.org/romania-immunoglobulin-shortage-affects-pid-patients/